[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Nationwide Children's Hospital\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":666},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,37,0,25,[9,54,84,111,134,155,181,205,228,266,291,317,341,361,383,416,440,464,489,512,537,567,586,616,645],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":17,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":21,"targetDuration":4,"studyType":24,"phases":25,"briefSummary":27,"conditions":28,"keywords":38,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":53},"100541748","early-phase-1-lorlatinib-for-newly-diagnosed-high-grade-glioma-with-ros-or-alk-fusion-100541748",false,"NCT06333899","Lorlatinib for Newly-Diagnosed High-Grade Glioma With ROS or ALK Fusion","A Pilot Study of Lorlatinib for Treatment of Children With Newly Diagnosed High-Grade Glioma With ROS-1 (ROS Proto-Oncogene 1, Receptor Tyrosine Kinase) or ALK (Anaplastic Lymphoma Kinase) Fusion","Inclusion Criteria:\n\n1. Patients must be ≥ 12 months and ≤ 21 years of age at the time of study enrollment on TarGeT-SCR.\n2. Diagnosis:\n\n   Patients with newly diagnosed high-grade glioma (HGG), including diffuse intrinsic pontine gliomas (DIPG), whose tumors harbor an ALK or ROS-1 fusion alteration are eligible. Patients must have had histologically verified high-grade glioma from diagnostic biopsy or resection. For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2\u002F3 of the pons, with histopathology consistent with diffuse WHO Grade 2-4. All other HGGs must be Grade 3 or 4.\n3. Disease Status:\n\n   Patients with disseminated DIPG or HGG are eligible only if the patient is to receive chemotherapy only, i.e. no craniospinal RT is intended to be given. MRI of spine must be performed if disseminated disease is suspected clinically by the treating physicians. Patients with primary spinal tumors are eligible only if the patient is to receive either chemotherapy or focal radiation therapy, i.e., no craniospinal RT is intended to be given. Patients with leptomeningeal disease only, with no definitive identifiable primary tumor, and documented ALK or ROS-1 fusion, must be discussed with the Study Chair on a case-by-case basis.\n4. Performance Level:\n\n   Karnofsky ≥ 50% for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n5. Prior Therapy:\n\n   * Patients must not have received any prior anti-cancer chemotherapy.\n   * Prior use of corticosteroids is allowed (see below Exclusion Criteria)\n6. Organ Function Requirements 6.1 Adequate Bone Marrow Function Defined as:\n\n   * Peripheral absolute neutrophil count (ANC) ≥ 1000\u002FμL\n   * Platelet count ≥ 100,000\u002FμL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)\n   * Hemoglobin \\>8 g\u002FdL (may receive transfusions) 6.2 Adequate Renal Function Defined as:\n   * Serum creatinine within normal institutional limits OR Creatinine clearance or radioisotope GFR ≥ 70ml\u002Fmin\u002F1.73 m2 6.3 Adequate Liver Function Defined as:\n   * Total bilirubin ≤ 2 × institutional upper limit of normal\n   * AST(aspartate aminotransferase)\u002FALT(alanine transaminase) ≤ 2.5 × institutional upper limit of normal 6.4 Adequate Pulmonary Function Defined as: Pulse oximetry \\> 94% on room air if there is clinical indication for determination (e.g. dyspnea at rest).\n\n6.5Adequate Cardiac Function Defined as: QTc ≤ 470 msec (by Bazett formula) 6.6 Adequate Neurologic Function Defined as: Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled.\n\n6.7 Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines\n\nExclusion Criteria:\n\n1. Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.\n\n   Females of reproductive potential must use an effective non-hormonal method of contraception, since lorlatinib can render hormonal contraceptives ineffective, during study treatment and for at least 6 months after the final dose. Males with female partners of reproductive potential must use effective contraception during treatment with lorlatinib and for 3 months after the final dose.\n2. Concomitant Medications\n\n   * Investigational Agents\u002FDrugs: Patients who have previously received or are currently receiving another investigational drug are not eligible.\n   * Anti-cancer Agents: Patients who have previously received or are currently receiving other anti-cancer agents, including chemotherapy, immunotherapy, monoclonal antibodies, biologic or targeted therapy, are not eligible\n3. Infection: Patients must not have any active, uncontrolled systemic bacterial, viral or fungal infection.\n4. Patients who have received prior solid organ transplantation are not eligible.\n5. Patients must not have malabsorption syndrome or other condition affecting oral absorption.\n6. Patients must not be receiving any treatment with a strong cytochrome P450 3A4 (CYP3A4) inhibitor or inducer. Discontinue strong CYP3A inducers for 3 plasma half-lives of the strong CYP3A inducer prior to treatment with loraltinib. Moderate inducers of CYP3A4 should be avoided\n7. Avoid concomitant use of lorlatinib with certain CYP3A substrates, for which minimal concentration changes may lead to serious therapeutic failures. If concomitant use is unavoidable, increase the CYP3A substrate dosage in accordance with approved product labeling.\n8. P-glycoprotein (P-gp) substrates: Lorlatinib is considered a moderate P-gp inducer. Co-administration of lorlatinib with P-gp substrates including but not limited to digoxin should be avoided as the concentration of these drugs may be reduced by lorlatinib.\n9. Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.\n10. Patients with a known personal history of acute or chronic severe psychiatric disorders or current history of suicidal ideation and history of suicide attempt.",true,"ALL","1 Year","21 Years",{"count":22,"type":23},15,"ESTIMATED","INTERVENTIONAL",[26],"EARLY_PHASE1","The goal of this study is to determine the response of the study drug loratinib in treating children who are newly diagnosed high-grade glioma with a fusion in ALK or ROS1. It will also evaluate the safety of lorlatinib when given with chemotherapy or after radiation therapy.",[29,30,31,32,33,34,35,36,37],"High Grade Glioma","Diffuse Intrinsic Pontine Glioma","Anaplastic Astrocytoma","Infant Type Hemispheric Glioma","Glioblastoma","Glioblastoma Multiforme","WHO Grade III Glioma","WHO Grade IV Glioma","Diffuse Midline Glioma, H3K27-altered",[39,40,29,30],"ALK fusion","ROS fusion","RECRUITING","2026-06-25",{"date":44,"type":45},"2026-06-29","ACTUAL",{"date":47,"type":45},"2025-08-03",{"date":49,"type":23},"2035-06-01",{"name":51,"class":52},"Nationwide Children's Hospital","OTHER",18,{"id":55,"slug":56,"hasResults":12,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":60,"eligibilityCriteria":61,"healthyVolunteers":12,"sex":18,"minAge":62,"maxAge":63,"enrollmentInfo":64,"targetDuration":4,"studyType":24,"phases":66,"briefSummary":68,"conditions":69,"keywords":71,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":83},"100524907","gerd-infant-feeding-therapeutics-trial-gift-trial-100524907","NCT06114836","GERD Infant Feeding Therapeutics Trial (GIFT Trial)","Pathophysiological Evidence Driven Management of GERD in Neonatal ICU Infants: Randomized Controlled Trial","GIFT","Inclusion Criteria:\n\n* NICU infants of any gestational age who are between 37 - 47 weeks postmenstrual age at inception of the study meeting the following requirements:\n\n  * GERD diagnosis using pH-impedance criteria (Acid Reflux Index ≥ 3% plus at least one of the following: # GER events \\>70 \u002F day, Symptom Associated Probability ≥ 95%, Discal Baseline Impedance \\\u003C 900 Ω)\n  * Full enteral feeds\n  * No current GERD therapies\n\nExclusion Criteria:\n\n* Known lethal chromosomal abnormalities or complex congenital syndromes\n* Severe neurologic pathologies requiring neuroactive medications or neurosurgery\n* Positive airway pressure or oxygen flow \\> 4 LPM\n* Upper gastrointestinal malformations requiring surgery","1 Day","8 Months",{"count":65,"type":23},369,[67],"NA","The goal of this investigator-initiated, single-center, randomized controlled trial (RCT) is to compare the effects of four weeks of three therapies on clinical and mechanistic outcomes based on pH-Imp testing using a three-arm parallel design in NICU infants with objective GERD diagnosis. The three therapies being compared are natural maturation, proton pump inhibitor (PPI) use, and added rice (AR) formula use. The main goals are:\n\n* to evaluate and compare the efficacy of the three commonly used treatment interventions used in the NICU for GERD in a randomized controlled manner with the primary endpoint of oral feeding success and absence of troublesome symptoms (as defined below).\n* to characterize the mechanisms of primary end point (success or failure) using pH-Impedance metrics.",[70],"GERD in Infants",[72,73,74],"Proton Pump Inhibitor","pH Impedance","added rice formula","2026-06-19",{"date":77,"type":45},"2026-06-23",{"date":79,"type":45},"2024-03-14",{"date":81,"type":23},"2028-11-01",{"name":51,"class":52},1,{"id":85,"slug":86,"hasResults":12,"nctId":87,"briefTitle":88,"officialTitle":89,"acronym":4,"eligibilityCriteria":90,"healthyVolunteers":12,"sex":18,"minAge":91,"maxAge":92,"enrollmentInfo":93,"targetDuration":4,"studyType":24,"phases":95,"briefSummary":97,"conditions":98,"keywords":4,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":103,"lastUpdatePostDateStruct":104,"startDateStruct":106,"completionDateStruct":108,"leadSponsor":110,"locationsCount":4},"100642450","phase-2-study-of-relatlimab-and-nivolumab-opdualag-in-replication-repair-deficient-hgg-and-dipg-100642450","NCT07644312","Study of Relatlimab and Nivolumab (Opdualag) in Replication Repair Deficient HGG and DIPG","Feasibility and Phase 2a Study of Relatlimab and Nivolumab (Opdualag™) in Adolescent and Young Adult Patients Newly Diagnosed With Replication Repair Deficient High-Grade Glioma (HGG), Including Diffuse Intrinsic Pontine Glioma (DIPG)","Inclusion Criteria:\n\n* Patients must be ≥12 years and ≤39 years of age at the time of enrollment on TarGeT-SCR.\n* Within the United States, patients must weight ≥ 40 kg at the time of enrollment. For all other countries, patients must weight ≥ 30 kg at the time of enrollment.\n\nDiagnosis:\n\n* Patients with newly-diagnosed HGG, including DIPG, are eligible. All patients mut have histologic confirmation from diagnostic biopsy or resection. The diagnosis of HGG, including DIPG must have been confirmed through TarGeT-SCR.\n* All HGGs must be WHO Grade 3 or 4. Note: WHO Grade 2 gliomas with pontine epicenter and diffuse involvement of at least 2\u002F3 of the pons are eligible.\n\nDisease Status:\n\n* Patients must be newly diagnosed.\n* Measurable disease is not required.\n* Patients with primary spinal tumors are eligible.\n* Patients should have no evidence of herniation or impending herniation, and no mass effect leading to severe midline shift.\n* Patients with prior malignancy are eligible.\n* Metastatic disease is excluded.\n* Disseminated or multifocal disease: discussion with Study Chairs is required. Patients with multifocal disease who received upfront CSI are not eligible.\n\nDemonstration of DNA replication repair deficiency (RRD) by fulfilling at least 2 of the following criteria:\n\n* Tumor mutational burden greater than or equal to 5 mutations\u002Fmegabase (Intermediate and high TMB)\n* Genetic diagnosis of germline Constitutional Mismatch Repair Deficiency (CMMRD), Lynch Syndrome, or Polymerase-Proofreading Deficiency (PPD)\n* Validated functional genomic assay (e.g. LOGIC) confirming mismatch repair (MMR) deficiency\n* Immunohistochemistry (IHC) demonstrating loss of protein expression of any of the 4 MMR genes\n\nPerformance Level: Karnofsky ≥ 50 for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age\n\nPrior Therapy for HGG:\n\n* Surgery, RT, and\u002For dexamethasone are permissible. Temozolomide administration is highly discouraged with concurrent RT, but permissible. Anti-VEGF treatment used for vasogenic edema or steroid weaning is permitted. No other prior anticancer therapy for HGG will be allowed. Anti-VEGF use is well-established to not impact outcomes in HGG and is not considered as oncologic treatment but rather as a supportive measure to minimize steroid use in this setting. Discussion with Study Chairs is highly recommended.\n* RT requirements: Patients must have received photon or proton focal RT and administered at a standard dose, including:\n* 54 Gy in 30 fractions for DIPG\n* 54-59.4 Gy in 30-33 fractions for other HGG\n* 45-54 Gy for primary spinal cord HGG Note: Variances in RT dose within 10% of standard doses listed above are acceptable.\n* Timing between diagnosis and start of RT: Patients must have started RT \\\u003C 42 calendar days from initial diagnosis.\n* Timing post-RT: The earliest patent can begin protocl treatment is 28 days post-completion of RT. It is recommended treatment should begin within 25 days, however patients must start treatment on TarGeT-F no later than 8 weeks post-completion of RT.\n\nOrgan Function Requirements:\n\n* ANC ≥ 1000\u002Fmm3\n* Platelet count ≥ 100,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).\n* Hemoglobin \\> 8g\u002FdL (may be transfused)\n* Creatinine clearance or radioisotope GFR≥ 70 mL\u002Fmin\u002F1.73 m2 OR serum creatinine based on age\u002Fgender as follows:\n\n  10 to \\\u003C 13 years: 1.2 mg\u002FdL for males and females 13 to \\\u003C 16 years: 1.5 mg\u002FdL for males and 1.4 mg\u002FdL for females\n  * 16 years: 1.7 mg\u002FdL for males and 1.4 mg\u002FdL for females\n* AST\u002FALT \\\u003C 3 times the ULN. For the purpose of this study, the ULN for ALT and AST is 45 U\u002FL.\n* Ejection fraction greater than or equal to 50% as measured by echocardiogram or multiple-gated acquisition\n* QTc ≤ 480 msec (by Bazett formula)\n* Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled.\n* TSH within institutional guidelines for normal range.\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding patients are excluded.\n* Patients with uncontrolled infection.\n* Patients with bone marrow failure syndrome.\n* Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.\n* Inflammatory bowel disease\n* Moderate to severe pulmonary conditions defined by need for medical intervention and\u002For limiting activities of daily living or shortness of breath with limited exertion.\n* Personal history of pneumonitis.\n* Cardiac conditions.\n* Patients with a history of severe or life-threatening adverse dermatologic reactions such as SJS or toxic epidermal necrolysis, or other severe reactions that have been associated with prior immune checkpoint inhibitor therapy.\n* Active tuberculosis\n* Active autoimmune disease requiring systemic treatment in the past 2 years\n* Chronic HBV infections with active disease\n* Personal known history of HPC who have not completed curative antiviral treatment\n* Personal known history of HIV\n* Receipt of any organ transplantation\n* Treated with other malignancy within 1 year prior to enrollment with the exception of the following: curatively treated basal cell carcinoma, squamous cell carcinoma of the skin, and in situ cervical cancer.\n* Receipt of a live or live-attenuated vaccine within 4 weeks prior to enrollment\n* Previous treatment with relatlimab\n* Patients with ongoing or clinically significant illness, medical or psychiatric conditions that, in the investigator's opinion, could affect the safety of the participant, or could impair assessment of the study's results are not eligible.","12 Years","39 Years",{"count":94,"type":23},12,[96],"PHASE2","The goal of this study is to further evaluate feasibility and tolerability of Opdualag for patients with replication repair deficient HGG, including DIPG.",[29,30,99,100,101],"WHO Grade 3 Glioma","WHO Grade 4 Glioma","Replication Repair Deficient","NOT_YET_RECRUITING","2026-06-08",{"date":105,"type":45},"2026-06-12",{"date":107,"type":23},"2027-03-01",{"date":109,"type":23},"2038-03-01",{"name":51,"class":52},{"id":112,"slug":113,"hasResults":12,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":12,"sex":18,"minAge":118,"maxAge":92,"enrollmentInfo":119,"targetDuration":4,"studyType":121,"phases":4,"briefSummary":122,"conditions":123,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":103,"lastUpdatePostDateStruct":126,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":133},"100503755","targeted-pediatric-high-grade-glioma-therapy-100503755","NCT05839379","Targeted Pediatric High-Grade Glioma Therapy","Molecularly-Guided Phase II Umbrella Trial for Children, Adolescents, and Young Adults Newly Diagnosed With High-Grade Glioma, Including Diffuse Intrinsic Pontine Glioma","Inclusion Criteria:\n\n1. Age: Patients must be ≥12 months and ≤39 years of age at the time of enrollment onto this screening protocol.\n2. Diagnosis: Patients with newly diagnosed HGG, including DIPG are eligible. Diagnosis must have histologic confirmation from biopsy or resection. The diagnosis of HGG must have been confirmed by pathology review at the local site. For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2\u002F3 of the pons, with histopathology consistent with diffuse WHO grade 2-4 glioma (eg, diffuse astrocytoma, anaplastic astrocytoma, glioblastoma, H3K27-altered diffuse midline glioma). For all other tumors, histologic grade must be WHO grade 3-4.\n3. Disease Status: There are no disease status requirements for enrollment.\n\n   * Measurable disease is not required. Patients without measurable disease are eligible.\n   * Patients with metastatic\u002Fdisseminated or multifocal disease or gliomatosis cerebri are eligible.\n   * Patients with a primary spinal tumor are eligible.\n   * Patients with secondary, radiation related HGG are eligible.\n4. Prior Therapy for HGG: Surgery, radiation, and\u002For dexamethasone are permissible. Temozolomide concurrent with radiation is permissible. Prior administration of avastin\u002Fbevacizumab is allowed (individual treatment arms have different washout period requirements, check individual arm eligibility). No other prior anticancer therapy for HGG will be allowed.\n\n   * Participants screening for assignment to TarGeT-L may not have received radiation.\n\n   Timing from surgery to start of RT: For patients who have started RT, radiation must have started \\\u003C42 days from definitive surgery or biopsy, however it is strongly recommended patients start RT within 31 days from definitive surgery (if patient had two surgeries, radiation must have started within 31 days from second surgery).