[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"medulloblastoma-recurrent\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:medulloblastoma-recurrent":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,9,0,[8,55,122,145,224,250,282,312,345],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":33,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":44,"lastUpdatePostDateStruct":45,"startDateStruct":48,"completionDateStruct":50,"leadSponsor":52,"locationsCount":4},"100644286","phase-1-ngfus-nivo-neuronavigation-guided-focused-ultrasound-with-nivolumab-in-relapsed-and-progressive-dmg-and-other-high-grade-brain-tumors-100644286",false,"NCT07664176","NgFUS NIVO: NeuroNavigation-Guided Focused Ultrasound With Nivolumab in Relapsed and Progressive DMG and Other High Grade Brain Tumors","A Safety and Feasibility Study of NeuroNavigation-Guided Low Intensity Focused Ultrasound With Microbubbles to Enhance Nivolumab Delivery for the Treatment of Relapsed and Progressive Diffuse Midline Glioma and Other High Grade Brain Tumors","Inclusion Criteria:\n\n* Age ≥ 3 and ≤ 25 years.\n* Diagnosis of brainstem DMG\u002FDIPG or any high-grade brain tumor.\n\n  * Group A: Relapsed or progressive brainstem DMG.\n  * Group B: Relapsed or progressive high-grade intracranial brain tumor requiring surgical resection.\n* Lansky\u002FKarnofsky rating ≥ 60.\n* Patients must have received at least one line of prior therapy upfront for their disease.\n* At least four weeks from radiation therapy, prior immunotherapy, or monoclonal antibody therapy.\n* At least 2 weeks from prior myelosuppressive chemotherapy and post nadir meeting organ function criteria.\n* At least 1 week or 5 half-lives (whichever is longer) from last targeted therapy.\n* If on steroids, stable or decreasing dose for at least 7 days prior to study entry and ≤ 0.4 mg\u002Fm2\u002Fday of dexamethasone or equivalent.\n* Stable or improving neurological status for 7 days prior to study entry.\n* Organ function:\n\n  * Absolute Neutrophil Count (ANC) ≥750\u002FμL.\n  * Absolute Lymphocyte Count (ALC) \\>500\u002FμL.\n  * Platelets ≥75K, unsupported.\n  * Coagulation studies: PT and PTT \\\u003C1.5 ULN and INR (\\\u003C1.5).\n  * Bilirubin ≤1.5x upper limit of normal (ULN).\n  * AST\u002FALT ≤5x ULN.\n  * Serum creatinine within normal limits for age.\n  * Pulse oximetry \\>93% on room air.\n  * Ejection Fraction (EF) above institutional lower limit of normal (LLN).\n* For females of childbearing potential (FOCBP): negative pregnancy test within 7 days of study entry.\n* Patients of childbearing or child-fathering potential must agree to use contraceptive measures for at least 5 months following nivolumab infusion.\n* Patient or parent\u002Fguardian capable of providing informed consent.\n\nExclusion Criteria:\n\n* Symptoms and signs of increased intracranial pressure.\n* Patients with metallic ventricular peritoneal shunts. Subjects with nonmetallic VP shunts or similar will have a technical evaluation of the screening non-contrast CT scan of the head. During the mapping of the target area, if the technical NaviFUS specialist determines that the patient cannot be treated within the safety limits of the system, the patient will not be eligible and will be considered a screen failure.\n* Tumor presenting with the following imaging characteristics:\n\n  * Evidence of uncal herniation.\n  * Edema and\u002For mass effect that causes hydrocephalus.\n  * Significant areas of necrosis within the tumor that the neurosurgeon feels cannot be avoided during the ultrasound sonication.\n  * Evidence of a significant new hemorrhage. Area of microhemorrhage (defined as less than 5 mm in diameter) in the treatment area can be acceptable but requires the review of the neurosurgeon.\n  * Containing calcifications in the focused ultrasound sonication beam path and system tools cannot tailor the treatment around these calcification spots.\n  * Patients who are deemed to have overly bulky tumor by the PI of the study.\n* The sonication pathway to the tumor involves:\n\n  * More than 30% of the skull area traversed by the sonication pathway is covered by scars, scalp disorders (e.g., eczema), or atrophy of the scalp.\n  * Clips, or other non-MRI compatible metallic implanted objects in the skull or the brain, except for shunts.\n* Patients receiving anti-coagulant therapy, or medications known to increase risk of hemorrhage, (e.g., ASA, non-steroidal anti-inflammatory drugs \\[NSAIDs\\], statins). There is no required washout for eligibility assessment, but patients should be off agents for at least 3 days at the time of procedure or until 5 half-lives of the agent, whichever is longer.\n* History of a bleeding disorder, coagulopathy or with a history of clinically significant spontaneous tumor hemorrhage.\n* Cerebral or systemic vasculopathy, including intracranial thrombosis, vascular malformation, cerebral aneurysm, or vasculitis.\n* Immunosuppression (corticosteroids to prevent\u002Ftreat brain edema are permitted).\n* Patients with uncontrolled HIV.\n* Active seizure disorder or epilepsy (clinically significant seizures despite medical treatment) within four weeks prior to first cycle\u002FNaviFUS BBBO procedure captured by history.\n* Known sensitivity to gadolinium-based contrast agents.\n* Known sensitivity to Lumason® ultrasound contrast agent or known hypersensitivity to sulphur hexafluoride microsphere or its components, e.g., polyethylene glycol.\n* Patients unable to fit comfortably into the MRI scanner (generally \\>250 lbs.).\n* Evidence of cranial or systemic infection.","ALL","3 Years","25 Years",{"count":20,"type":21},30,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for progressive and relapsed diffuse midline glioma (DMG) and other progressive and relapsed high-grade brain tumors. This study combines intravenous nivolumab therapy infused following transient blood-brain barrier opening (BBBO) using low-intensity focused ultrasound with microbubble (LIFU-MB) treatment using NeuroNavigation-Guided Focused Ultrasound (NgFUS).\n\nThere are two groups in this study:\n\n* Group A: Patients with relapsed or progressive diffuse midline glioma in the brainstem\n* Group B: Patients with relapsed or progressive high grade brain tumor that clinically require surgical resection\n\nThe primary outcome is to evaluate the safety and feasibility of 3 cycles of nivolumab with BBB disruption using NgFUS with microbubbles in pediatric patients with progressive or relapsed brainstem DMG or with high grade brain tumors after surgery. Secondary outcomes include preliminary efficacy and immunological effects.",[27,28,29,30,31,32],"Diffuse Midline Glioma or Diffuse Intrinsic Pontine Glioma","High Grade Gliomas","Medulloblastoma Recurrent","Ependymoma Recurrent","Atypical Teratoid\u002FRhabdoid Tumor (ATRT) of the CNS","Brain Tumor Recurrent",[34,35,36,37,38,39,40,41,42],"Focused Ultrasound","Pediatrics","Diffuse Midline Glioma","DMG","Diffuse Intrinsic Pontine Glioma","DIPG","High Grade Glioma","HGG","Nivolumab","NOT_YET_RECRUITING","2026-06-17",{"date":46,"type":47},"2026-06-24","ACTUAL",{"date":49,"type":21},"2026-06-01",{"date":51,"type":21},"2030-12",{"name":53,"class":54},"Children's National Research Institute","OTHER",{"id":56,"slug":57,"hasResults":11,"nctId":58,"briefTitle":59,"officialTitle":60,"acronym":61,"eligibilityCriteria":62,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":63,"targetDuration":4,"studyType":22,"phases":65,"briefSummary":67,"conditions":68,"keywords":87,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":112,"lastUpdatePostDateStruct":113,"startDateStruct":115,"completionDateStruct":117,"leadSponsor":119,"locationsCount":121},"100562786","phase-1-study-in-children-and-adolescents-of-177lu-dotatate-lutathera-combined-with-the-parp-inhibitor-olaparib-for-the-treatment-of-recurrent-or-relapsed-solid-tumours-expressing-somatostatin-receptor-sstr-luparped-100562786","NCT06607692","Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib for the Treatment of Recurrent or Relapsed Solid Tumours Expressing Somatostatin Receptor (SSTR) (LuPARPed).","Single-arm Open-label Phase I\u002FII Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib for the Treatment of Recurrent or Relapsed Solid Tumours Expressing Somatostatin Receptor (SSTR) (LuPARPed).","LUPARPED","Inclusion criteria:\n\n* 18 months - 18 years of age at the time of the initial diagnosis.