\n5. Tumor Sample Availability OR results from previous molecular profiling\u002Ftargeted sequencing\n\n   * If a patient screens through OPTION #1, tumor sample in addition to normal comparator tissue (peripheral blood, saliva, or buccal swab) must be submitted for comprehensive molecular screening at the time of screening enrollment.\n   * If a patient screens through OPTIONS #2 or #3, results from previously performed molecular profiling must be submitted following enrollment. It is highly recommended that results be uploaded within 7 days of enrollment (if results are available at time of enrollment) or within 7 days of results becoming available (if pending at time of enrollment) to allow adequate time for central review.\n6. Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.\n7. Enrollment timeline: Patients are eligible to enroll on the TarGeT-SCR anytime between diagnosis and the following specific timepoints post completion of RT (if relevant)\n\n   * Patients screening through OPTION #1 are eligible to enroll anytime between diagnosis and 10 days post RT (if completing RT).\n   * Patients screening through OPTIONS #2 or #3 are eligible to enroll anytime between diagnosis and 21 days post RT (if completing RT).\n   * Participants screening for TarGeT-L (lorlatinib) are eligible to enroll on TarGeT-SCR anytime between diagnosis and 31 days post definitive surgery (to allow time for molecular review).\n\nHowever, it is important to note the following:\n\n* For treatment protocols that include targeted therapy administered concurrently with RT, patients must start treatment within 10 calendar days of starting RT.\n* For treatment protocols that only include maintenance\u002Fadjuvant therapy (no systemic therapy given concurrently with radiation), patients must start treatment by 35 days post RT\n\n  #SCREENING OPTIONS\n* OPTION1: Molecular screening through CONNECT TarGeT Clinical Testing Laboratories\n* OPTION2: Molecular screening through a national comprehensive tumor profiling program\n* OPTION3: Clinically validated targeted sequencing or focused profiling\n\nExclusion Criteria:\n\n-Tumors that do not meet HGG and DIPG diagnoses specified above","12 Months",{"count":120,"type":23},350,"OBSERVATIONAL","The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.",[29,30,31,33,34,124,125,35,36],"Diffuse Midline Glioma, H3 K27M-Mutant","Metastatic Brain Tumor",{"date":127,"type":45},"2026-06-10",{"date":129,"type":45},"2024-08-02",{"date":131,"type":23},"2034-08-28",{"name":51,"class":52},21,{"id":135,"slug":136,"hasResults":12,"nctId":137,"briefTitle":138,"officialTitle":139,"acronym":4,"eligibilityCriteria":140,"healthyVolunteers":12,"sex":18,"minAge":118,"maxAge":92,"enrollmentInfo":141,"targetDuration":4,"studyType":24,"phases":143,"briefSummary":144,"conditions":145,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":147,"lastUpdatePostDateStruct":148,"startDateStruct":150,"completionDateStruct":152,"leadSponsor":153,"locationsCount":154},"100504051","phase-2-study-of-ribociclib-and-everolimus-in-hgg-and-dipg-or-ribociclib-and-temozolomide-in-dhg-h3g34-mutant-100504051","NCT05843253","Study of Ribociclib and Everolimus in HGG and DIPG or Ribociclib and Temozolomide in DHG, H3G34-mutant","Phase 2 Study of Ribociclib-Containing Post-Radiotherapy Combinations in Pediatric and Young Adult Patients Newly Diagnosed With High-Grade Glioma, Including Diffuse Intrinsic Pontine Glioma: Ribociclib and Everolimus for HGG\u002FDIPG Which Harbor Alterations of the Cell Cycle and\u002For PI3K\u002FmTOR Pathways AND Ribociclib and Temozolomide for DHG, H3G34-mutant","TarGeT-A study strata definitions Part1: Initial Feasibility Study for the combination of ribociclib PfOS formulation with everolimus: Enrollment on this cohort will be limited to patients aged \\\u003C21 years with primary intracranial localized HGG and DIPG\n\nPart 2\n\n* Stratum A: Patients with localized, intracranial, non-pontine, and non-thalamic HGG (who do not meet criteria for strata C-D)\n* Stratum B: Patients with DIPG\n* Stratum C: Patients with primary thalamic, spinal cord, and\u002For secondary\u002Fradiation-related HGG.\n* Stratum D: Patients with metastatic\u002Fdisseminated HGG, multifocal HGG, and\u002For gliomatosis cerebri who received CSI.\n\nStratum E\n\n* Stratum E: Patients with localized DHG, H3G34-mutant.\n\nInclusion Criteria:\n\n1. Inclusion criteria already met to enroll on TarGeT-SCR (central molecular and histopathologic screening) based on:\n\n   1.1) Age: patients must be ≥12 months and ≤39 years of age at the time of enrollment on TarGeT-SCR. For the Part 1 Initial Feasibility Cohort (receiving ribociclib and everolimus) only: patients must be \\\u003C21 years of age at the time of enrollment on this protocol.\n\n   1.2) Diagnosis: patients with newly-diagnosed HGG, including DIPG are eligible. All patients must have histologic confirmation tumor tissue from diagnostic biopsy or resection, without exceptions. The diagnosis of HGG, including DIPG, must have been confirmed through TarGeT-SCR:\n   * For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2\u002F3 of the pons, with histopathology, consistent with diffuse WHO grade 2-4 glioma\n   * All other HGGs must be WHO grade 3 or 4.\n\n   1.3) Disease status: There are no disease status requirements for enrollment\n   * Patients without measurable disease are eligible.\n   * Patients with metastatic or multifocal disease or gliomatosis cerebri who received upfront CSI are eligible\n   * Patients with a primary spinal HGG are eligible\n   * Patients with secondary, radiation-related HGG are eligible.\n2. Inclusion criteria for assignment to TarGeT-A, for all strata:\n\n2.1) Presence of at least one relevant actionable somatic alteration, detailed here:\n\n* Pathogenic alterations presumed to cause activation of cell cycle:\n* Amplification of CDK4 or CDK6\n* Deletion of CDKN2A, CDKN2B, or CDKN2C\n* Amplification of CCND1 or CCND2\n* Pathogenic alterations presumed to cause activation of the PI3K\u002FmTOR pathway:\n* Deletion or mutation of PTEN\n* Mutation or amplification of PIK3CA\n* Mutation of PIK3R1\n* Deletion or mutation of TSC1 or TSC2\n* Patients with evidence of homozygous (biallelic) RB1 loss by sequencing are excluded from TarGeT-A\n* Patients whose tumors harbor other alterations suspected to activate the cell cycle and\u002For PI3K\u002FmTOR pathway could potentially also be eligible, but only following consensus recommendation by the international multidisciplinary molecular screening committee.\n* For Stratum E: H3G34 (R\u002FV) mutation\n\n2.2) Performance Level: Karnofsky ≥ 50% for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of ag. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n\n2.3) Prior Therapy for HGG:\n\n* Surgery, RT, dexamethasone are permissible. Temozolomide administered concurrently with RT is permissible. Avastin\u002Fbevacizumab use is permitted given the last dose was administered \\> 21 days prior to enrollment. No other prior anticancer therapy for HGG will be allowed.\n* Patients must have received photon or proton RT.\n* Patients must have started RT \\\u003C 42 calendar days from initial diagnosis defined as the date of diagnostic biopsy or resection. If a patient underwent 2 upfront surgeries (e.g., biopsy then resection or debulking), this is the date of the second surgery.\n* RT delivered via photon or proton beam, must have been administered at a standard dose including (54 Gy in 30 fractions for DIPG, 54-59.4 Gy in 30-33 fractions), 45 Gy-54 Gy for primary spinal disease, and\u002For 36 Gy-39.6 Gy craniospinal for patients with spinal or leptomeningeal metastatic disease with supplemental boost to 45-54 Gy for metastasis within the thecal sac and 54 Gy-60 Gy for intracranial metastasis. Any variances in the radiotherapy dose within 10% of the standard doses outlined above will be discussed with the Sponsor-Investigator to confirm eligibility prior to study enrollment.\n* Patients must enroll and start treatment No later than 35 calendar days post-completion of RT. The earliest patients can begin protocol treatment is 28 calendar days post-completion of RT.\n\n2.4) Organ Function Requirements\n\n2.4.1) Adequate Bone Marrow Function Defined as:\n\n* Peripheral absolute neutrophil count (ANC) ≥ 1000\u002Fmm3\n* Platelet count ≥ 100,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)\n* Hemoglobin \\>8 g\u002FdL (may be transfused)\n\n2.4.2) Adequate Renal Function Defined as:\n\n* Creatinine clearance or radioisotope GFR ≥ 70ml\u002Fmin\u002F1.73 m2 OR\n* Maximum serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age\u002Fgender as follows: 1 to \\\u003C 2 years=0.6 mg\u002FdL for males and females; 2 to \\\u003C 6 years=0.8 mg\u002FdL for males and females; 6 to \\\u003C 10 years= 1.0 mg\u002FdL for males and females; 10 to \\\u003C 13 years=1.2 mg\u002FdL for males and females. 13 to \\\u003C 16 years=1.5 mg\u002FdL for males and 1.4 mg\u002FdL for females.\n\n2.4.3) Adequate Liver Function Defined as:\n\n* Total bilirubin must be ≤ 1.5 times institutional upper limit of normal for age\n* AST(SGOT)\u002FALT(SGPT) ≤ 3 times institutional upper limit of normal\n* Serum albumin ≥ 2g\u002FdL\n\n2.4.4) Adequate Cardiac Function Defined as:\n\n* Ejection fraction of ≥ 50% by echocardiogram\n* QTc ≤ 450 msec (by Bazett formula)\n\n2.4.5) Adequate Neurologic Function Defined as: Patients with seizure disorder may be enrolled if well-controlled on anticonvulsants that are not strong inducers or inhibitors of CYP3A4\u002F5.\n\n2.4.6) Adequate Pulmonary Function Defined as: No evidence of dyspnea at rest, and a pulse oximetry \\>94% on room air if there is clinical indication for determination.\n\n2.5) Ability to take medications by mouth: For ribociclib and everolimus strata, patients must be able to take study medications by mouth as administration via NG\u002FNJ\u002FG tube is not allowed.\n\n2.6) Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines\n\n2.7) Contraception: Male and female patients of childbearing potential must be willing to use a highly effective contraception method.\n\nExclusion Criteria\n\n1. Pregnant or Breast-Feeding Pregnant or breast-feeding women will not be entered on this study due to known potential risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are post-menarchal. Patients of childbearing or child fathering potential must agree to use at least one highly effective method of contraception while being treated on this study and for 3 months after completing therapy. A woman is considered of childbearing potential if she is fertile, following menarche and until becoming post-menopausal unless permanently sterile. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient. A man is considered fertile after puberty unless permanently sterile by bilateral orchidectomy. Male participants should refrain from sperm donation throughout the duration of treatment and for 3 months after completion of therapy\n\n   A highly effective contraception method is defined as one that results in a low failure rate (\\\u003C1% per year) when used consistently and correctly. The following are considered highly effective contraception methods:\n   * Combined estrogen and progesterone containing hormonal contraception associated with inhibition of ovulation.\n   * Progesterone-only hormonal contraception associated with inhibition of ovulation.\n   * Intra Uterine Device (IUD)\n   * Intra Uterine hormone releasing system\n   * Bilateral tubal occlusion\n   * Vasectomized partner\n   * Sexual abstinence (avoiding having heterosexual intercourse) The following contraceptive measures are NOT considered effective\n   * Progesterone-only hormonal contraception (birth control pill) that that does NOT stop ovulation\n   * Male or female condom with or without spermicide\n   * Cap, diaphragm or sponge with spermicide\n2. Concomitant Medications\n\n   * Patients receiving corticosteroids are eligible. The use of corticosteroids must be reported.\n   * Patients who are currently receiving another investigational drug are not eligible.\n   * Patients who are currently receiving other anti-cancer agents are not eligible, with the exception of temozolomide given concurrently with RT only.\n   * Patients who are receiving enzyme inducing anticonvulsants that are strong inducers or inhibitors of CYP3A4\u002F5 are not eligible.\n   * Patients who are receiving strong inducers or inhibitors of CYP3A4\u002F5 are not eligible and should be avoided from 14 days prior to enrollment to the end of the study.\n   * Patients who are receiving medications known to prolong QTc interval are not eligible.\n   * Patients who are receiving therapeutic anticoagulation with warfarin or other coumadin-derived anticoagulants are not eligible. Therapy with heparin, low molecular weight heparin (LMWH), or fondaparinux is allowed as long as the patient has adequate coagulation defined as aPTT \\\u003C 1.5Xs ULN and INR \\\u003C 1.5.\n3. Patients who have an uncontrolled infection are not eligible.\n4. Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study are not eligible.\n5. Patients with known clinically significant active malabsorption syndrome or other condition that could affect absorption are not eligible.\n6. Patients with prior or ongoing clinically significant medical or psychiatric condition that, in the investigator's opinion, could affect the safety of the subject, or could impair the assessment of study results are not eligible.",{"count":142,"type":23},120,[96],"The goal of this study is to determine the efficacy of the 1) ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K\u002FmTOR) that these drugs target or 2) ribociclib and temozolomide to treat pediatric and young adult patients newly diagnosed with diffuse hemispheric glioma (DHG), H3G34-mutant.\n\nThe main question the study aims to answer is whether the combinations of ribociclib and everolimus or ribociclib and temozolomide can prolong the life of patients diagnosed with HGG\u002FDIPG or DHG H3G34-mutant.",[29,30,31,33,34,124,125,35,36,146],"Diffuse Hemispheric Glioma, H3 G34-Mutant","2026-05-27",{"date":149,"type":45},"2026-05-29",{"date":151,"type":45},"2024-08-22",{"date":131,"type":23},{"name":51,"class":52},20,{"id":156,"slug":157,"hasResults":12,"nctId":158,"briefTitle":159,"officialTitle":160,"acronym":4,"eligibilityCriteria":161,"healthyVolunteers":12,"sex":18,"minAge":118,"maxAge":92,"enrollmentInfo":162,"targetDuration":4,"studyType":24,"phases":164,"briefSummary":165,"conditions":166,"keywords":172,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":173,"lastUpdatePostDateStruct":174,"startDateStruct":176,"completionDateStruct":178,"leadSponsor":180,"locationsCount":154},"100580544","phase-2-act001-for-the-treatment-of-diffuse-intrinsic-pontine-gliomas-and-h3k27-altered-high-grade-gliomas-100580544","NCT06838676","ACT001 for the Treatment of Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas","A Phase II Trial of ACT001 in Children and Adolescents With Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas","Inclusion Criteria:\n\n1. Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.\n2. Diagnosis:\n\n   * Cohort A: Newly Diagnosed DIPG\n\n     * Patients with newly-diagnosed DIPG with typical MRI findings (tumors with a pontine epicenter and diffuse involvement of at least 2\u002F3 of the pons) with or without biopsy and have completed radiation therapy (RT) within 28 to 35 calendar day prior to start of therapy.\n     * Patients must have started RT \\\u003C42 calendar days from radiographic diagnosis (for non-biopsied DIPG patients only) or definitive surgery, whichever is later.\n\n       * If a biopsy was performed, the date of surgical biopsy will be considered the date of definitive diagnostic surgery; if a patient underwent two upfront surgeries \\[e.g., biopsy then debulking\\], this is the date of the second surgery)\n   * Cohort B: progressive\u002Frecurrent DIPG or H3K27-altered HGG OR refractory disease\n\n     * Patients with DIPG (no biopsy required), pathologically-confirmed (at diagnosis or recurrence) H3K27-altered DIPG, or extra-pontine H3K27-alteredHGG who have progressive\u002Frecurrent or refractory disease\n     * Progressive\u002Frecurrent: patients who have progressive or recurrent disease following frontline treatment must have included at least focal RT. New lesions since completion of frontline RT qualify as progressive disease.\n     * Refractory disease is defined as: Presence of persistent, measurable, abnormality on conventional MRI that is further distinguished by histology or advanced imaging, OR as determined by the treating physician and discussed with the Study Chair(s) prior to enrollment.\n     * Patients with H3K27-altered spinal HGG are eligible.\n     * Patients with metastatic disease are eligible.\n3. Disease Status\n\n   * Cohort A: patients may have any disease status but must have completed initial radiation therapy (RT) before enrollment.\n   * Cohort B: Patients must have measurable disease assessable by MRI. Patients may have extra neuronal disease.\n4. Performance Level: Karnofsky Performance Scale score ≥ 50% for patients \\> 16 years of age and Lansky Performance Scale score \\> 50% for patients ≤ 16 years of age (applies to all patients) Note: Patients who are unable to walk because of paralysis, but who are capable of using a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n5. Prior anti-cancer therapy:\n\n   * For Cohort A ONLY:\n   * Surgery, radiation (focal to disease) and\u002For steroids (dexamethasone with goal to wean dexamethasone throughout protocol therapy) are permissible. Temozolomide administered concurrently with RT is permissible. Bevacizumab use is permitted given the last dose was administered \\>\u002F= 21 days prior to enrollment. No other prior anticancer therapy for DIPG will be allowed.\n   * Patients must enroll and start treatment on study between 28 and 35 calendar days post-completion of RT.\n   * Patients must have started RT \\\u003C42 calendar days of initial diagnosis (defined as the date of diagnostic biopsy or resection; if a patient underwent two upfront surgeries \\[e.g., biopsy then resection or debulking\\], this is the date of the second surgery).\n   * Radiotherapy must have been administered at standard dose of 54 Gy for DIPG patients. Any variances in the radiotherapy dose within 10% of the standard doses outlined above will be discussed with the Study Chair to confirm eligibility prior to study enrollment.\n   * For Cohort B ONLY: Patients must have fully recovered from the acute treatment related toxicities (defined as \\\u003C\u002F= Grade 1 if not defined in eligibility criteria) of all prior chemotherapy, immunotherapy, radiotherapy, or any other treatment modality prior to entering on this study, with the exception of alopecia.