\n* ≥ 3 years at the moment of inclusion in the trial.\n* Diagnosis: relapsed\u002Frefractory solid tumours with positive uptake on SSTR-PET (PET-CT or PET-MRI), performed in the previous three months before entering the study.\n\nThe evaluation of SSTR expression will be classified according to a qualitative 4-point scale: SSTR expression V (visual score):\n\n* Score = 0: Below or equal to blood pool\n* Score = 1: Above blood pool and lower than liver\n* Score = 2: Equal to or above liver and lower than spleen\n* Score = 3: Equal to or above spleen\n\nPatients with scores ≥ 2 in the majority of the tumoral lesions will be considered to have a positive SSTR-PET and will be therefore eligible for the trial. Patients with a higher score are presumed to have a better response to the treatment.\n\nIt is admissible to have non-measurable disease only (e.g., HR-NB with bone-only or bone-marrow-only active disease).\n\n* Performance status ≥ 50% according to Lansky scale (\\\u003C16 years old) or Karnofsky scale (for ≥16 years old).\n* Life expectancy of at least 3 months.\n* Availability of ability to swallow tablets or capsules.\n* Adequate organ function within 28 days prior to enrolment, as defined by:\n\n  * Hb ≥10 g\u002Fdl (packed red blood transfusion is acceptable up to 24 hours prior starting treatment);\n  * White blood cell (WBC) count ≥ 2500\u002FμL (equivalent to 2.5 x 109\u002FL)\n  * Absolute Neutrophil Count (ANC) ≥ 1000\u002Fμl;\n  * Platelets ≥ 100.000\u002Fμl, without transfusion in the prior ≥7 days;\n  * Serum plasma creatinine ≤ 1.5 x upper limit of normal (ULN) OR estimated glomerular filtration rate (eGFR) ≥ 60 mL\u002Fmin\u002F1.73m2 (assessed by 2009-Schwartz formula).\n  * Total bilirubin ≤ 1.5 x the institutional ULN. For patients with known Gilbert's Syndrome ≤ 3.0 ULN is permitted.\n  * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 3.0 ULN.\n  * Albumin \\>3.0 g\u002FdL (3.0 g\u002FdL is equivalent to 30 g\u002FL)\n* A negative serum or urine pregnancy test in women with onset of menses or ≥12 years of age.\n* Patients of reproductive potential must agree to use highly effective contraceptive methods for the entire study duration and up to 7 months, in case of females, and 4 months in case of males, after the last dose of Lutathera, or up to 6 months, in case of females, and 3 months in case of males, after the last dose of olaparib, whichever takes places later.\n* Have the ability to comprehend and willingness to provide written informed consent (ICF) for the study before patient registration or any trial-related screening procedures. If the patient is \\\u003C18 years old, the written informed consent must be signed by the parent(s) or legal guardian(s) according to national regulations. In the case of patients between 12 and 17 years, they must sign an assent form, and if the patient turns 18 during their participation in the study, they must sign an informed consent form.\n* Adequate recovery from major surgery prior to receiving study treatment.\n* Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial.\n\nExclusion criteria\n\n* Previous significant drug-induced hepatitis toxicity experienced in the past that has required treatment dose reductions, treatment discontinuation or that, at the investigator discretion, could infer a risk.\n* Having received more than one previous treatment with other radiolabelled somatostatin analogues.\n* Inability to swallow tablets or capsules.\n* Subjects who are currently receiving any other anticancer and\u002For investigational agents (e.g. chemotherapy, immunotherapy or biological therapy \\[including monoclonal antibodies\\]). There must be at least 28 days of washout from any prior treatment. In case of checkpoints inhibitors, there should be at least 4 months of washout. Palliative Radiation Therapy for symptom control (e.g. pain relief) could be acceptable, at the discretion of the investigator.\n* Treatment with long-acting somatostatin analogues within 28 days prior the administration of 177Lu-DOTATATE.\n* Known hypersensitivity to any of the excipients.\n* Subjects who have an uncontrolled infection.\n* Lactating women.",{"count":64,"type":21},25,[24,66],"PHASE2","Study in children and adolescents of 177Lu DOTATATE (Lutathera®) combined with the PARP inhibitor olaparib for treatment of recurrent or relapsed solid tumours expressing somatostatin receptors (SSTR) (LuPARPed)",[69,70,71,28,72,73,74,75,76,39,77,78,79,80,81,82,83,84,29,85,86,30],"Solid Tumor Cancer","Medulloblastoma","High Risk Neuroblastoma","Meningioma","Paraganglioma","Pheochromocytoma","Neuroendocrine Tumours (NET)","Adrenal Tumours","Glioblastoma","Glioblastoma (GBM)","Osteosarcoma Recurrent","Carcinoma Adrenal","Synovial Sarcomas","Esthesioneuroblastoma","Neuroblastoma Recurrent","Solid Tumor Refractory to Conventional Treatment","Paraganglioma\u002F Phaeochromocytoma","Ependymoma",[88,89,90,91,92,93,94,95,96,97,98,99,100,39,101,102,103,104,105,106,107,108,109,86,110],"solid tumours","somatostatin receptors","SSTR","LUTETIUM","OLAPARIB","MEDULLOBLASTOMA","NEUROBLASTOMA","HIGH GRADE GLIOMA","MEININGIOMA","PARAGANGLIOMA","PHEOCHROMOCYTOMA","NEUROENDOCRINE TUMOURS","ADRENAL TUMOR","GLIOBLASTOMA","OSTEOSARCOMA","CARCINOMA ADRENAL","SYNOVIAL SARCOMA","ESTHESIONEUROBLASTOMA","RECURRENT","RELAPSED","RELAPSE","REFRACTORY","Ependimoma Recurrent","RECRUITING","2026-06-02",{"date":114,"type":47},"2026-06-04",{"date":116,"type":47},"2024-09-20",{"date":118,"type":21},"2029-12",{"name":120,"class":54},"Fundación de investigación HM",1,{"id":123,"slug":124,"hasResults":11,"nctId":125,"briefTitle":126,"officialTitle":127,"acronym":128,"eligibilityCriteria":129,"healthyVolunteers":11,"sex":16,"minAge":130,"maxAge":18,"enrollmentInfo":131,"targetDuration":4,"studyType":22,"phases":133,"briefSummary":134,"conditions":135,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":137,"lastUpdatePostDateStruct":138,"startDateStruct":139,"completionDateStruct":141,"leadSponsor":143,"locationsCount":121},"100619556","phase-1-liothyronine-in-combination-with-bit-regimen-for-medulloblastoma-with-or-without-minimal-residual-disease-100619556","NCT07346157","Liothyronine in Combination With BIT Regimen for Medulloblastoma With or Without Minimal Residual Disease","PNOC044: Liothyronine (L-T3) in Combination With Bevacizumab, Irinotecan and Temozolomide (BIT) for Progressive or Relapsed Medulloblastoma (Cohort 1) or as Monotherapy for Medulloblastoma With Minimal Residual Disease (Cohort 2)","PNOC044","Inclusion Criteria:\n\n1. Phase 1 and Phase 2, Cohort 1:\n\n   Participants must have histologically confirmed medulloblastoma that is relapsed\u002Fprogressive following standard upfront therapy. Tissue confirmation of medulloblastoma diagnosis is required at diagnosis and not required at the time of relapse for entry into the study.