\n\n   Notes to the above for Cohort B: Patients with chronic Grade 2 toxicities may be eligible per discretion of the Investigator and Sponsor (e.g., Grade 2 chemotherapy-induced neuropathy). Grade 2 or 3 toxicities from prior anti-tumor therapy that are considered irreversible - defined as having been present and stable for \\> 6 months (such as ifosfamide-related proteinuria) may be allowed if they are not otherwise described in the exclusion criteria AND there is agreement to allow by both the Investigator and Sponsor.)\n\n   The wash out period between the prior anti-cancer chemotherapy, and enrollment must be:\n   1. Myelosuppressive chemotherapy: At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).\n   2. Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g. Neulasta) or 7 days for short-acting growth factor.\n   3. Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.\n   4. Immunotherapy: At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines.\n   5. Monoclonal antibodies: \\> 21 days must have elapsed from the infusion of last dose of antibody\n   6. Radiation therapy:\n\n      * All Cohort B patients: Patients must have received their last fraction of focal irradiation to new sites of progressive disease \\> 14 days prior to enrollment.\n\n        * Patients who received CSI must have received their last fraction \\> 3 months prior to enrollment.\n      * Progressive\u002Frecurrent disease: Patients who received re-irradiation to primary disease must have received their last fraction \\> 3 months prior to study enrollment.\n      * Refractory Disease:\n\n        * Patients must have completed frontline RT \\> 6 months prior to enrollment.\n        * Patients who received re-irradiation to primary disease must have received their last fraction \\> 3 months prior to study enrollment.\n   7. Stem Cell Transplant: Patients must be ≥ 3 months since autologous stem cell transplant. Patients who received allogenic stem cell transplant or solid organ transplant are not eligible for study\n6. Organ Function Requirements (applies to all patients)\n\n   1. Adequate bone marrow function defined as:\n\n      * Peripheral absolute neutrophil count (ANC) \\> 1000\u002Fmm³\n      * Platelet count \\> 100,000\u002Fmm³ (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)\n   2. Adequate renal function defined as:\n\n      * Creatinine clearance or radioisotope GFR ≥ 70 mL\u002Fmin\u002F1.73 m² or\n      * A serum creatinine based on age\u002Fgender as follows (Schwartz et al. J. Peds, 106:522, 1985): Age Maximum Serum Creatinine (mg\u002FdL) Male Female 1 to \\\u003C 2 years 0.6 0.6 2 to \\\u003C 6 years 0.8 0.8 6 to \\\u003C 10 years 1.0 1.0 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.4 ≥ 16 years 1.7 1.4\n   3. Adequate liver function defined as:\n\n      * total bilirubin must be \\\u003C\u002F=1.5X institutional ULN for age\n      * AST (serum glutamic-oxaloacetic transaminase \\[SGOT\\]) \u002F ALT (serum glutamic-oxaloacetic transaminase \\[SGPT\\]) ≤ 2.5 × institutional upper limit of normal\n      * Serum albumin ≥ 2 g\u002FdL\n   4. Adequate cardiac function defined as:\n\n      * Ejection fraction of ≥ 50% by echocardiogram\n      * QTc ≤ 450 msec (by Bazett formula)\n   5. For Cohort B: Adequate neurologic function defined as:\n\n      * Patients with seizure disorders may be enrolled if seizures are well- controlled. Well controlled is defined by no increase in seizure frequency in the 7 days prior to enrollment.\n      * Patients with neurological deficits should have deficits that are stable for at least the 7 days prior to enrollment.\n7. Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.\n8. Absence of other clinically significant concomitant active medical disorder, based on the investigator's judgement.\n\nExclusion Criteria:\n\nA patient who meets any of the following exclusion criteria will not be eligible in the study (Applies to both cohorts except where noted below):\n\n1. Cohort A only: Patients with metastatic disease.\n2. Concomitant medications:\n\n   * Corticosteroids:\n\n     * Cohort A - Patients receiving corticosteroids are eligible regardless of dosing\n     * Cohort B - Patients receiving corticosteroids who have been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are eligible\n   * Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible (Refer study inclusion criteria relating to anti-cancer therapies)\n\n     * Cohort A - Patients that have received any anti-cancer treatment other than surgery, RT, temozolomide concurrent with RT, and\u002For previous bevacizumab with appropriate washout period are not eligible.\n   * Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible.\n   * Anticonvulsants should be used as clinically indicated. The use of enzyme inducing anticonvulsants is not permitted\n   * LHRH agonist \u002F antagonists are not permitted\n   * High Dose Biotin (B7) supplements are not permitted\n3. Concomitant medications used with caution: selective serotonin reuptake inhibitor (SSRI) such as Lexapro, Fluoxetine (Prozac), fluvoxamine (Luvox), paroxetine (Paxil), sertraline (Zoloft), citalopram (Celexa), and escitalopram should be used with caution.\n4. Infection: Patients who currently have an uncontrolled infection (in the opinion of the PI) are not eligible.\n5. Patients who have received a prior solid organ transplantation are not eligible.\n6. Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are postmenarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.\n7. Patients of childbearing or child fathering potential must agree to use adequate contraceptive methods (hormonal or barrier method of birth control; abstinence) while being treated on this study and for 3 months after completing therapy. Note: The definition of effective contraception will be based on the judgement of the principal investigator or a designated associate.\n8. Patients who are in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.\n9. Patients who have previously received either ACT001 or parthenolide are not eligible.",{"count":163,"type":23},60,[96],"This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.",[167,168,169,170,171],"Diffuse Intrinsic Pontine Gliomas (DIPG)","Progressive DIPG","Refractory DIPG","Recurrent DIPG","H3K27-altered High Grade Glioma",[29,30],"2026-05-13",{"date":175,"type":45},"2026-05-15",{"date":177,"type":45},"2025-07-10",{"date":179,"type":23},"2035-07",{"name":51,"class":52},{"id":182,"slug":183,"hasResults":12,"nctId":184,"briefTitle":185,"officialTitle":186,"acronym":4,"eligibilityCriteria":187,"healthyVolunteers":12,"sex":18,"minAge":188,"maxAge":92,"enrollmentInfo":189,"targetDuration":4,"studyType":24,"phases":190,"briefSummary":191,"conditions":192,"keywords":194,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":196,"lastUpdatePostDateStruct":197,"startDateStruct":199,"completionDateStruct":201,"leadSponsor":203,"locationsCount":204},"100446672","phase-2-pep-cmv-vaccine-targeting-cmv-antigen-to-treat-newly-diagnosed-pediatric-hgg-and-dipg-and-recurrent-medulloblastoma-100446672","NCT05096481","PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent Medulloblastoma","Phase 2 Trial of a Novel Peptide Vaccine (PEP-CMV) Targeting CMV Antigen for Newly Diagnosed Pediatric High-grade Glioma and Diffuse Intrinsic Pontine Glioma and Recurrent Medulloblastoma","Inclusion Criteria for patients with recurrent \u002Fprogressive medulloblastoma (stratum I)\n\n1. Age: Patients must be ≥3 and ≤39 years of age at the time of study enrollment\n2. Diagnosis: Patients must have a diagnosis of medulloblastoma that is recurrent, progressive or refractory. All patients must have histological verification of a medulloblastoma, at original diagnosis or relapse.\n\n   • Patients must have measurable disease defined as a lesion that can be measured in two perpendicular diameters on MRI.\n3. Metastatic Disease: Patients with M+ disease are eligible.\n4. Performance Status:\n\n   Karnofsky ≥ 50% for patients \\>16 years of age or Lansky ≥ 50 for patients ≤ 16 years of age. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n5. Prior Therapy:\n\n   1. Radiotherapy: prior radiotherapy requirements Patients must have received prior disease-directed therapy including radiotherapy for their initial diagnosis of medulloblastoma unless patients are less than 4 years of age at the time of enrollment.\n\n      For those less than 4 years of age at the time of enrollment, prior disease directed therapy does not have to include prior radiotherapy.\n\n      Patients must have had their last fraction of:\n      * Craniospinal irradiation, total body irradiation or radiation to ≥ 50% of pelvis \\> 3 months prior to enrollment.\n      * Focal irradiation \\> 4 weeks prior to enrollment\n   2. Myelosuppressive anticancer therapy: Patients must have received their last dose of myelosuppressive anticancer therapy at least 21 days prior to enrollment\n   3. Immunotherapy: Patients must have received their last dose of any immunotherapy agents at least 30 days prior to enrollment\n   4. Non-myelosuppressive anticancer agents: Patients must have received their last dose of non-myelosuppressive anticancer agents at least 7 days prior to study enrollment.\n   5. Antibodies: Patients must have received their last dose of any antibodies at least 21 days prior to enrollment.\n   6. Hematopoietic growth factors: Patients must have received their last dose of hematopoietic growth factors at least 14 days prior to enrollment for a long-acting growth factor (e.g. pegfilgrastim) or 7 days prior to enrollment for short-acting growth factor.\n   7. Autologous stem cell infusion: At least 90 days must have elapsed after an autologous stem cell infusion\n6. Organ Function Requirements:\n\n   1. Adequate bone marrow function defined as:\n\n      * ANC (Absolute neutrophil count) ≥ 1000\u002Fµl.\n      * Platelets ≥ 100,000\u002Fµl. (may be supported)\n      * Hemoglobin \\> 8 g\u002FdL. (may be supported)\n   2. Adequate Renal Function defined as: Creatinine clearance or radioisotope GFR ≥ 70ml\u002Fmin\u002F1.73 m2 or A serum creatinine based on age\u002Fgender as follows:\n\n      Age: Maximum Serum Creatinine (mg\u002FdL)\n      * 2 to \\\u003C 6 years: 0.8 (Male) 0.8 (Female)\n      * 6 to \\\u003C 10 years: 1 (Male) 1 (Female)\n      * 10 to \\\u003C 13 years: 1.2 (Male) 1.2 (Female)\n      * 13 to \\\u003C 16 years: 1.5 (Male) 1.4 (Female)\n      * ≥ 16 years: 1.7 (Male) 1.4 (Female)\n   3. Adequate Liver Function Defined as\n\n      * Total bilirubin ≤1.5 times institutional ULN\n      * AST(SGOT) ≤3 × institutional upper limit of normal\n      * ALT(SGPT) ≤3 × institutional upper limit of normal\n   4. Adequate Neurological Function Defined as\n\n      * Patients with neurological deficits should have deficits that are stable for a minimum of 2 weeks prior to enrollment.\n      * Patients with current seizure disorders may be enrolled if seizures are well- controlled on antiepileptic therapies.\n   5. Patients of childbearing or child-fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study and for three months after drug cessation.\n   6. Signed informed consent according to institutional guidelines must be obtained prior to enrollment.\n\nInclusion Criteria for Patients with Newly-Diagnosed High-Grade Gliomas (HGG) (stratum II, NOT CURRENTLY ENROLLING) and Newly-Diagnosed (DIPG) (stratum III)\n\n1. Age: Patients must be ≥3 and ≤39 years of age at the time of study enrollment\n2. Diagnosis\n\n   1. Stratum II (NOT CURRENTLY ENROLLING): patients must have histologically confirmed, newly-diagnosed HGG (such as anaplastic astrocytoma, glioblastoma, H3K27-altered DMG).\n\n      * Patients with a newly-diagnosed HGG must enroll within 6 weeks of their final dose of standard radiation therapy with or without chemotherapy.\n      * Patients with primary spinal cord tumors are eligible\n   2. Stratum III: Patients with a newly-diagnosed DIPG:\n\n      * Patients with a radiographically typical DIPG, defined as a tumor with a pontine epicenter and diffuse involvement of more than 2\u002F3 of the pons, are eligible without histologic confirmation.\n      * Patients with brainstem lesions that do not meet these radiographic criteria will be eligible if there is histologic confirmation of an infiltrating astrocytoma WHO grades II-IV.\n3. Metastatic Disease: Patients with M+ disease are eligible.\n4. Performance Status:\n\n   Karnofsky ≥ 50 for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n5. Prior Therapy requirements: Patients must have received no prior therapy other than surgery, radiation, chemotherapy during radiotherapy and\u002For steroids (dexamethasone with goal to wean dexamethasone throughout protocol therapy).\n\n   Patients with a newly diagnosed high-grade glioma or DIPG must enroll within 6 weeks of their final dose of standard of care radiation therapy with or without chemotherapy.\n   1. Patients with HGG or DIPG are permitted, but not required, to have received chemotherapy during radiation. Bevacizumab is permitted prior to enrollment in patients with DIPG or HGG. Patients must have received their last dose of bevacizumab at least 14 days prior to enrollment.\n   2. For HGG patients, Patients must have received radiotherapy at a standard dose of 54-59.4 Gy in 1.8 Gy fractions for approximately 6 weeks with an acceptable variance of 10%. Radiation therapy must have begun no later than 42 days after the date definitive surgery.\n   3. For patients with DIPG, Patients must have received radiotherapy at a standard dose of radiotherapy of 54 Gy in 1.8 Gy daily fractions for approximately 6 weeks with an acceptable variance rate of 10%. Radiation therapy must have begun no later than 42 days after the date of radiographic diagnosis or biopsy\n   4. For patients with spinal cord HGG: Patients must have received radiotherapy at a standard dose of 45-54 Gy in 1.8 Gy fractions for approximately 6-7 weeks with an acceptable variance of 10%\n   5. For patients with metastatic disease: Patients may have received standard dose CSI\n6. Organ Function Requirements:\n\n   1. Adequate bone marrow function defined as • ANC (Absolute neutrophil count) ≥ 1000\u002Fµl.\n\n      • Platelets ≥ 100,000\u002Fµl. (may be supported)\n\n      • Hemoglobin \\> 8 g\u002FdL. (may be supported)\n   2. Adequate Renal Function defined as: Creatinine clearance or radioisotope GFR ≥ 70ml\u002Fmin\u002F1.73 m2 or A serum creatinine based on age\u002Fgender as follows:\n\n      Age: Maximum Serum Creatinine (mg\u002FdL)\n\n      • 2 to \\\u003C 6 years: 0.8 (Male) 0.8 (Female)\n\n      • 6 to \\\u003C 10 years: 1 (Male) 1 (Female)\n\n      • 10 to \\\u003C 13 years: 1.2 (Male) 1.2 (Female)\n\n      • 13 to \\\u003C 16 years: 1.5 (Male) 1.4 (Female)\n\n      • ≥ 16 years: 1.7 (Male) 1.4 (Female)\n   3. Adequate Liver Function Defined as:\n\n      • Total bilirubin ≤1.5 times institutional ULN\n      * AST(SGOT) ≤3 × institutional upper limit of normal\n      * ALT(SGPT) ≤3 × institutional upper limit of normal\n   4. Adequate Neurological Function Defined as:\n\nPatients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.\n\nd. Patients of childbearing or child-fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study.\n\ne. Signed informed consent according to institutional guidelines must be obtained prior to registration and for 3 months after drug cessation.\n\nExclusion Criteria for all strata:\n\n1. Pregnancy or Breast-Feeding:\n\n   1. Pregnant or breast-feeding women will not be entered on this study due to known or unknown risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are post-monarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.\n   2. Pregnancy Prevention Patients of childbearing or child fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study and for 3 months after drug cessation.\n2. Study Specific:\n\n   1. Active infection requiring treatment\n   2. Patients with malignancy related to HIV or solid organ transplant: known history of HIV, HBV surface antigen positivity or positive HCV antibody are not eligible. Viral testing is not required unless clinically indicated in patients without a known history\n   3. Known immunosuppressive disease\n   4. Patients with active renal, cardiac (congestive cardiac failure, myocardial infarction, myocarditis), or moderate to severe pulmonary problems generally defined by need for medical intervention (e.g., oxygen, medications) and\u002For limiting activities of daily living (generally CTCAE Grade 2 or higher) or shortness of breath with limited exertion are not eligible. Pulmonary conditions include (but are not limited to) COPD, asthma, and hemi-pneumectomy\n   5. Patients receiving concomitant immunosuppressive agents for medical conditions; inhaled corticosteroids for asthma are allowed.\n   6. Patients receiving concomitant tumor-directed therapy\n   7. Patients receiving any other investigational drug therapy.\n   8. Patients on dexamethasone \\> 0.1 mg\u002FKg\u002Fday up to maximum dose of 4 mg\u002Fday or equivalent.\n   9. Patients with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction).\n   10. Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy\n   11. Patients at high risk for imminent neurologic decline due to extensive bulk disease, midline shift, or herniation on MRI. These patients should be discussed with the study chairs.","3 Years",{"count":142,"type":23},[96],"This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly-diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB).\n\nPEP-CMV is a vaccine mixture of a peptide referred to as Component A. Component A is a synthetic long peptide (SLP) of 26 amino acid residues from human pp65. The SLPs encode multiple potential class I, class II, and antibody epitopes across several haplotypes. Component A will be administered as a stable water:oil emulsion in Montanide ISA 51.\n\nFunding Source - FDA OOPD",[29,30,193],"Recurrent Medulloblastoma",[29,30,193,195],"Immunotherapy","2026-05-07",{"date":198,"type":45},"2026-05-11",{"date":200,"type":45},"2024-07-18",{"date":202,"type":23},"2030-06-15",{"name":51,"class":52},13,{"id":206,"slug":207,"hasResults":12,"nctId":208,"briefTitle":209,"officialTitle":210,"acronym":4,"eligibilityCriteria":211,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":92,"enrollmentInfo":212,"targetDuration":4,"studyType":24,"phases":214,"briefSummary":215,"conditions":216,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":219,"lastUpdatePostDateStruct":220,"startDateStruct":222,"completionDateStruct":224,"leadSponsor":226,"locationsCount":227},"100457192","phase-2-actemra-for-the-treatment-of-pediatric-adamantinomatous-craniopharyngioma-100457192","NCT05233397","ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma","Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (Tocilizumab) for the Treatment of Progressive\u002FRecurrent Pediatric Adamantinomatous Craniopharyngioma","Inclusion Criteria:\n\n1. Age: Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.