\n2. Phase 2, Cohort 2: Participants must have cerebrospinal fluid (CSF) with cell-free deoxyribonucleic acid (cf-DNA) + assessed in a Chemiluminescent immunoassay (CLIA)-certified or protocol-approved laboratory. After entry into the study, another CSF sample will be collected and analyzed centrally prior to initiation of protocol therapy to verify cf-DNA positivity.\n3. Evidence of Disease:\n\n   Phase 1 and Phase 2, Cohort 1:\n\n   Participants may have either Measurable or Evaluable Disease Measurable Disease: Participants must have clear residual disease at the time of enrollment, defined as tumor that is measurable in two perpendicular dimensions on MRI Evaluable Disease: Diffuse leptomeningeal disease OR clear MRI evidence of disease that may not be measurable in two perpendicular dimensions.\n\n   Phase 2, Cohort 2:\n\n   For cf-DNA positive cohort: Participants are not required to have measurable or evaluable disease but must have cf-DNA positivity in a CLIA-certified or protocol-approved laboratory, as above.\n4. Prior Therapy: Participants must have received standard upfront therapy for medulloblastoma (either with craniospinal radiation or high dose chemotherapy and autologous stem cell rescue. If other therapy utilized, must be discussed with study chairs prior to participation). Participants for Phase 1 and Phase 2 cohort 1 may have received further chemotherapy and\u002For radiation therapy beyond standard upfront therapy prior to trial enrollment. Participants within the Phase 2 cohort 1 must have experienced at least one, and at most, two relapses prior to study enrollment.\n5. Age 1-25 years old.\n6. Performance Score: Karnofsky ≥ 50 for participants \\> 16 years of age and Lansky ≥ 50 for participants ≤16 years of age (See Appendix A). Participants 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.\n7. For those participants currently treated with levothyroxine (Synthroid) they must have stable dosing for a minimum of 3 months prior to enrollment.\n8. Organ Function Requirements\n\n   1. Peripheral absolute neutrophil count (ANC) ≥ 1000\u002Fcubic millimeters (mm3)\n   2. Platelet count ≥ 75,000\u002Fmicroliter (uL) (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).\n   3. A serum creatinine \\\u003C 1.5 institutional\u002Freference range upper Limit normal (ULN) based on age and gender\n   4. Total bilirubin ≤ 3 x upper limit of normal (ULN); in presence of Gilbert's syndrome, total bilirubin ≤ 6 x ULN or direct bilirubin ≤ 3 x ULN\n\n      * Alanine aminotransferase (ALT) ≤ 5 x upper limit of normal (ULN)\n      * Aspartate aminotransferase (AST) ≤ 5 x ULN. 3.3.7.4 Adequate Neurologic Function Defined as:\n      * Participants with seizure disorder may be enrolled if well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug. 3.3.7.5 Adequate Cardiac Function Defined as:\n      * Normal left ventricular systolic function on baseline transthoracic echocardiogram (TTE)\n      * Normal left ventricular systolic function is defined as left ventricular ejection fraction (LVEF) \\>55% or shortening fraction (SF) \\>28%.\n\n   AND\n   * No clinically significant arrhythmia on baseline ECG (sinus arrhythmia, sinus tachycardia, sinus bradycardia, early repolarization and 1st degree atrioventricular block when partial response (PR) interval \\\u003C 300 millisecond (ms) are not considered clinically significant arrhythmias).\n   * In addition, the following ECG findings are not considered clinically significant in the setting of a normal echocardiogram:\n   * Left axis deviation\n   * Left atrial enlargement\n   * Right atrial enlargement\n   * Possible left ventricular hypertrophy\n   * Possible right ventricular hypertrophy\n   * Non-specific T wave abnormality 3.3.7.6 For the Phase 1 cohort, normal adrenal axis function is required. For those participants in the Phase 2 cohort, must have controlled adrenal insufficiency \\>3 months (no change in steroid replacement or stress dose plan for at least 3 months).\n   * Normal adrenal axis function as defined as:\n   * Morning (AM) cortisol \\>11mcg\u002Fdeciliter (dL)\n   * If AM cortisol is \\\u003C11 microgram (mcg)\u002FdL, cosyntropin stimulation test with rise to \\>18\n9. Endocrine conditions: Participants with diabetes insipidus, diabetes melitus, or being treated with levothyroxine, must have stable dosing and control for minimum of 3 months prior to enrollment\n10. For Cohort 1 only: participants must have recovered from any surgical procedure before enrolling on this study (see below for examples of major, intermediate, and minor surgical procedures):\n\n    1. Participants with a major surgical procedure within 28 days prior to enrollment should be excluded.\n    2. Participants with an intermediate surgical procedure within 14 days prior to enrollment should be excluded.\n    3. For minor surgical procedures (including Broviac line or infusaport placement), participants should not receive the first planned dose of bevacizumab until the wound is healed and at least 7 days have elapsed.\n    4. There should be no anticipation of need for major surgical procedures during the course of the study.\n11. The effects of L-T3 with chemotherapy on the developing human fetus are unknown. For this reason and because chemotherapy agents as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and 4 months after completion of L-T3 and chemotherapy administration. 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.\n12. Participants must be enroll on PNOC COMP if PNOC COMP is open to accrual at the enrolling institution.\n13. A legal parent\u002Fguardian or participant must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.\n\nExclusion Criteria:\n\n1. For Cohort 1 only: participants who have previously been treated with BIT in combination. Treatment with individual bevacizumab, irinotecan or TMZ is not an exclusion criteria.\n2. Participants who have had myelosuppressive chemotherapy within 3 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier. (Participants receiving chemotherapy directly into the CSF at doses not expected to be myelosuppressive may have received therapy up to 7 days prior to enrollment).\n3. Participants must be at least 7 days since the completion of therapy with a biologic or small molecule agent or non-myelosuppressive chemotherapy agent. For any agent with known adverse events that can occur beyond 7 days after administration, the period prior to enrollment must be beyond the time during which adverse events are known to occur. Such participants should also be discussed with study chairs.\n4. Radiation: For participants on the Phase 1 and Phase 2 Cohort 1, the tumor designated as \"measurable\" for protocol purposes must not have received radiation within 6 weeks prior to study entry and focal radiation to areas of symptomatic metastatic disease must not be given within 14 days of study entry. If a new lesion occurs outside the radiation field, the participant is eligible to enroll at any time point from completion of radiation. For Cohort 2 participants, there is no required washout for radiation therapy.\n5. Participants who are receiving any other investigational agents.\n6. History of allergic reactions attributed to compounds of similar chemical or biologic composition to L-T3 or other agents used in study.\n7. Participants receiving any medications or substances that are strong inhibitors or strong inducers of CYP450 enzymes are ineligible. 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\u002Ftable.aspx; medical reference texts such as the Physicians' Desk Reference may also provide this information. As part of the enrollment\u002Finformed consent procedures, the participant and\u002For legal parent or guardian will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering.\n8. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.\n9. Women of childbearing potential must not be pregnant or breast-feeding.\n10. Human immunodeficiency virus- (HIV) positive participants will be ineligible if HIV therapy regimen has not been stable for at least 4 weeks or there is intent to change the regimen within 8 weeks following enrollment, or if they are severely immunocompromised.\n11. Diagnosis of Graves' Disease or other pre-existing hyperthyroid disease.\n12. Participants with severe protein calorie malnutrition that in the opinion of the investigator may not tolerate protocol therapy.\n13. Participants with previous or active clinical cardiovascular disease, including the history of heart failure, myocardial infarction, cardiomyopathy, or ventricular systolic dysfunction on TTE (LVEF \\\u003C55% or SF \\\u003C28%), clinically significant arrhythmia (including atrial fibrillation, atrial flutter, frequent ventricular ectopy), clinically significant peripheral vascular disease.\n14. Participants with uncontrolled systemic hypertension (systolic blood pressure \\> 95th percentile for age and height if participant is ≤ 17 years old)\n15. Participants with uncontrolled diabetes mellitus (HbA1c \\>8%) or uncontrolled diabetes insipidus","1 Year",{"count":132,"type":21},69,[24,66],"This is a Phase 1\u002FPhase 2 study assessing liothyronine (L-T3) immunotherapy and in combination with standard chemotherapy (bevacizumab, irinotecan and temozolomide (BIT)) in children and young adults with medulloblastoma that is relapsed or progressive after standard upfront therapy.",[70,136,29],"Medulloblastoma, Childhood","2026-05-28",{"date":49,"type":47},{"date":140,"type":21},"2026-07-30",{"date":142,"type":21},"2031-03-31",{"name":144,"class":54},"Sabine Mueller, MD, PhD",{"id":146,"slug":147,"hasResults":11,"nctId":148,"briefTitle":149,"officialTitle":150,"acronym":4,"eligibilityCriteria":151,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":152,"enrollmentInfo":153,"targetDuration":4,"studyType":22,"phases":155,"briefSummary":156,"conditions":157,"keywords":190,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":214,"lastUpdatePostDateStruct":215,"startDateStruct":217,"completionDateStruct":219,"leadSponsor":221,"locationsCount":223},"100355699","phase-1-hsv-g207-in-children-with-recurrent-or-refractory-cerebellar-brain-tumors-100355699","NCT03911388","HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors","Phase 1 Trial of Engineered HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors","Inclusion Criteria:\n\n* Age ≥ 36 months and \\\u003C 22 years\n* Pathologically proven malignant cerebellar brain tumor (including medulloblastoma, glioblastoma multiforme, giant cell glioblastoma, anaplastic astrocytoma, primitive neuroectodermal tumor, ependymoma, atypical teratoid\u002Frhabdoid tumor, germ cell tumor, or other high-grade malignant tumor) which is progressive or recurrent despite standard care including surgery, radiotherapy, and\u002For chemotherapy. A pathologically proven secondary malignant cerebellar tumor without curative treatment options is eligible.\n* Lesion must be ≥ 1.0 cm ≤ 3.0 cm in diameter and surgically accessible as determined by MRI. Larger tumors may be surgically debulked and treated if ≤ 3.0 cm after debulking\n* Patients must have fully recovered from acute treatment related toxicities of all prior chemotherapy, immunotherapy or radiotherapy prior to entering this study.\n* Myelosuppressive chemotherapy: patients must have received their last dose at least 3 weeks prior (or at least 6 weeks if nitrosurea)\n* Investigational\u002FBiologic agents: patients must have recovered from any acute toxicities potentially related to the agent and received last dose ≥ 7 days prior to entering this study (this period must be extended beyond the time during which adverse events are known to occur for agents with known adverse events ≥ 7 days). For viral therapy, patients must have received viral therapy ≥ 3 months prior to study entry and have recovered from all acute toxicities potentially related to the agent.\n* Monoclonal antibodies: The patient must have received last dose ≥ 21 days prior.\n* Radiation: Patients must have received their last fraction of craniospinal radiation (\\>24 Gy) or total body irradiation ≥ 3 months prior to study entry. Patients must have received focal radiation to symptomatic metastatic sites or local palliative radiation ≥ 28 days prior to study entry.\n* Autologous bone marrow transplant: Patients must be ≥ 3 months since transplant prior to study entry.\n* Normal hematological, renal and liver function (absolute neutrophil count \\> 1000\u002Fmm3, platelets \\> 100,000\u002Fmm3, prothrombin time (PT) or partial thromboplastin time (PTT) \\\u003C 1.3 x control, creatinine within normal institutional limits OR creatinine clearance \\>60 mL\u002Fmin\u002F1.73 m2 for patients with creatinine levels above institutional normal, total bilirubin \\\u003C 1.5 mg\u002Fdl, transaminases \\\u003C 3 times above the upper limits of the institutional norm)\n* Patients \\\u003C 16 years, Modified Lansky performance score ≥ 60; patients ≥ 16 years, Karnofsky performance score ≥ 60\n* Patient life expectancy must be at least 8 weeks\n* Written informed consent in accordance with institutional and FDA guidelines must be obtained from patient or legal guardian\n\nExclusion Criteria:\n\n* Any treatment outside the allowable guidelines outlined in section 5.1.\n* Diffuse, widespread, abnormal tumor pattern involving 3 or more lobes of the brain\n* Acute infection, granulocytopenia or medical condition precluding surgery\n* Pregnant or lactating females\n* Diagnosis of encephalitis or CNS infection \\\u003C 3 months prior, or receiving ongoing treatment for encephalitis, CNS infection or multiple sclerosis\n* Tumor involvement which would require ventricular or brainstem inoculation or would require access through a ventricle in order to deliver treatment\n* Required steroid increase within 1 week prior to G207 inoculation or patients requiring \\>2 mg of dexamethasone daily\n* Known HIV seropositivity\n* Concurrent therapy with any drug active against HSV (acyclovir, valacyclovir, penciclovir, famciclovir, gancyclovir, foscarnet, cidofovir) or any immunosuppressive drug therapy (except dexamethasone or prednisone).