\n2. Diagnosis: Patients with histologically-confirmed adamantinomatous craniopharyngioma (ACP) Histologic confirmation of ACP may be made on solid tumor or, if no solid tumor can be safely obtained, cyst fluid with classic ACP characteristics of thick, cholesterol-rich, greenish-brown liquid in the context of imaging features consistent with craniopharyngioma, including lobulated, cystic\u002Fsolid mass with calcifications that originates in the sellar\u002Fsuprasellar region.\n3. Disease Status: Patients must have measurable disease.\n\n   * Stratum 1: Patients with progressive or recurrent ACP who demonstrate cystic and\u002For solid recurrence or progression at least 6 months post completion of radiation therapy\n   * Stratum 2 (CLOSED): Patients with measurable ACP who have undergone surgery but have NOT previously undergone irradiation (but may have received prior systemic or intracystic therapy). Progressive disease is allowed but not required.\n4. Performance Level: Karnofsky ≥ 50% for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Note: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n5. Prior Therapy: Patients must have recovered or stabilized from the acute toxic effects of prior treatments\n\n   * Biologic (anti-neoplastic agent): At least 7 days must have elapsed after the last (systemic or intracystic) dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair\n   * Immunotherapy: At least 42 days after the completion of any type of systemic immunotherapy, e.g. tumor vaccines.\n   * Monoclonal antibodies: At least 21 days after the last dose of a monoclonal antibody.\n   * Radiation therapy: Patients must have had their last (conventional or hypofractionated) fraction of: a) Focal irradiation \\> 6 months prior to enrollment and b) No prior craniospinal irradiation is permitted.\n   * Corticosteroids: Patients receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment\n   * Myelosuppressive systemic therapy: At least 21 days must have elapsed after the last systemic myelosuppressive therapy.\n   * Surgery: At least 6 weeks must have elapsed since major or intermediate surgery. Major surgery includes major craniotomy for tumor resection or cyst fenestration, organ resection, exploratory laparotomy. Intermediate procedures include ventriculoperitoneal shunt placement, stereotactic brain biopsy and intraventricular catheter placement. Minor procedures that are not excluded include skin biopsy\u002Fincision and drainage, bone marrow aspirate, and central venous catheter placement. Ommaya aspirations and Lumbar Punctures are considered minor procedures..\n6. Organ Function Requirements\n\n   Adequate Bone Marrow Function Defined as:\n   * Peripheral absolute neutrophil count (ANC) ≥1000\u002Fmm3\n   * Platelet count ≥100,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)\n   * Hemoglobin \\>8 g\u002FdL (may be transfused)\n\n   Adequate Renal Function Defined as:\n   * Creatinine clearance or radioisotope GFR \\> 70ml\u002Fmin\u002F1.73 m2 or\n   * A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age\u002Fgender as follows:\n\n     1 to \\\u003C 2 years: maximum serum creatinine 0.6 mg\u002FdL for males and females. 2 to \\\u003C 6 years: maximum serum creatinine 0.8 mg\u002FdL for males and females. 6 to \\\u003C 10 years: maximum serum creatinine 1.0 mg\u002FdL for males and females. 10 to \\\u003C 13 years: maximum serum creatinine 1.2 mg\u002FdL for males and females. 13 to \\\u003C 16 years: maximum serum creatinine 1.5 mg\u002FdL for males and 1.4 mg\u002FdL for females.\n\n     ≥ 16 years: maximum serum creatinine 1.7 mg\u002FdL for males and 1.4 mg\u002FdL for females.\n\n   Adequate Liver Function Defined as:\n   * Total bilirubin within normal institutional limits\n   * AST (SGOT) ≤ 2.5 × institutional upper limit of normal\n   * ALT (SGPT) ≤ 2.5 × institutional upper limit of normal\n\n   Adequate Neurologic Function Defined as:\n   * Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.\n   * Patients with current seizure disorders may be enrolled if seizures are well-controlled on antiepileptic therapies.\n7. Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.\n\nExclusion Criteria:\n\n1. Pregnancy or Breast-Feeding: Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for at least 90 days after discontinuation of drug for females and at least 60 days for males. For females of childbearing potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods (bilateral tubal ligation, male sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices; hormonal contraceptive methods must be supplemented by a barrier method) and agreement to refrain from donating eggs are required. For males of reproductive potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm.\n2. Gastrointestinal Disease: Patients with a history of serious gastrointestinal disease, including inflammatory bowel disease or gastrointestinal perforation\n3. Concomitant Medications\n\n   * Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.\n   * Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible.\n   * Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible.\n4. Study Specific:\n\n   * Patients who have an uncontrolled infection are not eligible.\n   * Patients who have received any live or attenuated vaccinations within three months prior to start of therapy are not eligible.\n   * Any significant concurrent medical or surgical condition that would jeopardize the patient's safety or ability to complete the study, including, but not limited to, disease of the nervous, renal, hepatic, cardiac (such as symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), pulmonary, or endocrine system\n   * Patients who have a history of Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus or Tuberculosis infection are not eligible.\n   * Patients who have received a prior solid organ transplantation are not eligible.\n   * Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.\n   * Patients who have a history of alcohol, drug, or chemical abuse within 6 months of screening.\n   * Patients who have had major or intermediate surgery within the last 6 weeks or who have concerns for poor postsurgical wound healing.\n   * Patients who have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to tocilizumab and its excipients are not eligible.",{"count":213,"type":23},30,[96],"ACTEMRA (tocilizumab) is an IL-6 receptor antagonist used for the treatment of adult Rheumatoid Arthritis as well as Polyarticular (PJIA) and Systemic (SJIA) Juvenile Idiopathic Arthritis. In this Phase II, the drug will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and\u002For radiation therapy.",[217,218],"Adamantinomatous Craniopharyngioma","Recurrent Adamantinomatous Craniopharyngioma","2026-04-20",{"date":221,"type":45},"2026-04-23",{"date":223,"type":45},"2022-12-16",{"date":225,"type":23},"2029-12",{"name":51,"class":52},14,{"id":229,"slug":230,"hasResults":12,"nctId":231,"briefTitle":232,"officialTitle":233,"acronym":4,"eligibilityCriteria":234,"healthyVolunteers":12,"sex":18,"minAge":235,"maxAge":92,"enrollmentInfo":236,"targetDuration":4,"studyType":24,"phases":238,"briefSummary":240,"conditions":241,"keywords":253,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":257,"lastUpdatePostDateStruct":258,"startDateStruct":260,"completionDateStruct":262,"leadSponsor":264,"locationsCount":265},"100460636","phase-1-lutathera-for-treatment-of-recurrent-or-progressive-high-grade-cns-tumors-100460636","NCT05278208","Lutathera for Treatment of Recurrent or Progressive High-Grade CNS Tumors","Phase I\u002FII Study of Lutathera in Patients With Recurrent and\u002For Progressive High-Grade Central Nervous System Tumors and Meningiomas That Demonstrate Uptake on DOTATATE PET","All participants must meet the following inclusion and exclusion criteria. No exceptions will be given. Imaging studies to establish eligibility must be done within three weeks prior to enrollment. All other clinical evaluations to establish eligibility (except for \\[68Ga\\]Ga-DOTATATE PET) must be done within 7 days prior to enrollment.\n\n1. Screening Criteria\n\n   1.1 Diagnosis Patient must have a diagnosis of primary high-grade CNS tumor (any histopathologic diagnosis that is WHO grade III-IV) or meningioma (any histologic grade) that is recurrent, progressive, or refractory. Note that patients with DIPG (based on radiographic\u002Fclinical diagnosis) who have undergone biopsy will be eligible with histologic diagnosis of grade II-IV infiltrating glioma. All tumors must have histologic verification either at the time of diagnosis or recurrence, except for patients meningioma who have not previously undergone biopsy or resection.\n\n   Note: Refractory disease is defined as the presence of persistent abnormality on conventional MRI that is further distinguished by histology (biopsy or sample of lesion) or advanced imaging, OR as determined by the treating physician and discussed with the primary investigator prior to enrollment.\n\n   1.2 Prior Therapy Patients must have recurred\u002Fprogressed following prior standard therapy for their tumor. Note: Patients with meningioma, atypical meningioma, or anaplastic meningioma must have received at least surgical resection or radiation.\n\n   1.3 Screening Consent Participant\u002Flegal guardian is willing to sign a screening consent for \\[68Ga\\]Ga-DOTATATE PET imaging. The screening consent is to be obtained according to institutional guidelines. Assent, when appropriate, will be obtained according to institutional guidelines.\n2. Eligibility Criteria\n\n   * Phase I Age Patient must be ≥ 4 and \\\u003C12 years of age at the time of enrollment. Disease Status: Patients who participate in the efficacy expansion cohort must have bi-dimensionally measurable disease, defined as at least one lesion that can be accurately measured in at least two dimensions Patients with measurable extraneural disease only are also eligible.\n   * Phase II Age Patient must be 12 to \\\u003C\u002F=39 years at the time of enrollment.\n3. Inclusion Criteria\n\n   3.1 Uptake on \\[68Ga\\]Ga-DOTATATE PET Patients must have uptake on DOTATATE PET\u002FCT in at least one tumor lesion (corresponding to known disease) equivalent to a Krenning score ≥2 (confirmed by central radiology review).\n\n   3.2 Prior Therapy Patients must have recovered from the acute treatment related toxicities (defined as ≤ grade 1 if not defined in eligibility criteria) of all prior chemotherapy, immunotherapy, radiotherapy, or any other treatment modality prior to entering this study.\n\n   3.3 Chemotherapy Patients must have received their last dose of known myelosuppressive anticancer therapy at least 21 days prior to enrollment or at least 42 days if nitrosourea.\n\n   3.4 Investigational\u002FBiologic Agent\n\n   ●Biologic or investigational agent (anti-neoplastic): Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the investigational or biologic agent ≥ 7 days prior to study enrollment.\n\n   For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur.\n\n   ●Monoclonal Antibodies and agents with known prolonged half-lives: Patient must have recovered from any acute toxicity potentially related to the agent and received their last dose of the agent ≥ 28 days prior to study enrollment.\n\n   3.5 Radiation\n\n   Patients must have had their last fraction of:\n   * Craniospinal irradiation or total body irradiation or radiation to \\> 50% of pelvis \\> 3 months prior to enrollment.\n   * Focal irradiation \\> 4 weeks prior to enrollment\n\n   3.6 Stem Cell Transplant\n\n   Patient must be:\n   * ≥ 6 months since allogeneic stem cell transplant prior to enrollment with no evidence of active graft vs. host disease\n   * ≥ 3 months since autologous stem cell transplant prior to enrollment\n\n   3.7 Growth Factors Patients must be off all colony-forming growth factor(s) for at least 1 week prior to enrollment (e.g. filgrastim, sargramostim or erythropoietin). Two weeks must have elapsed if patients received long-acting formulations.\n\n   3.8 Somatostatin analogs Patients must be off long-acting somatostatin analogs for at least 4 weeks and off short-acting somastatin analogs (i.e., octreotide) for at least 24 hours.\n\n   3.9 Neurologic Status\n   * Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment, documented by a detailed neurological exam.\n   * Patients with seizure disorders may be enrolled if seizures are well controlled.\n\n   3.10 Performance Status Karnofsky Performance Scale (KPS for \\> 16 years of age) or Lansky Performance Score (LPS for ≤ 16 years of age) assessed within two weeks of enrollment must be ≥ 50. Patients who are unable to walk because of neurologic deficits, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score\n\n   3.11 Organ Function\n\n   Patients must have adequate organ and marrow function, both for eligibility for enrollment, and to begin each subsequent cycle of Lutathera, as defined below:\n   * Adequate Bone Marrow Function as defined as:\n\n     * Absolute neutrophil count ≥ 1.0 x 109 cells\u002F L\n     * Platelets ≥100 x 109 cells\u002F L (unsupported, defined as no platelet transfusion within 7 days)\n     * Hemoglobin ≥8 g\u002Fdl (may receive transfusions)\n   * Adequate Renal Function as defined as:\n\n     * Creatinine clearance or radioisotope GFR \\>70mL\u002Fmin\u002F1.73m2 OR\n     * A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age\u002Fgender as follows:\n\n       1 to \\\u003C 2 years: maximum serum creatinine 0.6 mg\u002FdL for males and females. 2 to \\\u003C 6 years: maximum serum creatinine 0.8 mg\u002FdL for males and females. 6 to \\\u003C 10 years: maximum serum creatinine 1.0 mg\u002FdL for males and females. 10 to \\\u003C 13 years: maximum serum creatinine 1.2 mg\u002FdL for males and females. 13 to \\\u003C 16 years: maximum serum creatinine 1.5 mg\u002FdL for males and 1.4 mg\u002FdL for females.\n\n       ≥ 16 years: maximum serum creatinine 1.7 mg\u002FdL for males and 1.4 mg\u002FdL for females.\n   * Adequate Liver Function as defined as:\n\n     * Total bilirubin ≤ 3 times institutional upper limit of normal (ULN) for age\n     * AST(SGOT)\u002FALT(SGPT) ≤ 3 times institutional ULN\n     * Serum albumin ≥ 2g\u002FdL\n     * Coagulation parameters: INR \\\u003C1.5 times ULN and aPTT \\\u003C1.5 times ULN unless patients are receiving therapeutic anticoagulation which affects these parameters\n   * Adequate Cardiac Function as defined as:\n\n     * Ejection fraction of ≥ 55% by echocardiogram\n     * Serum electrolytes (Sodium, Potassium, Chloride) within institutional limits of normal (patients can be on enteral supplementation)\n\n   3.12 Corticosteroids Patients who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment, with a maximum dexamethasone dose of 2.5mg\u002Fm2\u002Fday.\n\n   3.13 Pregnancy Status Female patients of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to receiving the first dose of study medication. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required.\n\n   3.14 Pregnancy Prevention Patients of childbearing or child fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study and for at least 7 months after drug cessation in females of childbearing potential and for at least 4 months after drug cessation in males of child fathering potential.\n\n   3.15 Informed Consent The patient or parent\u002Fguardian is able to understand the consent and is willing to sign a written informed consent document according to institutional guidelines.\n4. Exclusion Criteria\n\n   4.1 Confirmed bone marrow metastatic disease Patients with confirmed metastatic disease to bone marrow are ineligible.\n\n   4.2 Presence of bulky disease Patients with bulky disease on imaging as described below are ineligible. Treating physicians are encouraged to request a rapid central imaging review to confirm fulfillment of these criteria if there are questions or concerns.\n\n   Bulky disease is defined as:\n   * Tumor with evidence of clinically significant uncal herniation or midline shift.\n   * Tumor with diameter of \\>5cm in one dimension on T2\u002FFLAIR.\n   * Tumor that in the opinion of the site investigator shows significant mass effect in either the brain or spine.\n\n   Note that patients with metastatic or multi-focal disease (with exception of bone marrow) are eligible as long as no sites of disease meet above criteria for bulky disease.\n\n   4.3 Breast-feeding Nursing mothers are excluded from this study. There is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Lutathera.\n\n   4.4 Concurrent Illness\n   * Patients with a history of any other malignancy, except patients with a secondary brain tumor if the patient's prior malignancy has been in remission for at least 5 years from the end of treatment.\n   * Patients with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that in the opinion of the investigator would compromise the patient's ability to tolerate protocol therapy, put them at additional risk for toxicity or would interfere with the study procedures or results.\n   * Patients with type I diabetes.\n\n   4.5 Concomitant Medications\n   * Patients who are receiving any other anti-cancer or investigational drug therapy are ineligible.\n   * Prior or current treatment with 177Lu-DOTATATE\u002FTOC or 90Y-DOTATATE\u002FTOC.\n\n   4.6 Prisoners will be excluded from this study.\n\n   4.7 Inability to participate: Patients who in the opinion of the investigator are unwilling or unable to return for required follow-up visits, obtain follow-up studies required to assess toxicity to therapy, or adhere to drug administration plan, other study procedures, and study restrictions.\n5. Inclusion of Women and Minorities Both males and females of all races and ethnic groups are eligible for this study.","4 Years",{"count":237,"type":23},65,[239,96],"PHASE1","This study will evaluate the safety and efficacy of Lutathera (177Lu-DOTATATE) in patients with progressive or recurrent High-Grade Central Nervous System (CNS) tumors and meningiomas that demonstrate uptake on DOTATATE PET. The drug will be given intravenously once every 8 weeks for a total of up to 4 doses over 8 months in patients aged 4 to \\\u003C12 years (Phase I) or 12 to \\\u003C\u002F=39 years (Phase II) to test its safety and efficacy, respectively.\n\nFunding Source - FDA OOPD (grant number FD-R-0532-01)",[29,242,243,244,245,246,247,193,248,249,250,251,252],"Meningioma","Embryonal Tumor","Medulloblastoma","Anaplastic Ependymoma","Recurrent Diffuse Intrinsic Pontine Glioma","Recurrent Malignant Glioma","Recurrent Primary Central Nervous System Neoplasm","Refractory Diffuse Intrinsic Pontine Glioma","Refractory Malignant Glioma","Refractory Medulloblastoma","Refractory Primary Central Nervous System Neoplasm",[254,255,256],"Somatostatin Receptor","DOTATATE","Lutathera","2026-04-08",{"date":259,"type":45},"2026-04-13",{"date":261,"type":45},"2022-11-21",{"date":263,"type":23},"2033-11",{"name":51,"class":52},4,{"id":267,"slug":268,"hasResults":12,"nctId":269,"briefTitle":270,"officialTitle":270,"acronym":4,"eligibilityCriteria":271,"healthyVolunteers":12,"sex":18,"minAge":272,"maxAge":273,"enrollmentInfo":274,"targetDuration":4,"studyType":24,"phases":275,"briefSummary":276,"conditions":277,"keywords":279,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":284,"lastUpdatePostDateStruct":285,"startDateStruct":286,"completionDateStruct":288,"leadSponsor":290,"locationsCount":83},"100595701","pediatric-cochlear-implant-remote-programming-and-assessment-100595701","NCT07035873","Pediatric Cochlear Implant Remote Programming and Assessment","Inclusion Criteria:\n\n* Cochlear implantation with an Advanced Bionics cochlear implant in at least one ear.\n* At least 6 months post-implantation listening experience and at least 2 weeks of listening experience with Marvel cochlear implant external processor.\n* Speech perception performance of at least 40% correct on standard of care speech perception measures in quiet for the ear to be used in the study\n* Ages 13 years to 20 years, inclusive\n* Ability and willingness to participate in remote programming sessions and self-administered assessment measures\n\nExclusion Criteria:\n\n* Children under the age of 13\n* Speech perception performance \\\u003C 40% on SOC speech perception measures\n* Cognitive or language deficits that preclude ability and willingness to participate in remote programming sessions and self-administered assessment measures, as identified by eMR diagnoses and the participant's primary clinical audiologist judgement.","13 Years","20 Years",{"count":22,"type":23},[67],"This is a prospective, interventional study to evaluate efficacy and end user satisfaction of remote cochlear implant programming in the pediatric population. Additionally, this study will evaluate the long-term replicability of digital audio streaming (DAS) self-assessment speech perception measures via iOS mobile application.",[278],"Cochlear Implant Listeners",[280,281,282,283],"pediatric","cochlear implant","remote programming","telehealth","2026-04-07",{"date":259,"type":45},{"date":287,"type":45},"2025-01-15",{"date":289,"type":23},"2027-03-31",{"name":51,"class":52},{"id":292,"slug":293,"hasResults":12,"nctId":294,"briefTitle":295,"officialTitle":296,"acronym":4,"eligibilityCriteria":297,"healthyVolunteers":12,"sex":18,"minAge":4,"maxAge":298,"enrollmentInfo":299,"targetDuration":4,"studyType":121,"phases":4,"briefSummary":301,"conditions":302,"keywords":304,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":310,"lastUpdatePostDateStruct":311,"startDateStruct":312,"completionDateStruct":314,"leadSponsor":316,"locationsCount":227},"100554767","observation-study-in-patients-age-0-5-years-with-lama2-related-congenital-muscular-dystrophy-100554767","NCT06503367","Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy","Establishing Clinical Trial Readiness for Children 0-5 Years With Congenital Muscular Dystrophy Secondary to LAMA2 Mutations (READY CMD LAMA2)","Inclusion Criteria:\n\n* Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and\u002For assent by the subject (when applicable).\n* Subject must be aged birth to less than 5.0 years of age at time of consent.\n* A confirmed diagnosis of LAMA2-RD confirmed via:\n\n  a: Two pathogenic variants in the LAMA2 gene (via a CLIA-approved laboratory) or: b. muscle biopsy with absence of merosin (laminin-211) and at least one pathogenic variant in the LAMA2 gene\n* Absence of another confirmed genetic disease.\n* Willingness to maintain current exercise and\u002For physical therapy regimen for the duration of the clinical study.\n* Willingness to comply with the study protocol, including but not limited to, all study procedures and visits.\n\nExclusion Criteria:\n\n* Acute medical illness or hospitalization within 30 days prior to informed consent.\n* Participation in a previous trial of any investigational agent for LAMA2-RD within 1 month prior to informed consent, or use of any other investigational therapy (including off-label use of Losartan) within 30 days prior to informed consent, or participation in other clinical studies, within 30 days (or 3 half-lives, whichever is longer) prior to informed consent, which in the opinion of the PI, may potentially confound results from this study.\n* Other significant medical condition, which in the opinion of the site Principal Investigator may confound interpretation of the clinical course of LAMA2- RD.","5 Years",{"count":300,"type":23},44,"The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems.\n\nParticipants will undergo:\n\n* Neuromuscular assessments\n* Blood collections\n* Swallowing and breathing assessments\n* Questionnaires",[303],"LAMA2-MD \\(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\\)",[305,306,307,308,309],"Early Phase 1","Observational","Natural History","Neuromuscular","Functional Assessments","2026-04-06",{"date":257,"type":45},{"date":313,"type":45},"2025-05-12",{"date":315,"type":23},"2028-09",{"name":51,"class":52},{"id":318,"slug":319,"hasResults":12,"nctId":320,"briefTitle":321,"officialTitle":322,"acronym":4,"eligibilityCriteria":323,"healthyVolunteers":12,"sex":18,"minAge":118,"maxAge":92,"enrollmentInfo":324,"targetDuration":4,"studyType":24,"phases":326,"briefSummary":327,"conditions":328,"keywords":331,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":334,"lastUpdatePostDateStruct":335,"startDateStruct":336,"completionDateStruct":338,"leadSponsor":340,"locationsCount":4},"100608845","phase-2-study-of-tovorafenib-in-high-grade-glioma-and-diffuse-intrinsic-pontine-glioma-dipg-100608845","NCT07206849","Study of Tovorafenib in High-Grade Glioma and Diffuse Intrinsic Pontine Glioma (DIPG)","A Phase 2 Study of Tovorafenib in Pediatric and Young Adult Patients Newly Diagnosed With High-Grade Glioma (HGG), Including Diffuse Intrinsic Pontine Glioma (DIPG), Which Harbor Alterations in the Mitogen-Activated Protein Kinase (MAPK) Pathway","Inclusion Criteria:\n\n* Patient must have previously enrolled on TarGeT-SCR.\n\n  1. Age Patients must be ≥12 months and ≤39 years of age at the time of enrollment on TarGeT-SCR.\n  2. Body Surface Area (BSA) Patients must have a BSA \\>0.3m2.\n  3. Diagnosis:\n\n     * Patients with a newly-diagnosed HGG, including DIPG, which harbor alterations in the MAPK pathway are eligible. All patients must have tumor tissue from diagnostic biopsy or resection. The diagnosis of HGG, including DIPG, must have been confirmed through TarGeT-SCR.\n     * For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2\u002F3 of the pons, with histopathology consistent with diffuse WHO Grade 2-4 glioma.\n     * All other HGGs must be WHO Grade 3 or 4.\n  4. Disease Status\n\n     • Patients must be newly diagnosed and enroll and start treatment within 35 days of completion of radiotherapy.\n\n     • Measurable disease is not required. Patients without measurable disease are eligible.\n     * Patients with primary spinal tumors are eligible.\n     * Patients with secondary or radiation-induced HGG are eligible.\n  5. TarGeT-B Strata Definitions\n\n     Patients must be able to be assigned to one of the strata below:\n\n     • Stratum A: Patients with intracranial, localized, non-pontine, and non-thalamic HGG harboring a BRAFV600 mutation (who do not meet criteria for Strata B or C).\n     * Stratum B: Patients with DIPG\u002FDMG as defined in Section 4.1.3 OR patients with localized, non-pontine, non-thalamic HGG harboring a MAPK alteration not included in Stratum A (KIAA1549:BRAF fusion, KRAS\u002FNRAS, CRAF\u002FRAF1, other RAF mutation, or FGFR alteration) OR patients with primary spinal tumors.\n     * Stratum C: Patients with metastatic HGG (including metastatic DIPG\u002FDMG) harboring a MAPK alteration (BRAFV600, KIAA1549:BRAF fusion, KRAS\u002FNRAS, CRAF\u002FRAF1, other RAF mutation, or FGFR alteration).\n  6. Presence of at least one relevant actionable somatic alteration:\n\n     • MAPK pathway alteration(s): BRAFV600 mutation (Strata A or C)\n     * KIAA1549:BRAF fusion (Strata B or C)\n     * KRAS\u002FNRAS alteration (Strata B or C)\n     * CRAF\u002FRAF1 alteration (Strata B or C)\n     * other RAF mutations (Strata B or C)\n     * FGFR alteration (Strata B or C)\n  7. Performance Level:\n\n     Karnofsky ≥ 50 for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (Appendix I). Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n  8. Prior Therapy for HGG:\n\n     o Surgery, radiation (RT), and\u002For dexamethasone are permissible. Temozolomide administered concurrently with RT is permissible but discouraged for patients with DIPG\u002FDMG. No other prior anticancer therapy for HGG will be allowed.\n\n     o Radiation therapy requirements: Patients must have received photon or proton focal radiotherapy if enrolling on Stratum A or B. Patients must have received craniospinal irradiation if enrolling on Stratum C.\n\n     o Radiotherapy, delivered photon or proton beam, must have been administered at a standard dose, including:\n\n     o 54 Gy in 30 fractions for DIPG\n\n     o 54-59.4 Gy in 30-33 fractions for other HGG\n\n     o 45-54 Gy for primary spinal cord HGG\n\n     o And\u002For 36-39.6 Gy craniospinal for patients with spinal or leptomeningeal metastatic disease with supplemental boost to 45-54 Gy for metastasis within the thecal sac and 54-60 Gy for intracranial metastasis.\n\n     o Any variances in the radiotherapy dose within 10% of standard doses outlined above will be discussed with the Sponsor-Investigator to confirm eligibility prior to study enrollment.\n\n     o Timing between diagnosis and start of RT: Patients must have started RT within 31 calendar days of initial diagnosis which is defined as the date of diagnostic biopsy or resection. If a patient underwent two upfront surgeries e.g., biopsy then resection or debulking, this is the date of the second surgery.\n     * Timing post-RT: Patients must enroll and start treatment on TarGeT-B no later than 35 calendar days post-completion of RT. The earliest patients can begin protocol treatment is 28 calendar days post-completion of RT.\n  9. Organ Function Requirements\n* Adequate Bone Marrow Function Defined as:\n\n  • Peripheral absolute neutrophil count (ANC) \\>= 1000\u002Fmm3.\n\n  • Platelet count \\>= 100,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).\n\n  • Hemoglobin \\>8 g\u002FdL (may be transfused).\n* Adequate Renal Function Defined as:\n\nCreatinine clearance or radioisotope GFR \\> 70ml\u002Fmin\u002F1.73 m2 OR serum creatinine based on age\u002Fgender as follows:\n\nMaximum Serum Creatinine (mg\u002FdL) Age Male Female 1 to \\\u003C 2 years 0.6 0.6 2 to \\\u003C 6 years 0.8 0.8 6 to \\\u003C 10 years 1 1 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.4\n\n* 16 years 1.7 1.4 The threshold creatinine values in this table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC.\n\n  * Adequate Liver Function Defined as:\n\n    * Total bilirubin ≤ 1.5 times institutional upper limit of normal (ULN).\n    * Aspartate aminotransferase (AST)\u002Falanine aminotransferase (ALT) \\\u003C 3 times the ULN.\n    * Serum albumin ≥ 2g\u002FdL.\n  * Adequate Cardiac Function Defined as:\n\n    • Left Ventricular Ejection fraction of ≥ 50% as measured by echocardiogram or multiple-gated acquisition (MUGA).\n    * QTc ≤ 450 msec (by Bazett formula).\n  * Adequate Neurologic Function Defined as:\n\n    * Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled (see Appendix II).\n  * Normal Thyroid Function Defined as:\n\n    • Free throxine (T4) within institutional guidelines for normal range. It is acceptable for patient to be on thyroid supplementation as long as free T4 is within institutional guidelines for normal range prior to starting treatment.\n\n    10\\) Informed Consent All patients and\u002For their parents or legally authorized representatives must sign a written Informed Consent and Assent, when appropriate, will be obtained according to institutional guidelines.\n\nExclusion Criteria:\n\n1. Pregnancy or breastfeeding.\n2. Other Exclusion Criteria\n\n   • Patients with neurofibromatosis type 1 (NF-1) are not eligible for this study.\n\n   • Infection: Patients who have an uncontrolled infection are not eligible.\n\n   • Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study are not eligible.\n\n   • Patients with uncontrolled GI disease or other condition that could affect absorption or predispose participant to gastrointestinal ulceration are not eligible.\n3. Concomitant Medications • Corticosteroids: Patients receiving corticosteroids are eligible, but the use of corticosteroids must be reported.\n\n   • Investigational Agents\u002FDrugs: Patients who are currently receiving another investigational drug are not eligible. This includes targeted agents, monoclonal antibodies, herbal supplements, or other investigational agents other than tovorafenib.\n\n   • Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible with the exception of temozolomide given concurrently with radiotherapy.\n\n   • Anticonvulsants: Patients who are receiving enzyme-inducing anticonvulsants as listed in Appendix II, are not eligible.\n\n   • Patients who are receiving medications known to prolong QTc interval as listed in Appendix III are not eligible.\n\n   • As tovorafenib is a substrate of CYP2C8, patients should not take strong inhibitors or inducers of CYP2C8 (See Appendix VI), as they could alter the drug's pharmacokinetics. Medications that are substrates of CYP2C8 or CYP3A4 are allowed but should be used with caution.\n\n   • Medications that are substrates of breast cancer resistance protein (BCRP) with a narrow therapeutic index are prohibited during this study (Appendix IV).\n   * Patients who are receiving duloxetine, alosetron, or theophylline (CYP1A2 inhibitors) are not eligible.\n   * Patients on beta-blockers are not eligible.\n   * Selective serotonin reuptake inhibitors (SSRIs) such as citalopram (Celexa), escitalopram (Lexapro), Fluoxetine (Prozac), fluvoxamine (Luvox), paroxetine (Paxil), sertraline (Zoloft) should be used with caution but are not contraindicated.\n   * Anticoagulants: patients who are receiving therapeutic anticoagulants including warfarin, low-molecular weight heparin are not eligible.\n\n5\\) Patients with prior or ongoing clinically significant medical or psychiatric condition that, in the investigator's opinion, could affect the safety of the participant, or could impair the assessment of study results are not eligible.",{"count":325,"type":23},79,[96],"The goal of this study is to determine the efficacy of the study drugs tovorafenib to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (MAPK) that this drug targets.\n\nThe main question the study aims to answer is whether tovorafenib can prolong the life of patients diagnosed with HGG, including DIPG.",[329,330,30,29,99,100,125],"High Grade Glioma (HGG) of the Brain With BRAF Aberration","High Grade Glioma (III or IV)",[332,333],"tovorafenib","high grade glioma","2026-04-03",{"date":257,"type":45},{"date":337,"type":23},"2026-05",{"date":339,"type":23},"2037-05",{"name":51,"class":52},{"id":342,"slug":343,"hasResults":12,"nctId":344,"briefTitle":345,"officialTitle":346,"acronym":4,"eligibilityCriteria":347,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":92,"enrollmentInfo":348,"targetDuration":4,"studyType":24,"phases":350,"briefSummary":351,"conditions":352,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":334,"lastUpdatePostDateStruct":354,"startDateStruct":355,"completionDateStruct":357,"leadSponsor":359,"locationsCount":360},"100461296","phase-2-mektovi-for-the-treatment-of-pediatric-adamantinomatous-craniopharyngioma-100461296","NCT05286788","MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma","Phase 2 Study of the MEK Inhibitor MEKTOVI® (Binimetinib) for the Treatment of Pediatric Adamantinomatous Craniopharyngioma","Inclusion Criteria:\n\n1. Age: Patients must be ≥ 12 months and ≤ 39 years of age at the time of study enrollment.\n2. Diagnosis: Patients with histologically-confirmed adamantinomatous craniopharyngioma (ACP) Histologic confirmation of ACP may be made on solid tumor or, if no solid tumor can be safely obtained, cyst fluid with classic ACP characteristics of thick, cholesterol-rich, greenish-brown liquid in the context of imaging features consistent with craniopharyngioma, including lobulated, cystic\u002Fsolid mass with calcifications that originates in the sellar\u002Fsuprasellar region.\n3. Disease Status: Patients must have measurable disease.\n\n   * Stratum 1: Patients with progressive or recurrent ACP who demonstrate cystic and\u002For solid recurrence or progression at least 6 months post completion of radiation therapy\n   * Stratum 2 (NOT CURRENTLY ENROLLING): Patients with measurable ACP who have undergone surgery but have NOT previously undergone irradiation (but may have received prior systemic or intracystic therapy). Progressive disease is allowed but not required.\n4. Performance Level: Karnofsky ≥ 50% for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Note: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n5. Prior Therapy: Patients must have recovered or stabilized from the acute toxic effects of prior treatments\n\n   * Biologic (anti-neoplastic agent): At least 7 days must have elapsed after the last (systemic or intracystic) dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair\n   * Immunotherapy: At least 42 days after the completion of any type of systemic immunotherapy, e.g. tumor vaccines.\n   * Monoclonal antibodies: At least 21 days after the last dose of a monoclonal antibody.\n   * Radiation therapy: Patients must have had their last (conventional or hypofractionated) fraction of: a) Focal irradiation \\> 6 months prior to enrollment and b) No prior craniospinal irradiation is permitted.\n   * Corticosteroids: Patients receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to enrollment\n   * Myelosuppressive systemic therapy: At least 21 days must have elapsed after the last systemic myelosuppressive therapy.