\n* Other current malignancy\n* Concurrent anticancer or investigational drug","21 Years",{"count":154,"type":21},24,[24],"This study is a clinical trial to determine the safety of inoculating G207 (an experimental virus therapy) into a recurrent or refractory cerebellar brain tumor. The safety of combining G207 with a single low dose of radiation, designed to enhance virus replication, tumor cell killing, and an anti-tumor immune response, will also be tested.\n\nFunding Source- FDA OOPD",[158,159,160,161,162,163,164,165,166,167,168,169,170,171,172,173,174,175,176,177,178,179,180,181,182,183,184,29,185,186,187,188,189],"Neoplasms, Brain","Glioblastoma Multiforme","Glioblastoma of Cerebellum","Neoplasms","Astrocytoma","Astrocytoma, Cerebellar","Neuroectodermal Tumors","Neuroectodermal Tumors, Primitive","Cerebellar PNET, Childhood","Cerebellar Neoplasms","Cerebellar Neoplasms, Primary","Cerebellar Neoplasm, Malignant","Cerebellar Neoplasm Malignant Primary","Neoplasm Metastases","Neoplasm Malignant","Neoplasms, Neuroepithelial","Neoplasms, Germ Cell and Embryonal","Neoplasms by Histologic Type","Neoplasms, Glandular and Epithelial","Neoplasms, Nerve Tissue","Central Nervous System Neoplasms, Primary","Central Nervous System Neoplasms, Malignant","Nervous System Neoplasms","Neoplasms by Site","Brain Diseases","Central Nervous System Diseases","Nervous System Diseases","HSV","Virus","Pediatric Brain Tumor","Nervous System Cancer","Primitive Neuroectodermal Tumor (PNET) of Cerebellum",[191,192,159,193,70,194,195,196,86,197,198,199,200,201,202,203,161,204,205,206,207,208,209,35,210,186,185,211,212,213],"Brain Tumor, Recurrent","Glioma","Gliosarcoma","Anaplastic Astrocytoma","Oligodendroglioma","Rhabdoid Tumor","Germ Cell Tumor","Choroid Plexus Carcinoma","Cerebral Primitive Neuroectodermal Tumor","Giant Cell Glioblastoma","Atypical teratoid\u002Frhabdoid tumor","Secondary Malignant Cerebellar Tumor","Embryonal Tumor","Oncolytic Virus Therapy","Virotherapy, Oncolytic","Immunotherapy","Central Nervous System Agents","Antineoplastic Agents","Pediatric","Oncolytic","Herpes Virus","G207","Oncolytic Herpes Virus","2026-05-13",{"date":216,"type":47},"2026-05-15",{"date":218,"type":47},"2019-09-12",{"date":220,"type":21},"2027-09-01",{"name":222,"class":54},"M.D. Anderson Cancer Center",3,{"id":225,"slug":226,"hasResults":11,"nctId":227,"briefTitle":228,"officialTitle":229,"acronym":230,"eligibilityCriteria":231,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":152,"enrollmentInfo":232,"targetDuration":4,"studyType":22,"phases":234,"briefSummary":236,"conditions":237,"keywords":4,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":242,"lastUpdatePostDateStruct":243,"startDateStruct":245,"completionDateStruct":247,"leadSponsor":249,"locationsCount":121},"100594313","early-phase-1-a-phase-01-study-of-cdna-for-tp53-checkpoint-inhibition-and-radiation-in-children-with-recurrent-progressive-or-refractory-cns-malignancies-100594313","NCT07017816","A Phase 0\u002F1 Study of cDNA for TP53, Checkpoint Inhibition and Radiation in Children With Recurrent, Progressive or Refractory CNS Malignancies.","JACOB: Joint Administration of cDNA for TP53, checkpOint Inhibition and Boost\u002FHypofractionated Radiation. A Phase 0\u002F1 Study in Children With Recurrent, Progressive or Refractory CNS Malignancies.","JACOB","Inclusion Criteria:\n\n* Tumor\n\n  a. Patients must have a recurrent, progressive, or refractory CNS malignancy for which there are not known curative options.\n\n  i. Medulloblastoma, ATRT, High grade gliomas, pineoblastomas, embryonal tumors NOS, CNS sarcomas, ependymomas, other high-grade malignancies that failed first line therapies.\n\nii. Patients should have received radiation for standard up-front therapy. iii. Patients must have received at least one line of standard of care treatment without limitation to the number of treatments received. b. Evidence of clinical and\u002For radiological progressive disease as defined by RAPNO criteria.\n\nc. Patients with metastatic disease are eligible d. Patients must have available archival (formalin-fixed paraffin embedded) or fresh tumor tissue for correlative studies.\n\n* Patient Characteristics a. The first 3 patients will be \\>7 years old to \\\u003C22 years old i. Following initial successful treatment of the first 3 patients: Patients must be ≥3yrs and \\\u003C22 years of age.\n\n  b. Must have recovered from all surgical interventions prior to the start of the radiation and maintenance phases c. Patients must have a BSA of 0.6m2 or more to be enrolled. 3. Previous treatment\n  1. Patients must have recovered from the acute effects of prior therapy. Adverse events resulting from prior surgical intervention will not be an ineligibility criterion.\n  2. Chemotherapy: Patients must have received their last dose of known chemotherapy at least two (2) weeks prior to receipt of SGT-53.\n  3. Biologic therapy: Patients must have received their last dose of biological agent \\>7 days prior to receipt of SGT-53.\n  4. Radiation therapy: Patients must be deemed to be a reasonable candidate for hypofractionated irradiation from a clinical standard of care perspective.\n\n  i. At least 3 months from craniospinal radiation therapy, ii. Other substantial bone marrow irradiation ≥6 weeks prior to enrollment, iii. Local palliative XRT (small volume) ≥2 weeks. iv. if progressive disease is documented by radiological evidence of disease in areas not previously radiated, no minimum time required.\n\n  e. Corticosteroids: Patients who are receiving dexamethasone or other corticosteroids must be on a stable or decreasing dose for at least one (1) week prior to enrollment. There will be a maximum allowable steroid dose of 0.5mg\u002Fm2\u002Fday dexamethasone equivalents f. Growth factors: Patients must have received their last dose of any short acting growth factor at least one week prior to treatment, for long acting or pegylated growth factors, the last dose must be at least two (2) weeks prior to start of treatment.\n\n  4\\. Neurologic Status\n\n  a. Patients should be at their neurological baseline for a minimum of one week prior to enrollment.\n\n  5\\. Performance Status\n\n  a. Performance status (KarnofskyPS for \\>16yrs, or Lansky PS for \\\u003C16yrs) assessed within two weeks must be \\>50.\n\n  i. Patients who are unable to walk because of neurologic deficits, but who are up in a wheelchair, will be considered ambulatory for assessing the performance score.\n\n  6\\. Organ Function - Patients must have normal organ and marrow function as defined below:\n  1. Absolute neutrophil count \\>750\u002Ful\n  2. Platelets ≥75,000\u002Ful\n  3. Hemoglobin ≥8g\u002FdL (may be supported with transfusion)\n  4. Total bilirubin ≤1.5x ULN\n  5. ALT\u002FAST ≤3x ULN\n  6. Serum albumin ≥2g\u002FdL\n  7. Normal cardiac function defined as ejection fraction within normal limits for age and gender\n  8. a serum creatinine based on age\u002Fgender as below or a creatinine clearance or serum GFR ≥ 70ml\u002Fmin\u002F1.73m2: Serum Creatinine for age\u002Fsex Age Maximum Serum Creatinine (mg\u002FdL) Male Female\n\n     1. \\\u003C2 years 0.6 0.6\n     2. \\\u003C6 years 0.8 0.8\n\n     6-\\\u003C10 years 1 1 10-\\\u003C13 years 1.2 1.2 13- \\\u003C16 years 1.5 1.4 \\> 16 years 1.7 1.4 The threshold creatinine values in this table were adapted 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     7\\. Pregnancy prevention- all patients of childbearing or child fathering potential must be willing to use an acceptable form of birth control while being treated on this study and at least 5 months after receiving the last study treatment.