\n   * Surgery: At least 6 weeks must have elapsed since major or intermediate surgery. Major surgery includes major craniotomy for tumor resection of cyst fenestration, organ resection, and exploratory laparotomy. Intermediate procedures include ventriculoperitoneal shunt placement, stereotactic brain biopsy, and intraventricular catheter placement. Minor procedures that are not excluded include skin biopsy\u002Fincision and drainage, bone marrow aspirate, and central venous catheter placement, ommaya aspirations, lumbar punctures, and nasal endoscopy to remove packing.\n6. Organ Function Requirements\n\n   Adequate Bone Marrow Function Defined as:\n   * Peripheral absolute neutrophil count (ANC) ≥1000\u002Fmm3\n   * Platelet count ≥100,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)\n   * Hemoglobin \\>8 g\u002FdL (may be transfused)\n\n   Adequate Renal Function Defined as:\n   * Creatinine clearance or radioisotope GFR \\> 70ml\u002Fmin\u002F1.73 m2 or\n   * A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age\u002Fgender as follows:\n\n     1. to \\\u003C 2 years: maximum serum creatinine 0.6 mg\u002FdL for males and females. 2 to \\\u003C 6 years: maximum serum creatinine 0.8 mg\u002FdL for males and females. 6 to \\\u003C 10 years: maximum serum creatinine 1.0 mg\u002FdL for males and females. 10 to \\\u003C 13 years: maximum serum creatinine 1.2 mg\u002FdL for males and females. 13 to \\\u003C 16 years: maximum serum creatinine 1.5 mg\u002FdL for males and 1.4 mg\u002FdL for females. ≥ 16 years: maximum serum creatinine 1.7 mg\u002FdL for males and 1.4 mg\u002FdL for females.\n\n   Adequate Liver Function Defined as:\n   * Total bilirubin ≤ 1.5 × institutional upper limit of normal\n   * AST (SGOT) ≤ 2.5 × institutional upper limit of normal\n   * ALT (SGPT) ≤ 2.5 × institutional upper limit of normal\n\n   Adequate Cardiac Function Defined as:\n   * Left Ventricular Ejection Fraction greater than the institutional lower limit of normal by echocardiogram\n   * QTc ≤ 480 msec (by Bazett formula)\n\n   Adequate Neurologic Function Defined as:\n   * Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.\n   * Patients with current seizure disorders may be enrolled if seizures are well-controlled on antiepileptic therapies.\n7. Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines\n\nExclusion Criteria:\n\n1. Pregnancy or Breast-Feeding: Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for at least 90 days after discontinuation of drug for females and at least 60 days for males. For females of childbearing potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods (bilateral tubal ligation, male sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices; hormonal contraceptive methods must be supplemented by a barrier method) and agreement to refrain from donating eggs are required. For males of reproductive potential, agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm.\n2. Gastrointestinal Disease:\n\n   * Patients with a history of serious gastrointestinal disease, including inflammatory bowel disease or gastrointestinal perforation\n   * Patients who are unable to absorb enteral medications\n   * Administration via NG\u002FNJ\u002FG-tube is allowed\n3. Concomitant Medications\n\n   * Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.\n   * Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible.\n   * Anti-cancer Agents: Patients who are currently receiving other anti-cancer agents are not eligible.\n4. Study Specific:\n\n   * Patients who have an uncontrolled infection are not eligible.\n   * Patients who have received any live or attenuated vaccinations within three months prior to start of therapy are not eligible.\n   * Any significant concurrent medical or surgical condition that would jeopardize the patient's safety or ability to complete the study, including, but not limited to, disease of the nervous, renal, hepatic, cardiac (such as symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia), pulmonary, or endocrine system\n   * Patients who have a history of Human Immunodeficiency Virus, Hepatitis B Virus, Hepatitis C Virus or Tuberculosis infection are not eligible.\n   * Patients who have received a prior solid organ transplantation are not eligible.\n   * Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.\n   * Patients who have a history of alcohol, drug, or chemical abuse within 6 months of screening.\n   * Patients who have had surgery within the last 6 weeks or who have concerns for poor postsurgical wound healing.\n   * Patients who have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to tocilizumab and its excipients are not eligible.",{"count":349,"type":23},38,[96],"MEKTOVI (binimetinib) is an oral, highly selective reversible inhibitor of mitogen-activated extracellular signal regulated kinase 1 (MEK1) and MEK2. The biological activity of binimetinib that has been evaluated bith in vitro and in vivo in a wide variety of tumor types In this Phase II, the drug will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and\u002For radiation therapy.",[353,218],"Adamantinous Craniopharyngioma",{"date":257,"type":45},{"date":356,"type":45},"2023-04-10",{"date":358,"type":23},"2027-04-10",{"name":51,"class":52},10,{"id":362,"slug":363,"hasResults":12,"nctId":364,"briefTitle":365,"officialTitle":366,"acronym":4,"eligibilityCriteria":367,"healthyVolunteers":17,"sex":18,"minAge":4,"maxAge":368,"enrollmentInfo":369,"targetDuration":4,"studyType":121,"phases":4,"briefSummary":370,"conditions":371,"keywords":373,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":376,"lastUpdatePostDateStruct":377,"startDateStruct":378,"completionDateStruct":380,"leadSponsor":382,"locationsCount":83},"100511448","dosc-and-association-with-neighborhood-opportunity-phase-ii-100511448","NCT05939570","DOSC and Association With Neighborhood Opportunity: Phase II","Pediatric Day-of-Surgery Cancellations and the Association With Neighborhood Opportunity and Indices of Vulnerability : Phase II","Inclusion Criteria:\n\n* Parents\u002Fguardians of children \\\u003C18 who had a day of surgery cancellation\n* Parents\u002Fguardians speak English, Spanish, Somali, Nepali or Arabic.\n\nExclusion Criteria:\n\n* Parents\u002Fguardians speak a different language other than above listed.","18 Years",{"count":213,"type":23},"Prospective, qualitative interview study to explore factors associated with pediatric day of surgery cancellations (DOSC) from the prospective of parents with hopes the interviews will reveal additional vulnerabilities not included in COI 2.0 that predispose to DOSC.",[372],"Pediatric Day of Surgery Cancellations",[374,375],"Pediatrics","Day of Surgery Cancellations","2026-04-02",{"date":334,"type":45},{"date":379,"type":45},"2023-07-25",{"date":381,"type":23},"2026-12",{"name":51,"class":52},{"id":384,"slug":385,"hasResults":12,"nctId":386,"briefTitle":387,"officialTitle":388,"acronym":4,"eligibilityCriteria":389,"healthyVolunteers":17,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":390,"targetDuration":4,"studyType":121,"phases":4,"briefSummary":392,"conditions":393,"keywords":395,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":409,"lastUpdatePostDateStruct":410,"startDateStruct":411,"completionDateStruct":413,"leadSponsor":415,"locationsCount":83},"100147550","genetics-of-congenital-heart-disease-100147550","NCT01192048","Genetics of Congenital Heart Disease","Genetic Testing of Individuals and Families With Congenital Heart Disease","Inclusion Criteria:\n\n* Subjects must have a diagnosis of Congenital Heart Disease or be related to individuals with Congenital Heart Disease.\n\nExclusion Criteria:\n\n* Healthy individuals unrelated to those with Congenital Heart Disease",{"count":391,"type":23},5000,"Congenital heart disease (CHD) is the most common type of birth defect but the cause for the majority of cardiac birth defects remains unknown. Numerous epidemiologic studies have demonstrated evidence that genetic factors likely play a contributory, if not causative, role in CHD. While numerous genes have been identified by us and other investigators using traditional genetic approaches, these genes account for a minority of the non-syndromic CHDs. Therefore, we are now utilizing whole genome sequencing (WGS), with the addition of more traditional genetic techniques such as chromosomal microarray or traditional linkage analysis, to identify genetic causes of familial and isolated CHD. With WGS we are able to sequence all of the genetic material of an individual and apply different data analysis techniques based on whether we are analyzing a multiplex family or a cohort of trios (mother, father and child with CHD) with a specific isolated CHD. Therefore, WGS is a robust method for identification of novel genetic causes of CHD which will have important diagnostic and therapeutic consequences for these children.",[394],"Congenital Heart Disease",[394,396,397,398,399,400,401,402,403,404,405,406,407,408],"birth defect","genetics","gene","DNA","direct sequencing","microarray","single nucleotide polymorphism","whole genome array comparative genomic hybridization","chromosomal copy number change","nucleotide sequence variation","exome sequencing","whole exome sequencing","whole genome sequencing","2026-04-01",{"date":284,"type":45},{"date":412,"type":4},"2009-12",{"date":414,"type":23},"2030-12",{"name":51,"class":52},{"id":417,"slug":418,"hasResults":12,"nctId":419,"briefTitle":420,"officialTitle":421,"acronym":422,"eligibilityCriteria":423,"healthyVolunteers":12,"sex":18,"minAge":424,"maxAge":368,"enrollmentInfo":425,"targetDuration":4,"studyType":24,"phases":427,"briefSummary":428,"conditions":429,"keywords":431,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":434,"lastUpdatePostDateStruct":435,"startDateStruct":436,"completionDateStruct":438,"leadSponsor":439,"locationsCount":83},"100531050","improving-status-epilepticus-treatment-times-100531050","NCT06194747","Improving Status Epilepticus Treatment Times","Quality Improvement in Time to Treatment of Status Epilepticus","QuITT-SE","Inclusion Criteria:\n\n* SE episode occurs in a male or female child aged between \\> 30 days to \\\u003C 19 years\n* Seizures meeting AT LEAST ONE of the following criteria:\n\n  1. continuous clinically apparent seizure lasting greater than 5 minutes\n  2. continuous clinically apparent seizure of any duration receiving BZD\n  3. repeated seizures without return to neurological baseline within 5 minutes\n\nExclusion Criteria:\n\n* SE episode occurs in a child with infantile spasms\n* SE episode occurs in a child with electrographic-only seizures without clinical signs other than encephalopathy","30 Days",{"count":426,"type":23},450,[67],"This is a stepped-wedge cluster randomized effectiveness-implementation hybrid study aimed at determining the effect of dissemination of a QI bundle on the time to treatment of SE among hospitalized, non-critically ill children. The primary study endpoint is to decrease the time from the SE diagnosis to treatment with the first dose of a benzodiazepine (BZD) as measured during hospitalization, which will decrease chances of morbidity and mortality.",[430],"Status Epilepticus",[430,432,433],"Quality Improvement","Interventions","2026-03-27",{"date":409,"type":45},{"date":437,"type":45},"2024-02-01",{"date":289,"type":23},{"name":51,"class":52},{"id":441,"slug":442,"hasResults":12,"nctId":443,"briefTitle":444,"officialTitle":445,"acronym":4,"eligibilityCriteria":446,"healthyVolunteers":12,"sex":18,"minAge":4,"maxAge":20,"enrollmentInfo":447,"targetDuration":4,"studyType":24,"phases":448,"briefSummary":449,"conditions":450,"keywords":451,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":456,"lastUpdatePostDateStruct":457,"startDateStruct":459,"completionDateStruct":461,"leadSponsor":463,"locationsCount":133},"100412811","early-phase-1-a-pilot-study-of-larotrectinib-for-newly-diagnosed-high-grade-glioma-with-ntrk-fusion-100412811","NCT04655404","A Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion","A Pilot and Surgical Study of Larotrectinib for Treatment of Children With Newly-Diagnosed High-Grade Glioma With NTRK Fusion","Inclusion Criteria:\n\n* Age: Patients ≤ 21 years of age (birth to 21 years of age) at the time of study enrollment will be eligible.\n* Diagnosis: Patients with newly-diagnosed high-grade (HGG), including diffuse intrinsic pontine gliomas (DIPG), whose tumors are documented in a CLIA\u002FCAP certified lab (or clinically equivalent method considered standard in non-US sites) to harbor an NTRK fusion alteration by FISH, PCR, or next generation sequencing are eligible. Patients must have had histologically verified high-grade glioma such as anaplastic astrocytoma, glioblastoma, or H3 K27-mutant diffuse midline glioma verified at a CONNECT site.\n\nFor sites that do not have CLIA-certified equivalent (certified laboratory) to assess NTRK fusion, testing will be conducted centrally at NCH. NTRK testing will be performed by NGS using targeted RNA-sequencing (Archer Solid Tumor analysis) Please submit 10 unstained sections on charged slides at 10uM thickness, or 10 scrolls cut at 10uM thickness, along with submission of an H\\&E slide. Formalin-fixed paraffin embedded (FFPE) tissue block and FFPE tissue scroll specimens must contain minimum of 25% tumor Snap-frozen tissue specimens are also acceptable and they must contain a minimum of 10% tumor. Please note that turn-around time for this test is up to 21 days.\n\n* Disease Status: Patients with disseminated DIPG or HGG are eligible only if the patient is to receive chemotherapy only, i.e. no craniospinal RT is intended to be given. MRI of spine must be performed if disseminated disease is suspected clinically by the treating physicians. Patients with primary spinal tumors are eligible only if the patient is to receive either chemotherapy or focal radiation therapy, i.e. no craniospinal RT is intended to be given. Patients with leptomeningeal disease only, with no definitive identifiable primary tumor, and documented NTRK fusion, must be discussed with the Study Chair on a case-by-case basis.\n* Surgical Cohort ONLY: Patients with newly-diagnosed HGG with NTRK fusions who have undergone prior biopsy and for whom further resection is indicated for a more definitive surgery at an enrolling site will be eligible to enroll onto the surgical study. DIPG patients are not eligible for the surgical cohort.\n* Performance Level: Karnofsky ≥ 50% for patients \\> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n* Prior Therapy: Patients must not have received any prior anti-cancer chemotherapy. Prior use of corticosteroids are allowed (see below Exclusion Criteria)\n* Organ Function Requirements: Adequate Bone Marrow Function Defined as:\n\nPeripheral absolute neutrophil count (ANC) ≥ 1000\u002Fmm3 Platelet count ≥ 100,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) Hemoglobin \\>8 g\u002FdL (may receive transfusions)\n\n\\- Adequate Renal Function Defined as: Serum creatinine within normal institutional limits, or Creatinine clearance or radioisotope GFR ≥ 70ml\u002Fmin\u002F1.73 m2\n\n\\- Adequate Liver Function Defined as: Total bilirubin ≤ 2.5 × institutional upper limit of normal AST(SGOT)\u002FALT(SGPT) ≤ 2.5 × institutional upper limit of normal\n\n\\- Adequate Pulmonary Function Defined as: Pulse oximetry \\> 94% on room air if there is clinical indication for determination (e.g. dyspnea at rest).\n\n\\- Adequate Neurologic Function Defined as: Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled. See Section 5.5.2 and Appendix III for EIAED guidelines.\n\n\\- Informed Consent: All patients and\u002For their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.\n\nExclusion Criteria:\n\n* Pregnancy or Breast-Feeding: Pregnant or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.\n* Concomitant Medications Investigational Drugs: Patients who have previously received or are currently receiving another investigational drug are not eligible.\n\nAnti-cancer Agents: Patients who have previously received or are currently receiving other anti-cancer agents, including chemotherapy, immunotherapy, monoclonal antibodies, biologic or targeted therapy, are not eligible\n\n* Infection: Patients must not have any active, uncontrolled systemic bacterial, viral or fungal infection.\n* Patients who have received prior solid organ transplantation are not eligible.\n* Patients must not have malabsorption syndrome or other condition affecting oral absorption.\n* Patients must not be receiving any treatment with a strong cytochrome P450 3A4 (CYP3A4) inhibitor or inducer. (See Appendix III.) Strong inducers or inhibitors of CYP3A4 should be avoided from 7 days prior to enrollment to the end of the study.\n* Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.",{"count":22,"type":23},[26],"This is a pilot study that will evaluate disease status in children that have been newly diagnosed high-grade glioma with TRK fusion. The evaluation will occur after 2 cycles of the medication (Larotrectinib) have been given.\n\nThe study will also evaluate the safety of larotrectinib when given with chemotherapy in your children; as well as the safety larotrectinib when given post-focal radiation therapy.",[29,30],[452,453,454,455],"NTRK gene fusion","BABYPOG","HIT-SKK","Larotrectinib","2026-03-16",{"date":458,"type":45},"2026-03-18",{"date":460,"type":45},"2021-04-08",{"date":462,"type":23},"2036-12-01",{"name":51,"class":52},{"id":465,"slug":466,"hasResults":12,"nctId":467,"briefTitle":468,"officialTitle":469,"acronym":470,"eligibilityCriteria":471,"healthyVolunteers":12,"sex":18,"minAge":62,"maxAge":472,"enrollmentInfo":473,"targetDuration":4,"studyType":24,"phases":475,"briefSummary":477,"conditions":478,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":480,"lastUpdatePostDateStruct":481,"startDateStruct":483,"completionDateStruct":485,"leadSponsor":487,"locationsCount":488},"100459838","phase-2-targeted-reversal-of-inflammation-in-pediatric-sepsis-induced-mods-100459838","NCT05267821","Targeted Reversal of Inflammation in Pediatric Sepsis-induced MODS","Targeted Reversal of Inflammation in Pediatric Sepsis-induced MODS (TRIPS)","TRIPS","Inclusion Criteria:\n\n* ≥ 40 weeks corrected gestational age to \\\u003C 18 years; AND\n* Admission to the PICU or CICU; AND\n* Onset of ≥ 2 new organ dysfunctions within the last 3 calendar days (compared to pre-sepsis baseline) as measured by the modified Proulx criteria; AND\n* Documented or suspected infection as the MODS inciting event.