\n\n     8\\. Pregnancy status: Female patients must not be pregnant or nursing. Female patients of reproductive potential must also have a negative serum pregnancy test at the time of enrollment.\n\n     9\\. Informed Consent - Patient and\u002For guardian have the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines.\n\n     Exclusion Criteria:\n* 1\\. Low-grade-glioma, craniopharyngioma, and other non-malignant CNS tumors are excluded.\n\n  2\\. Patients with diagnosis of diffuse midline gliomas (DMGs) are excluded. 3. Patients with any clinically significant unrelated systemic illness (serious infection or significant cardiac, pulmonary, hepatic, or other organ dysfunction) that is likely to interfere with ability to tolerate study therapy or study procedure results.\n\n  4\\. Patients with pre-existing cardiac dysfunction defined as baseline EF of less than 35% 5. Patients with pre-existing pulmonary fibrosis, interstitial lung disease, and clinically significant pulmonary lung disease.\n\n  6\\. Patients who are receiving any other investigational drug therapy 7. Patients who in the opinion of the investigator cannot adhere to protocol requirements 8. Patients with history of autoimmune diseases that required treatment in the last 2 years are not eligible. Asymptomatic laboratory abnormalities (e.g. ANA, rheumatoid factor, altered thyroid function studies) will not render a patient ineligible in the absence of a diagnosis of an autoimmune disorder. Replacement therapy (e.g. thyroxine, insulin or physiologic corticosteroid replacement therapy) is not considered a form of systemic treatment.\n\n  9\\. Patients who have received a live vaccine within 30 days of start of study treatment are not eligible.\n\n  10\\. Patients with known HIV\u002FAIDS or acute\u002Fchronic Hepatitis B or C are excluded.\n\n  11\\. Patients who are prohibited from receiving radiation therapy (reached maximum lifetime dose) are not eligible.",{"count":233,"type":21},18,[235],"EARLY_PHASE1","This clinical trial is studying a drug called SGT-53 along with radiation and another drug called Nivolumab. It's for children with brain tumors that have come back, gotten worse, or didn't get better with earlier treatments. The main questions it aims to answer are:\n\nWhat is the right dose of SGT-53 that children can safely receive when it is used with radiation and Nivolumab? This dose will be used in the second phase of the trial.\n\nWhat side effects are there of SGT-53 when it is used with radiation and Nivolumab?\n\nHow does SGT-53 move through the body when given with radiation and Nivolumab?\n\nHow much of the SGT-53 drug is found in the tumor tissue? This will be tested in a small group of patients?\n\nParticipants will:\n\nFor the first treatment cycle:\n\nGet SGT-53 twice per week Get Nivolumab every 2 weeks Receive radiation therapy during week 2\n\nFor Cycles 2-6:\n\nGet SGT-53 once per week during even cycles and twice per week during odd cycles Get Nivolumab every 2 weeks For Cycles 7+ Get both SGT-53 and Nivolumab every 2 weeks In the phase 0 part of the study, 4 participants will have genetic testing performed on their tumor tissue after receiving SGT-53. These samples will be compared to another sample taken from the skin.",[238,29,239,240,241,30,28],"CNS Malignancies","ATRT Recurrent","Pineoblastoma","Embryonal Neoplasm","2026-04-24",{"date":244,"type":47},"2026-04-29",{"date":246,"type":47},"2025-12-16",{"date":248,"type":21},"2028-05-01",{"name":53,"class":54},{"id":251,"slug":252,"hasResults":11,"nctId":253,"briefTitle":254,"officialTitle":255,"acronym":256,"eligibilityCriteria":257,"healthyVolunteers":11,"sex":16,"minAge":258,"maxAge":259,"enrollmentInfo":260,"targetDuration":4,"studyType":22,"phases":262,"briefSummary":264,"conditions":265,"keywords":268,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":272,"lastUpdatePostDateStruct":273,"startDateStruct":275,"completionDateStruct":277,"leadSponsor":279,"locationsCount":281},"100443691","individualized-treatment-plan-in-children-and-young-adults-with-relapsed-medulloblastoma-and-ependymoma-100443691","NCT05057702","Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma","A Pilot Feasibility and Efficacy (Phase 2) Trial of Real Time Drug Screening and Genomic Testing to Determine an Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma","PNOC027","Inclusion Criteria:\n\n1. Participants must have recurrent medulloblastoma or recurrent ependymoma previously histologically confirmed. Participants must be experiencing their first or second relapse to be eligible.\n2. Participants must have surgically accessible disease.\n3. Prior Therapy:\n\n   1. The participant must have received at least one prior therapy at the time of initial diagnosis.\n   2. Relapsed medulloblastoma or relapsed ependymoma are eligible.\n   3. Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study and would be eligible for surgical resection per institutional guidelines\n\n      * Participants must have received last chemotherapy or biologic agent at least 7 days prior to registration.\n      * Monoclonal antibody treatment: \\> 21 days prior to registration.\n      * Bevacizumab participants must have received last dose \\> 21 days prior to study registration\n4. Participant must be a candidate for surgical resection or biopsy with anticipated ability to obtain the minimum tissue requirements for study.\n5. Radiation - Participants must have:\n\n   1. Had their last fraction of local irradiation to primary tumor \\>= 12 weeks prior to registration.\n   2. Had their last fraction of craniospinal irradiation or total body irradiation \\>= 12 weeks prior to registration\n   3. At least 14 days after local palliative radiation (small-port)\n6. Age \\>=12 months to \\\u003C= 39 years of age.\n7. Karnofsky \\>= 50 for participants \\> 16 years of age and Lansky \\>= 50 for participants \\\u003C= 16 years of age. Participants 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.\n8. Corticosteroids: Participants who are receiving dexamethasone or equivalent must be on a stable or decreasing dose for at least 1 week prior to registration.\n9. Organ Function Requirements (within 7 days prior to study registration)\n\n   1. Adequate Bone Marrow Function Defined as:\n\n      * Peripheral absolute neutrophil count (ANC) \\>= 750\u002Fmm\\^3\n      * Platelet count \\>= 75,000\u002Fmm\\^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).\n      * Hemoglobin \\>= 8 g\u002Fdl.\n   2. Adequate Renal Function Defined as:\n\n      * Creatinine clearance or radioisotope GFR \\>= 70 milliliter\u002Fminute (mL\u002Fmin) \u002F1.73 m\\^2 or\n      * A serum creatinine based on age\u002Fsex as follows:\n\n      Age \u002F Maximum Serum Creatinine (mg\u002FdL) Male \u002F Maximum Serum Creatinine (mg\u002FdL) Female.\n      * 1 to \\\u003C 2 years \u002F 0.6 \u002F 0.6.\n      * 2 to \\\u003C 6 years \u002F 0.8 \u002F 0.8.\n      * 6 to \\\u003C 10 years \u002F 1 \u002F 1.\n      * 10 to \\\u003C 13 years \u002F 1.2 \u002F 1.2.\n      * 13 to \\\u003C 16 years \u002F 1.5 \u002F 1.4.\n      * \\>= 16 years \u002F 1.7 \u002F 1.4.\n      * \\- The threshold creatinine values in this table were derived from the Schwartz formula for estimating Glomerular filtration rate (GFR) utilizing child length and stature data published by the Center for Disease Control (CDC) (Schwartz GJ and Gauthier B 1985).\n   3. Adequate Liver Function Defined as:\n\n      * Total Bilirubin \\\u003C= 1.5 x upper limit of normal (ULN) for age; in presence of Gilbert's syndrome, total bilirubin \\\u003C 3 x ULN or direct bilirubin \\\u003C 1.5 x ULN.\n      * Alanine aminotransferase (ALT) \\\u003C= 3x ULN.\n      * Aspartate aminotransferase (AST) \\\u003C= 3x ULN.\n10. The effects of the agents used in this study on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and 4 months after completion of therapy administration. Should a woman become pregnant or suspect pregnancy while participating in this study, the treating physician should be informed immediately.\n11. Adequate neurologic function defined as participants with seizure disorder may be enrolled if seizures are well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug.\n12. Participants must enroll on the Protocol for Children and Young Adults Diagnosed with a Central Nervous System (CNS) Tumor to Assess Cognitive, Quality of Life (QOL), and Comprehensive Effects of Therapies (PNOC COMP) study if PNOC COMP is open to accrual at the enrolling institution\n13. A legal parent\u002Fguardian or participant must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.\n\nExclusion Criteria:\n\n1. Participants who have had chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.\n2. Participants who are receiving any other investigational agents.\n3. Participants must be at least 7 days since the completion of therapy with a biologic or small molecule agent. For any agent with known adverse events that can occur beyond 7 days after administration, the period prior to enrollment must be beyond the time during which adverse events are known to occur. Such participants should also be discussed with study chairs.\n4. Participants who are currently taking any anti-cancer direct therapy. Steroids are not considered anti-cancer therapy.\n5. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.\n6. Women of childbearing potential must not be pregnant or breast-feeding. A negative serum or urine pregnancy test is required prior to start of therapy.\n7. Participants must not receive any tumor-directed therapy after enrollment, except for surgical resection\u002F biopsy.\n\nImportant note: The eligibility criteria listed above are interpreted literally and cannot be waived.","12 Months","39 Years",{"count":261,"type":21},74,[263],"NA","The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in the pilot phase of performing real-time drug screening on tissue taken during surgery in patients with relapsed medulloblastoma or ependymoma and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed medulloblastoma and ependymoma to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer.",[70,136,29,86,266,267],"Ependymoma Malignant","Ependymoma of Brain",[269,270,271],"Individualized Treatment Plan","Genetic Screening","Screening","2026-04-13",{"date":274,"type":47},"2026-04-15",{"date":276,"type":47},"2022-02-22",{"date":278,"type":21},"2028-01-31",{"name":280,"class":54},"University of California, San Francisco",8,{"id":283,"slug":284,"hasResults":11,"nctId":285,"briefTitle":286,"officialTitle":287,"acronym":4,"eligibilityCriteria":288,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":289,"enrollmentInfo":290,"targetDuration":292,"studyType":293,"phases":4,"briefSummary":294,"conditions":295,"keywords":299,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":303,"lastUpdatePostDateStruct":304,"startDateStruct":305,"completionDateStruct":307,"leadSponsor":309,"locationsCount":311},"100611621","relapsed-and-progressive-sonic-hedgehog-medulloblastoma-with-u1-mutation-registry-study-100611621","NCT07242963","Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study","Registry Study of Children and Adults Patients With Relapsed, Refractory, or Progressive Sonic Hedgehog Medulloblastoma Harboring U1 Mutation","Inclusion Criteria:\n\nFor Groups 1 and 2, subjects are eligible to be included in the study only if all of the following criteria are met:\n\n* Age Patients must be ≥ 3 and ≤ 50 years of age at the time of initial diagnosis.\n* Diagnosis Participants must have a diagnosis of SHH medulloblastoma by histologic or molecular criteria at the time of original diagnosis or relapse.\n* Disease status The disease must be recurrent, refractory, or progressive following therapy, including radiotherapy and chemotherapy.\n* Available tumor tissue sample for U1 testing Participants must have available tumor tissue samples to be tested for the U1 mutation.\n\nFor Group 3, biological parent(s) of a subject participating in Group 1 or 2 are eligible.\n\nExclusion Criteria:\n\nSubjects not meeting the inclusion criteria will be excluded.","50 Years",{"count":291,"type":21},300,"5 Years","OBSERVATIONAL","The purpose of this study is to create a biobank for patients diagnosed with Sonic Hedgehog Medulloblastoma at Baylor College of Medicine\u002FTexas Children's Cancer Center. A biobank is a facility that stores and manages biological samples (such as blood, tissue, or DNA) from individuals, along with detailed health information, for use in medical research to study diseases and develop new treatments. The investigators are requesting participants' permission to add their information and samples to this biobank.\n\nBeing in this research study is voluntary; it is the participant's choice. If the participant joins this study, they can still stop at any time.\n\nIf the participant decides to participate, the investigators will review the participant's clinical medical records, demographics, treatment history, family history, and imaging. The investigators will also collect biological samples from the participant and the biological parents' buccal swabs (optional).\n\nThe participation in this biobank will last about 5 years from the decision to participate.\n\nWhy am I being asked to participate? The participant or their child is invited to participate in this study if the participant or their tumor may have a U1 mutation. U1 mutation is associated with an error in the gene that splices the tumor DNA, leading to random splicing that may increase the tumor mutation burden and generate novel tumor neoantigens (targets). Studying the U1 mutation will enable the investigator to design more effective therapies and guide future treatments for patients with relapsed or refractory sonic hedgehog medulloblastoma, thereby improving their outcomes and quality of life. Moreover, the investigators aim to determine whether germline mutations inherited from parents may increase the risk of medulloblastoma in their offspring. The participant will receive no direct benefit from their participation in this study. However, participation in this study may help the investigators better understand SHH Medulloblastomas and benefit other patients in the future.",[29,296,297,298],"Medulloblastoma, Childhood, Recurrent","Medulloblastoma, SHH-activated and TP53 Mutant","Medulloblastoma, SHH-activated and TP53 Wildtype",[300,301,302],"SHH medulloblastoma","medulloblastoma","medulloblastoma Recurrent","2026-04-09",{"date":272,"type":47},{"date":306,"type":47},"2025-09-30",{"date":308,"type":21},"2035-12-30",{"name":310,"class":54},"Mohammad H. Abu