\n\nExclusion Criteria:\n\n* Weight \\\u003C3kg; OR\n* Limitation of care order at the time of screening; OR\n* Patients at high likelihood of progression to brain death in opinion of the clinical team; OR\n* Moribund condition in which the patient is unlikely to survive the next 48 hours in opinion of the clinical team; OR\n* Current or prior diagnosis of hemophagocytic lymphohistiocytosis or macrophage activation syndrome; OR\n* Peripheral white blood cell count \\\u003C 1,000 cells\u002Fmm3 as the result of myeloablative therapy OR receipt of myeloablative therapy within the previous 14 days; OR\n* Known allergy to anakinra, or E. coli-derived products; OR\n* Known pregnancy; OR\n* Lactating females; OR\n* Receipt of anakinra within the previous 28 days; OR\n* Resolution of MODS by MODS Day 2; OR\n* Previous enrollment in the TRIPS study.","17 Years",{"count":474,"type":23},500,[96,476],"PHASE3","The TRIPS study is a prospective, multi-center, double-blind, adaptively randomized, placebo-controlled clinical trial of the drug anakinra for reversal of moderate to severe hyperinflammation in children with sepsis-induced multiple organ dysfunction syndrome (MODS).",[479],"Pediatric Sepsis-induced Multiple Organ Dysfunction Syndrome (MODS)","2026-03-11",{"date":482,"type":45},"2026-03-13",{"date":484,"type":45},"2022-06-14",{"date":486,"type":23},"2028-08-31",{"name":51,"class":52},32,{"id":490,"slug":491,"hasResults":12,"nctId":492,"briefTitle":493,"officialTitle":494,"acronym":4,"eligibilityCriteria":495,"healthyVolunteers":12,"sex":18,"minAge":496,"maxAge":4,"enrollmentInfo":497,"targetDuration":4,"studyType":24,"phases":499,"briefSummary":500,"conditions":501,"keywords":4,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":504,"lastUpdatePostDateStruct":505,"startDateStruct":507,"completionDateStruct":509,"leadSponsor":511,"locationsCount":4},"100626450","tetragraph-to-evaluate-onset-and-recovery-times-of-rocuronium-100626450","NCT07435792","TetraGraph to Evaluate Onset and Recovery Times of Rocuronium","Train-of-four Monitoring Using the TetraGraph Electromyographic Neuromuscular Transmission Monitor to Evaluate Onset and Recovery Times of a Single Bolus Dose of Rocuronium","Inclusion Criteria:\n\n* consenting American Society of Anesthesiologists (ASA) Physical Status 1-4 patients\n* undergoing a surgical procedure with general anesthesia and requiring the administration of rocuronium\n\nExclusion Criteria:\n\n* Patients with history of a progressive or degenerative peripheral neurologic or neuropathic disorder\n* Patients undergoing a surgical procedure in which neuromuscular blockade is not required\n* Edematous patients","1 Month",{"count":498,"type":23},130,[67],"In most clinical instances, direct laryngoscopy and endotracheal intubation is performed after a set time (generally 60 seconds) following the administration of a neuromuscular (NM) blocking agent, rocuronium. Although the average onset time is 60 seconds, clinical studies show a standard deviation around the mean onset time of 10-20 seconds, meaning that a significant number of patients do not achieve complete NM blockade by 60 seconds. Given the above noted variability, complete NM blockade may not be achieved in 60 seconds in a significant percentage of patients, resulting in more problematic direct laryngoscopy and endotracheal intubation. Various studies have shown a higher incidence of perioperative respiratory events and postoperative concerns (sore throat, vocal cord injury) when NM blockade is not complete prior to endotracheal intubation.\n\nThis study aims to use the TetraGraph monitor to clearly identify onset times of rocuronium. Additionally, whenever feasible based on the clinical needs of the case, recovery times after the single dose of rocuronium will be recorded.",[502,503],"Surgery","Neuromuscular Blockade","2026-02-25",{"date":506,"type":45},"2026-02-27",{"date":508,"type":23},"2026-06-01",{"date":510,"type":23},"2029-05-31",{"name":51,"class":52},{"id":513,"slug":514,"hasResults":12,"nctId":515,"briefTitle":516,"officialTitle":516,"acronym":517,"eligibilityCriteria":518,"healthyVolunteers":12,"sex":18,"minAge":519,"maxAge":520,"enrollmentInfo":521,"targetDuration":4,"studyType":24,"phases":523,"briefSummary":524,"conditions":525,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":528,"lastUpdatePostDateStruct":529,"startDateStruct":531,"completionDateStruct":533,"leadSponsor":535,"locationsCount":536},"100487996","phase-1-a-multi-institution-study-of-tgf-imprinted-ex-vivo-expanded-universal-donor-nk-cell-infusions-as-adoptive-immunotherapy-in-combination-with-gemcitabine-and-docetaxel-in-patients-with-relapsed-or-refractory-pediatric-bone-and-soft-tissue-100487996","NCT05634369","A Multi-Institution Study of TGFβ Imprinted, Ex Vivo Expanded Universal Donor NK Cell Infusions as Adoptive Immunotherapy in Combination With Gemcitabine and Docetaxel in Patients With Relapsed or Refractory Pediatric Bone and Soft Tissue","TINKS","Inclusion Criteria:\n\n1. Patients must be between the ages ≥ 2 years and ≤ 40 years of age and have had a relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma or non-rhabdomyosarcoma soft tissue sarcoma.\n2. Patients must have measurable disease using RECIST 1.1 criteria\n3. Patients must have had at least one and no more than four total lines of cytotoxic systemic treatment for relapse sarcoma. Local control with surgical resection or radiation therapy of the primary tumor and any metastatic sites as clinically indicated as standard of care per the treating physician must be considered prior to enrollment.\n4. Prior Therapy: Therapy may not have been received more recently than the timeframes defined below:\n\n   * Myelosuppressive chemotherapy: Patients must not have received myelosuppressive therapy within 14 days of protocol therapy\n   * Radiation: At least 2 weeks must have elapsed from the start of protocol therapy since local palliative XRT (small port); 4 weeks must have elapsed for all other radiation therapy\n   * Hematopoietic Cell Transplant (HCT): Patients must have at least 6 weeks elapsed after autologous and allogeneic hematopoietic cell transplant\n   * Biologic (anti-neoplastic agent): At least 7 days or 5 half-lives of the drug, whichever is longer, must have elapsed from the start of protocol therapy since the completion of therapy with a biologic agent.\n   * Monoclonal antibodies: At least 3 weeks must have elapsed from the start of protocol therapy since prior therapy that included a monoclonal antibody.\n   * Prior use of Gemcitabine and\u002For Docetaxel: Patients who have received these agents for prior treatment may be included if previous treatments were given ≥ 6 months prior to enrollment on this study, and there were no allergic reactions, pulmonary edema or fibrosis, Grade 3 or higher neuropathy or other non-hematologic Grade 4 adverse events related to gemcitabine and\u002For docetaxel therapies.\n\n4\\) Performance status: Karnofsky ≥ 60 for patients ≥16 years of age. Lansky score of ≥ 60 for patients \\\u003C 16 years of age (see Appendix A) 5) Organ Function Requirements: Patients must have normal organ and marrow function within 7 days of starting protocol therapy as defined below:\n\n* Absolute Neutrophil Count ≥1000\u002FmcL\n* Platelet count ≥100,000\u002FmcL transfusion independent defined as no platelet transfusions within the last 72 hours\n* Total bilirubin \\\u003C 1.5x upper limit of normal for age\n* AST(SGOT)\u002FALT(SGPT) ≤ 2.5 x institutional upper limit of normal\n* Serum creatinine \\\u003C 1.5 x upper limit of normal based on age\u002Fgender (Table 3) OR creatinine clearance ≥70 mL\u002Fmin\u002F1.73 m2 for patients with creatinine levels above institutional normal\n* Shortening fraction ≥ 27% by ECHO OR ejection fraction of ≥ 50% by ECHO or gated radionuclide study\n\n  * Echocardiogram done within 12 months of study entry will be acceptable. If patient has required anthracycline chemotherapy since last ECHO and enrollment on this study, echocardiogram should be repeated.\n* No evidence for dyspnea at rest, no chronic oxygen requirement, and room air pulse oximetry \\>94% if there is a clinical indication for pulse oximetry 6) Neuropathy: Patients must have ≤ Grade 2 neuropathy at enrollment 7) Patients with seizure disorders may be enrolled if seizures are well controlled on anti-convulsant, with the exception of diazepam given its potential deleterious effects on NK cell activity.\n\n  8\\) Contraception: The effects of expanded NK cells on the developing human fetus are unknown. For this reason and because the chemotherapeutic preparative agents as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of preparatory regimen administration.\n\n  9\\) All patients and\u002For their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent\u002Fassent document.\n\nExclusion Criteria:\n\n1. Patients who are receiving any other investigational agents.\n2. Patients must not be receiving any additional medicines being given for the specific purpose of treating cancer\n3. Patients with a history of allergic reactions attributed to docetaxel, gemcitabine, or peg-filgrastim or biosimilar\n4. Patients who have received any prior cellular therapies, such as CAR-T cells or other expanded or manufactured cellular products.\n5. Patients with bone marrow only disease are not eligible for this study.\n6. Patients with any of the following \"Intermediate\" (rarely metastasizing) or \"malignant\" Grade 2 or Grade 3 tumors of any size, as defined in the WHO Classification of Soft Tissue Tumors are not eligible for this study:\n\n   * So-called fibrohistiocytic tumors - plexiform fibrohistiocytic tumor, giant cell tumor of soft tissues\n   * Fibroblastic\u002Fmyofibroblastic tumors - solitary fibrous tumor, malignant solitary fibrous tumor, inflammatory myofibroblastic tumor, low grade myofibroblastic sarcoma, myxoinflammatory fibroblastic sarcoma, atypical myxoinflammatory fibroblastic tumor, myxofibrosarcoma, low grade fibromyxoid sarcoma, sclerosing epithelioid fibrosarcoma\n   * Tumors of uncertain differentiation - epithelioid sarcoma, alveolar soft part sarcoma, clear cell sarcoma of soft tissue, angiomatoid fibrous histiocytoma, ossifying fibromyxoid tumour, myoepithelioma, myoepithelial carcinoma, extraskeletal myxoid chondrosarcoma, neoplasms with perivascular epithelioid cell differentiation (PEComa), initial sarcoma, atypical fibroxanthoma, mixed tumor NOS, phosphaturic mesenchymal tumor, malignant ossifying fibromyxoid tumor, malignant mixed tumor, malignant phosphaturic mesenchymal tumor\n   * Chondro-osseous tumors - extraskeletal osteosarcoma\n   * Pericytic (perivascular) tumors - malignant glomus tumor\n   * Nerve sheath tumors - malignant peripheral nerve sheath tumor, malignant granular cell tumor, epithelioid malignant peripheral nerve sheath tumor, malignant Triton tumor\n   * Undifferentiated sarcomas (with a specific pathologic category in the WHO classification) - undifferentiated round cell sarcoma, undifferentiated epithelioid sarcoma, undifferentiated spindle cell sarcoma\n7. Patients who, in the judgment of the treating physician, has tumors near critical structures for which transient swelling would cause substantial symptoms, such as tumor within the bowel mucosa\n8. Patients with CNS metastatic disease will not be eligible for this study.\n9. Concomitant Medications:\n\n   * Due to their effect on NK cell function, systemic corticosteroids outside of the supportive dexamethasone given from day 7 through 9 should be used ONLY for life-threatening conditions (i.e., life-threatening allergic reactions and anaphylaxis such as bronchospasm, stridor) unresponsive to other measures. The use of dexamethasone as an anti-emetic is not permitted. Corticosteroid therapy can be used as a premedication for transfusion in patients known to have a history of transfusion reactions or for treatment of an unexpected transfusion reaction (hydrocortisone 2 mg\u002Fkg or less or an equivalent dose of an alternative corticosteroids). The use of steroids during protocol therapy other than the study- required prophylactic dexamethasone doses requires clear justification and documentation of use for a life-threatening condition.\n   * The following are also prohibited while on study treatment\n\n     * Strong CYP3A4 inducers. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated list such as http:\u002F\u002Fmedicine.iupui.edu\u002Fclinpharm\u002Fddis\u002F; medical reference texts such as the Physicians' Desk Reference may also provide this information.\n     * Diazepam\n     * Chemotherapeutic agents other than the study drugs\n10. Uncontrolled intercurrent illness including, but not limited to:\n\n    * ongoing or active infection\n    * psychiatric illness\u002Fsocial situations that would limit compliance with study requirements\n11. Pregnancy or Breast-Feeding: Pregnant or breast-feeding woman will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal\u002Fhuman studies with Gemcitabine and Docetaxel\n12. HIV Infection: HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with the study medications. In addition, these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated\n13. Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.","2 Years","40 Years",{"count":522,"type":23},50,[239,96],"The purpose of this study is to determine if the addition of infusions of a type of immune cell called a \"natural killer\", or NK cell to the sarcoma chemotherapy regimen GEM\u002FDOX (gemcitabine and docetaxel) can improve outcomes in people with childhood sarcomas that have relapsed or not responded to prior therapies.\n\nThe goals of this study are:\n\n* To determine the safety and efficacy of the addition of adoptive transfer of universal donor, TGFβ imprinted (TGFβi), expanded NK cells to the pediatric sarcoma salvage chemotherapeutic regimen gemcitabine\u002Fdocetaxel (GEM\u002FDOX) for treatment of relapsed and refractory pediatric sarcomas To determine the 6-month progression free survival achieved with this treatment in patients within cohorts of relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma and non-rhabdomyosarcoma soft tissue sarcoma.\n* To identify toxicities related to treatment with GEM\u002FDOX + TGFβi expanded NK cells\n\nParticipants will receive study drugs that include chemotherapy and NK cells in cycles; each cycle is 21 days long and you can receive up to 8 cycles.\n\n* Gemcitabine (GEM): via IV on Days 1 and 8\n* Docetaxel (DOX): via IV on Day 8\n* Prophylactic dexamethasone: Day 7-9 to prevent fluid retention and hypersensitivity reaction\n* Peg-filgrastim (PEG-GCSF) or biosimilar: Day 9 to help your white blood cell recover and allow more chemotherapy to be given\n* TGFβi NK cells: via IV on Day 12",[526,527],"Pediatric Sarcoma, Refractory","Pediatric Sarcoma, Relapsed","2026-01-26",{"date":530,"type":45},"2026-01-28",{"date":532,"type":45},"2022-11-14",{"date":534,"type":23},"2027-12-01",{"name":51,"class":52},22,{"id":538,"slug":539,"hasResults":12,"nctId":540,"briefTitle":541,"officialTitle":542,"acronym":4,"eligibilityCriteria":543,"healthyVolunteers":12,"sex":18,"minAge":544,"maxAge":545,"enrollmentInfo":546,"targetDuration":4,"studyType":24,"phases":548,"briefSummary":549,"conditions":550,"keywords":553,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":559,"lastUpdatePostDateStruct":560,"startDateStruct":562,"completionDateStruct":564,"leadSponsor":566,"locationsCount":4},"100595704","novel-pioneer-protocol-rct-100595704","NCT07035912","Novel PIONEER© Protocol: RCT","Randomized Controlled Trial Evaluating the Impact of a Novel Parent Implemented Oral Nutrition, Eating, and Esophageal Reflexes Reintegration Program on Hospitalized Infants With Feeding Challenges","INCLUSION CRITERIA:\n\n1. Nasogastric tube fed infants referred for diagnostic manometry and Gastrostomy tube planning evaluations.\n2. Physiologically stable convalescing infants on full enteral feeds at greater than or equal to 37.0 weeks and less than 46 weeks postmenstrual age.\n3. Presence of aerodigestive reflexes on diagnostic manometry.\n\nEXCLUSION CRITERIA\n\n1. Potentially lethal congenital or chromosomal anomalies\n2. Craniofacial defects (cleft lip or palate)\n3. History of GI surgery, ENT surgery, or neurosurgery\n4. Need for supplemental respiratory support of \\>2 LPM","1 Week","6 Months",{"count":547,"type":23},134,[67],"Neonatal ICU survivors often face challenges with oral eating, swallowing difficulties, and various aerodigestive issues, and commonly need chronic nasogastric tube feeding or gastrostomy feeding, which escalates socioeconomic burdens, hospital stays, neurodevelopmental delays, and parental suffering. This proposal addresses a vital need in neonatal gastroenterology by studying unique swallowing mechanisms and defining the basis for preventative and corrective therapies through new translational research initiatives via a randomized controlled trial using the novel intervention initiative- Parent Implemented Oral Nutrition, Eating, and Esophageal reflexes Reintegration (PIONEER©) protocol.",[551,552],"Deglutition Disorders in Infants","Chronic Tube Feeding in Hospitalized Infants",[554,555,556,557,558],"Neonatal","Deglutition Disorders","NICU infants","Gastrostomy prevention","Chronic tube feeding","2026-01-14",{"date":561,"type":45},"2026-01-16",{"date":563,"type":23},"2026-07-01",{"date":565,"type":23},"2032-06-30",{"name":51,"class":52},{"id":568,"slug":569,"hasResults":12,"nctId":570,"briefTitle":571,"officialTitle":571,"acronym":4,"eligibilityCriteria":572,"healthyVolunteers":12,"sex":18,"minAge":4,"maxAge":298,"enrollmentInfo":573,"targetDuration":4,"studyType":24,"phases":575,"briefSummary":576,"conditions":577,"keywords":4,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":578,"lastUpdatePostDateStruct":579,"startDateStruct":581,"completionDateStruct":582,"leadSponsor":584,"locationsCount":585},"100615322","phase-3-comparison-of-neurocognitive-outcome-in-two-standard-regimen-for-treatment-of-low-risk-medulloblastoma-100615322","NCT07291102","Comparison of Neurocognitive Outcome in Two Standard Regimen for Treatment of Low-risk Medulloblastoma","Inclusion Criteria for screening:\n\n* Age at diagnosis \\\u003C 5 years\n* Patients with institutional suspicion or diagnosis of SHH-activated MB\n* Patient and family in social circumstances that will allow neuropsychological follow-up\n* Ability of parents\u002Flegal representatives to understand the patient information and to personally sign and date the informed consent to participate in screening procedures\n* Patient and the parents\u002Flegal representative are able and willing to participate in the entire study (if patient is eligible)\n\nExclusion Criteria for overall study:\n\n* Patients previously treated for any other brain tumor or any type of malignant disease\n* Patients, in whom compliance with toxicity management guidelines and study procedures cannot be assured\n* History of hypersensitivity to an investigational medicinal product or to any drug with similar chemical structure or to any excipient present in the pharmaceutical form of an investigational medicinal product.\n* Patients\u002Fparents who do not wish to abstain from treatment with live vaccines during study participation\n* Patients with a language barrier too extensive to complete neuropsychological tests based on the investigator's judgement\n* Patients with severe premorbid developmental delay (based on the investigator's judgement), which will not allow WPPSI-IV assessment after 2.5 years\n* Patients cannot undergo MRI\n\nInclusion Criteria for Bridging chemotherapy (carboplatin\u002Fetopiside) in interventional arms:\n\n* Patients with SHH-activated MB, TP53-wt demonstrated by IHC for Gab1 or p75-NGFR, Yap1, beta-catenin, and TP53 (lack of strong and widespread nuclear p53 positivity) on central review according to WHO classification 2021.\n* No clinical evidence of extra-CNS metastases\n* Negative CSF cytology\n* No prior therapy for MB other than surgery\n* No other medical contraindications to chemotherapy:\n* No uncontrolled invasive fungal infection or other severe systemic infection requiring system\u002Fparental therapy\n* No other severe organ dysfunctions, which cannot be clinically controlled\n* No concomitant use with yellow fever vaccine and with live virus and bacterial vaccines\n* No demyelinating form of Charcot-Marie-Tooth syndrome\n* Assessment of hearing function completed\n* No evidence of cancer predisposition syndromes other than Gorlin syndrome or ELP1, GPR161 germline alterations.\n* Provided written informed consent by parent(s)\u002Fparent representative(s) by bridging chemotherapy\n* Patient should be enrolled within 28 days after diagnosis. Bridging chemotherapy can start as early as criteria for enrollment are met, and must start no later than 33 days after diagnosis\n\nExclusion Criteria for bridging chemotherapy:\n\n* One or more of the inclusion criteria for bridging chemotherapy are lacking\n* Other histology than SHH MB\n\nInclusion Criteria for randomization:\n\n* Patient has received bridging chemotherapy as described in this protocol\n* Patients with centrally reviewed SHH-activated MB, TP53-wt, according to WHO classification 2021\n* Absence of metastatic disease on central radiology review\n* Exclusion of TP53-mutation by DNA sequencing of the TP53-gene from tumor tissue by central review. Results from local institution will be accepted if raw data of this analysis is forwarded to the national central review institution\n* Confirmation of SHH activation by DNA methylation-based classification on central review. Results from local institution will be accepted if raw data of this analysis is forwarded to the national central review institution\n* No amplification of MYC (amplification of MYCN allowed). Array-based technologies (850k array, molecular inversion probe assay (MIP)), array-based comparative genomic hybridization (array-CGH) or next generation sequencing (NGS) DNA sequencing coverage MYC locus will be used. If these alternative assays give any indication of possible amplification, FISH will be performed on central review.\n* No other medical contraindications to chemotherapy:\n* No uncontrolled invasive fungal infection or other severe systemic infection requiring system\u002Fparental therapy\n* No other severe organ dysfunctions, which cannot be clinically controlled\n* No concomitant use with yellow fever vaccine and with live virus and bacterial vaccines\n* No demyelinating form of Charcot-Marie-Tooth syndrome\n* Retrospective assessment of pre-operative health-related QoL (HR-QoL) measured by Pediatric Quality of Life Inventory\n* Provided written informed consent by parent(s)\u002Fparent representative(s) for randomization\n\nExclusion criteria for randomization:\n\n-Patients are excluded from the interventional study if any of the following criteria are met:\n\n1. One or more of the inclusion criteria for randomization are lacking\n2. Patients with metastatic disease\n3. TP53-mutated SHH MB\n4. MYC amplified MB\n5. Pre-existing condition incompatible with scheduled therapy (e.g. Fanconi anemia)\n6. Patients with non-communicating hydrocephalus, e.g. due to perinatal intracranial haemorrhage, adueductal stenosis, or meningitis\n7. Contraindication for any components of the randomized therapies, including HDCT and intraventricular chemotherapy. Note: postperative hydrocephalus is not a contraindication for i.ventr. MTX.",{"count":574,"type":23},96,[476],"This is a trial to compare neurocognitive outcomes in the intent-to-treat population 2.5 years after diagnosis between patients with newly diagnosed, non-metastatic, SHH-activated, TP53-wt, non-MYC amplified MF randomized to the interventional arms A (\"Head Start 4\") or B (HIT-SKK).",[244],"2025-12-17",{"date":580,"type":45},"2025-12-18",{"date":563,"type":23},{"date":583,"type":23},"2038-10",{"name":51,"class":52},2,{"id":587,"slug":588,"hasResults":12,"nctId":589,"briefTitle":590,"officialTitle":591,"acronym":4,"eligibilityCriteria":592,"healthyVolunteers":12,"sex":18,"minAge":593,"maxAge":472,"enrollmentInfo":594,"targetDuration":4,"studyType":24,"phases":595,"briefSummary":596,"conditions":597,"keywords":602,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":609,"lastUpdatePostDateStruct":610,"startDateStruct":612,"completionDateStruct":614,"leadSponsor":615,"locationsCount":83},"100539426","vr-pat-and-fnirs-to-identify-cns-biomarkers-of-pain-100539426","NCT06303687","VR-PAT and fNIRS to Identify CNS Biomarkers of Pain","Imaging Pain in a Virtual World: Neuroimaging Biomarkers for Assessing Brain Mechanisms Mediating Virtual Reality Pain Management in Children","Inclusion Criteria:\n\n1. Being treated for acute burn injury\n2. Age 6-17 years, inclusive\n3. Admitted to NCH burn unit for treatment\n4. Have a dressing that requires daily changes over 3 days\n5. Patient and family caregivers can communicate (read and write) using English\n\nExclusion Criteria:\n\n1. Any wounds that may interfere with study procedures\n2. Vision, hearing, or cognitive\u002Fmotor impairments preventing valid administration of study measures\n3. History of motion sickness, seizure disorder, dizziness, or migraine headaches precipitated by visual auras\n4. Minors in foster care, prisoners, or currently pregnant\n5. Suspected child abuse\n6. Unable to communicate in English","6 Years",{"count":265,"type":23},[67],"This study aims to use functional near-infrared spectroscopy (fNIRS) to identify and quantify pain biomarkers during burn dressing changes and pain relief induced by virtual reality (VR).",[598,599,600,601],"Acute Pain","Pediatric ALL","Procedural Pain","Burns",[603,604,605,606,607,608],"Virtual Reality","VR","fNIRS","Pain","Biomarker","Neuroimaging","2025-11-19",{"date":611,"type":45},"2025-11-24",{"date":613,"type":23},"2026-07",{"date":381,"type":23},{"name":51,"class":52},{"id":617,"slug":618,"hasResults":12,"nctId":619,"briefTitle":620,"officialTitle":621,"acronym":622,"eligibilityCriteria":623,"healthyVolunteers":12,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":624,"targetDuration":4,"studyType":24,"phases":626,"briefSummary":627,"conditions":628,"keywords":630,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":637,"lastUpdatePostDateStruct":638,"startDateStruct":640,"completionDateStruct":642,"leadSponsor":644,"locationsCount":83},"100575211","preparing-for-life-and-academics-for-young-childhood-cancer-survivors-100575211","NCT06769334","Preparing for Life and Academics for Young Childhood Cancer Survivors","Digital Health Intervention to Improve Neurodevelopmental Outcomes for Young Childhood Cancer Survivors in Underserved Communities","PLAY","For the caregiver:\n\n* Caregiver is the legal guardian of the child (e.g., mothers, fathers, grandparents, adoptive parents)\n* Caregiver lives with the child more than 50% of the time\n* Caregiver primary language is English\n* Caregiver lives within 150 miles of Nationwide Children's Hospital\n\nFor the childhood cancer survivor:\n\n* Received treatment for their cancer (e.g., chemotherapy, surgical resection, radiation therapy)\n* Completed cancer treatment or on maintenance therapy only, without evidence of recurrent disease\n* Childhood cancer survivor is currently between the ages of 3 years old to 6 years old, but no older than 6.5 years old at study enrollment\n\nExclusion Criteria for the childhood cancer survivor:\n\n* Has a documented neurodevelopmental disorder prior to their cancer diagnosis (e.g., neurofibromatosis, tuberous sclerosis, Down's syndrome, autism)\n* Did not receive any treatment for their cancer (e.g., chemotherapy, surgical resection, radiation)\n* Treated for a benign tumor\n\nFor the teacher:\n\nClassroom or daycare teacher of the childhood cancer survivor",{"count":625,"type":23},105,[67],"The goal of this clinical trial is to test and improve an online program for caregivers of young childhood cancer survivors called the Preparing for Life and Academics for Young Survivors program (PLAY). The PLAY program was created with a group of caregivers of young children with cancer and healthcare providers. Ultimately, investigators hope to see if the program can improve positive parenting behaviors, reduce caregiver stress, and help get young children ready for school. In the preliminary phase of this clinical trial, investigators are aiming to answer these questions:\n\n1. Are caregivers of childhood cancer survivors willing to participate in the PLAY program and complete assessments before and after the program?\n2. Do caregivers of childhood cancer survivors rate the PLAY program as easy to use?\n3. Are caregivers of childhood cancer survivors satisfied with the PLAY program?\n4. How can the PLAY program be improved in the future?\n5. Does PLAY help improve how caregivers interact and read with their children or how they manage their own stress?\n\nWhen the child is transitioning to maintenance phase therapies or is no longer actively in cancer treatment, caregivers will be invited to participate. If they agree to participate, they will complete surveys when they begin the study as well as a videotaped interaction task reading and playing with their child. Children will complete brief developmental testing at the beginning of the program.\n\nCaregivers will then complete the PLAY Program, which will involve completing 7 weekly or bi-weekly online modules and meeting with a trained coach by videoconference for up to 10 one-hour sessions over three months. Sessions will focus on helping their child get ready for school and helping their family adjust and cope with stress.\n\nCaregivers will repeat the surveys and videotaped interaction task again three months later, after the PLAY program is completed. They will also be invited to participate in an interview to learn about their experience in the program.\n\nThis study will happen over two phases. Participants in Phase I of this study will complete all parts of the study as described above. Participants in Phase II (beginning in winter 2025) will complete all parts of the study as described above and a six month follow up that will involve repeating the similar questionnaires and an additional videotaped interaction with their child. Teachers of children will also take part in Phase II with questionnaires at baseline and 3-months later, after participants have completed the PLAY program.",[629],"Childhood Cancer",[631,632,633,634,635,636],"positive parenting","development","school readiness","digital health","caregiver stress","school advocacy","2025-11-14",{"date":639,"type":45},"2025-11-17",{"date":641,"type":45},"2024-12-12",{"date":643,"type":23},"2027-01-30",{"name":51,"class":52},{"id":646,"slug":647,"hasResults":12,"nctId":648,"briefTitle":649,"officialTitle":650,"acronym":651,"eligibilityCriteria":652,"healthyVolunteers":12,"sex":18,"minAge":19,"maxAge":92,"enrollmentInfo":653,"targetDuration":4,"studyType":24,"phases":655,"briefSummary":656,"conditions":657,"keywords":4,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":659,"lastUpdatePostDateStruct":660,"startDateStruct":662,"completionDateStruct":663,"leadSponsor":665,"locationsCount":83},"100595014","phase-1-phase-iii-clinical-trial-of-universal-donor-cd33-car-natural-killer-cells-for-aml-100595014","NCT07026942","Phase I\u002FII Clinical Trial of Universal Donor CD33 CAR Natural Killer Cells for AML","A Phase I\u002FII Clinical Trial Evaluating the Safety and Efficacy of Universal Donor CD33 CAR Natural Killer Cells for Treatment of Relapsed\u002FRefractory Acute Myeloid Leukemia","CD33 CAR NK","Inclusion Criteria:\n\n1. Patients with relapsed or primary refractory CD33+ AML, including:\n\n   * Patients with relapsed AML (patients in second or subsequent relapse, or any relapse after HSCT, are eligible).\n   * Refractory AML defined as failure to achieve a complete response after 2 cycles of induction or reinduction chemotherapy, including persistent MRD positivity.\n   * Patients with isolated CNS or extramedullary disease are eligible. Patients with CNS disease are excluded from the phase I dose escalation portion but are eligible for the phase II portion of the study.\n2. 1-39.99 years of age (note: the first three subjects treated AND the first subject on each dose level must be ≥ 16 years of age)\n3. Negative serum test to rule out pregnancy within 14 days prior to enrollment in females of childbearing potential\n\n   o Sexually active males and females of childbearing potential must agree to use a form of contraception considered effective and medically acceptable by the Investigator for 6 months after the last dose of chemotherapy and\u002For NK cell infusion\n4. Organ function requirements:\n\n   * Renal function: Creatinine ≤ 2 times the institutional upper limit of normal for age OR creatinine clearance \\> 60 ml\u002Fmin\u002F1.73m2 (measured by 24 hour- urine specimen or radioisotope GFR)\n   * Liver function: Total bilirubin ≤ 2 mg\u002Fdl (unless Gilbert's syndrome), AST and ALT ≤ 5 times the upper limit of normal (unless related to leukemic involvement). Upper limit of normal should be determined by the institutional defined normal laboratory range.\n   * Cardiac function: left ventricular ejection fraction ≥ 40% or shortening fraction ≥20%. May be eligible after cardiology clearance if qualitatively normal function or repeat measures are normal.\n   * CNS: Patients with seizure disorder may be eligible if seizures are well controlled\n   * Pulmonary function: baseline oxygen saturation \\>92% on room air at rest\n5. Due to the risk of hematopoietic toxicity from CD33 targeting, enrolled subjects must have an allogeneic HCT donor identified and be eligible and willing to undergo a subsequent HSCT in the event of aplasia.\n6. All patients or their legal guardian must be able to understand and willing to sign a written informed consent document.\n7. All patients must consent to enroll in a separate long term follow up study for cell and gene therapy\n\nExclusion Criteria:\n\n1. Prior therapies:\n\n   * AML directed therapies in the 14 days prior to beginning treatment on this protocol (except for hydroxyurea) Note: There is no waiting period required for patients having received intrathecal cytarabine, methotrexate and\u002For hydrocortisone\n   * Gemtuzumab or other CD33-targeted antibody within 42 days of enrollment\n   * CNS radiation within 28 days of enrollment\n   * DLI or adoptive cell therapy within 30 days of enrollment\n   * Allogeneic SCT within 90 days of enrollment\n\n     * Patients with CNS disease are excluded from the phase I portion of the study but are eligible for the phase II expansion phase.\n     * Patients on immunosuppressive therapy\n   * Patients must be off of systemic immunosuppressive therapy for at least 14 days prior to enrollment with no evidence of recurrent GVHD\n   * Patients on hydrocortisone for treatment of adrenal insufficiency are permitted on study\n   * Patients on corticosteroids ≤ 0.5mg\u002Fkg\u002Fday (prednisone equivalent) for any other indication are permitted on study\n2. Uncontrolled, symptomatic, intercurrent illness or social situations that would limit compliance with study requirements or in the opinion of the site PI would pose an unacceptable risk to the subject\n3. Patients who are breastfeeding\n4. Patients with prior solid organ transplantation\n5. Performance status: Karnofsky or Lansky Performance Scale (PS) \\\u003C 50\n6. Uncontrolled infection, defined as an infection which has not resolved or does not show evidence of significant resolution after initiating appropriate therapy\n\n   o Asymptomatic viremia such as CMV, HPV, BK virus, HCV, etc. is NOT considered as an exclusion criterion\n7. Uncontrolled arrhythmias or uncontrolled symptomatic cardiac disease\n8. Active acute or chronic GVHD of any grade at the time of enrollment. \"Active GVHD\" is defined as a patient who requires immunosuppressive therapy for control of their GVHD symptoms.",{"count":654,"type":23},42,[239,96],"This phase 1\u002F2 study is testing a new treatment for acute myeloid leukemia (AML) that has come back or has not responded to other treatments. The treatment uses specially modified immune cells (called CD33 CAR-NK cells) from a healthy, unrelated donor to attack the cancer.\n\nThe first part of the study (Phase I) will focus on finding the safest and most effective dose. The second part (Phase II) will test how well the treatment works at that dose.\n\nPatients will undergo screening, chemotherapy (Fludarabine and Cytarabine, in combination with Venetoclax) followed by the infusion of the CD33 CAR NK cells. Some patients may receive 2 doses of CD33 CAR NK cells infused 1 week apart. The investigator will let participants know if they will receive 1 or 2 doses. Patients will be hospitalized for the chemotherapy and CD33 CAR NK cell infusion for close monitoring and will remain in the hospital until blood counts recover. If patients are discharged from the hospital before day 35, they will be followed in clinic weekly for blood work and a physical exam.\n\nA bone marrow biopsy will be performed around day 28-35 to see if the patient's leukemia is in remission. Lumbar puncture or imaging may also be done if the study doctor thinks it is necessary.\n\nPatients will continue to be followed for research studies and clinical outcomes (leukemia relapse, survival) for 1 year. After 1 year, patients will have completed their study participation, but can be monitored for up to 15 years for potential long term side effects of the cell therapy. Some patients may undergo a bone marrow transplant after the study treatment. Patients who proceed to bone marrow transplant will have one blood sample drawn about a month after the transplant and then will have completed study participation.",[658],"Relapsed\u002FRefractory AML","2025-11-10",{"date":661,"type":45},"2025-11-12",{"date":409,"type":23},{"date":664,"type":23},"2038-07-01",{"name":51,"class":52},""]