Arja",2,{"id":313,"slug":314,"hasResults":11,"nctId":315,"briefTitle":316,"officialTitle":317,"acronym":318,"eligibilityCriteria":319,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":320,"enrollmentInfo":321,"targetDuration":4,"studyType":22,"phases":323,"briefSummary":324,"conditions":325,"keywords":327,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":335,"lastUpdatePostDateStruct":336,"startDateStruct":338,"completionDateStruct":340,"leadSponsor":342,"locationsCount":344},"100160063","phase-2-antiangiogenic-therapy-for-children-with-recurrent-medulloblastoma-ependymoma-atrt-and-rare-cns-tumors-100160063","NCT01356290","Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors","A Phase II Study of Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent\u002FProgressive Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors","MEMMAT","Inclusion criteria for patients Stratum I: Relapsed or progressive medulloblastoma - completed Stratum II: Relapsed or progressive ependymoma (at least one site of untreated recurrent disease) Stratum III: Relapsed or progressive ATRT (at least one site of untreated recurrent disease) Stratum IV: Relapsed or progressive medulloblastoma (at least one site of untreated recurrent disease) Stratum V: Relapsed or progressive CNS tumor of various histologies or patients with exclusion criteria or adult patients (explorative) Histological confirmation at diagnosis or relapse Stratum IV: Confirmation of the medulloblastoma group by methylation; IDAT (Intensity Data; raw data of methylation array) Female or male, aged from 0 to \\\u003C20 years (at time of original diagnosis) Participants must have normal organ and bone marrow function (ALT \\\u003C5x institutional upper limit of normal, creatinine \\\u003C1.5x institutional upper limit of normal for age, WBC \\>1000\u002Fmm3, platelets \\> 20,000\u002Fmm3. Patients with values less than WBC 2000\u002Fmm3 or platelets 50,000\u002Fmm3 will require initiation of treatment with etoposide and cyclophosphamide at a lower starting dose as defined within the protocol Karnofsky performance status ≥50. For infants and children less than 12 years of age, the Lansky play scale ≥50% will be used Written informed consent of patients and \u002F or legal guardian\n\nExclusion criteria for patients VP- or subdural peritoneal shunt dependency (can be included in Stratum V) Prior treatment with temozolomide\u002Firinotecan (can be included in Stratum V) Active infection, pregnancy or breast feeding Treatment for current relapse (surgery may be performed before MEMMAT treatment; patients with sites of disease not irradiated are still eligible for the protocol) Known hypersensitivity to any of the drugs in the protocol Active peptic ulcer Any significant cardiovascular disease not controlled by standard therapy e.g. systemic hypertension Anticipation of the need for major elective surgery during the course of the study treatment Any disease or condition that contraindicates the use of the study medication\u002Ftreatment or places the patient at an unacceptable risk of experiencing treatment-related complications Non-healing surgical wound A bone fracture that has not satisfactorily healed","19 Years",{"count":322,"type":21},232,[66],"Patients with with recurrent or progressive medulloblastoma, ependymoma, atypical teratoid rhabdoid tumor (ATRT), and CNS tumors of various histologies have a very poor prognosis whether treated with conventional chemotherapy, high-dose chemotherapy with stem cell rescue, irradiation or combinations of these modalities.\n\nAntiangiogenesis therapy has emerged as a new treatment option in solid malignancies. The frequent delivery of low doses of chemotherapy, referred to as metronomic or antiangiogenic chemotherapy, targets endothelial cells while reducing the toxicity associated with standard dose chemotherapy.\n\nThe aim of the study is to extend therapy options for children with recurrent or progressive medulloblastoma, ependymoma, ATRT, and CNS tumors of various histologies, for whom no known curative therapy exists, by prolonging survival while maintaining good quality of life.\n\nThe study will be conducted in independent strata. Stratum I (recurrent medulloblastoma): recently completed (Peyrl, 2023). Stratum II (recurrent ependymoma), III (recurrent ATRT) and V (recurrent CNS tumors of various histologies, patients with exclusion criteria and adult patients): The primary objective is to determine the response rate defined as the percentage of patients with complete response (CR), partial response (PR), stable disease (SD) or lack of recurrence at 6 months after start of antiangiogenic treatment. Stratum IV (recurrent medulloblastoma): To determine whether temozolomide, irinotecan, bevacizumab, thalidomide, celecoxib, fenofibrate, etoposide ivt, cytarabine ivt can increase the response rate after 6 months of treatment, compared with etoposid, cyclophosphamide, bevacizumab, thalidomide, celecoxib, fenofibrate, etoposide ivt, cytarabine ivt. Additionally, PFS, OS, toxicity, QoL, performance status, predictive and prognostic markers will be examined.\n\nIn stratum II and III, the study will follow an open label, single arm phase 2 design, and an open label randomized two-arm phase 2 design in Stratum IV, and the exploratory Stratum V.",[29,30,239,326],"Rare CNS Tumor Recurrent",[70,86,328,329,330,331,332,333,334],"ATRT","Relapse","Children","antiangiogenic","metronomic","intraventricular","Rare CNS tumor","2026-02-19",{"date":337,"type":47},"2026-02-23",{"date":339,"type":47},"2014-04",{"date":341,"type":21},"2030-04",{"name":343,"class":54},"Medical University of Vienna",22,{"id":346,"slug":347,"hasResults":11,"nctId":348,"briefTitle":349,"officialTitle":350,"acronym":4,"eligibilityCriteria":351,"healthyVolunteers":11,"sex":16,"minAge":352,"maxAge":152,"enrollmentInfo":353,"targetDuration":4,"studyType":22,"phases":355,"briefSummary":356,"conditions":357,"keywords":366,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":369,"lastUpdatePostDateStruct":370,"startDateStruct":372,"completionDateStruct":374,"leadSponsor":376,"locationsCount":311},"100622970","phase-1-b7-h3cd28zcart-in-cns-neoplasms-100622970","NCT07390539","B7-H3.CD28Z.CART in CNS Neoplasms","A Phase 1\u002F1b Study of Autologous b7-h3 Chimeric Antigen Receptor t Cells (b7-h3.cd28z.Cart) in Children and Young Adults With Recurrent or Progressive Cns Neoplasms Expressing b7-h3 Target","Pre-screening Inclusion Criteria:\n\n* Participants must have histologically and\u002For molecularly confirmed CNS embryonal tumor (see eligible tumor types below), OR ependymoma that is recurrent\u002Fprogressive following standard of care treatment.\n\n  --Eligible CNS embryonal tumor types include:\n  * Medulloblastoma\n  * Atypical Teratoid Rhabdoid Tumor (ATRT)\n  * Embryonal Tumor with Multilayered Rosettes (ETMR) Pineoblastoma\n  * Other CNS embryonal tumor types, at the discretion of the study chair (or designee)\n* Participants must have adequate pre-trial tumor material available to determine B7- H3 expression status. Tumor tissue from the most recent resection or biopsy of recurrent disease is preferred. If unavailable, tumor tissue from prior recurrences or from time of initial diagnosis is acceptable. Biopsies will not be performed for participation in this research trial or for research purposes.\n* Pre-screening IHC Consent: All participants ≥ 18 years of age must be able to give informed consent. For participants \\\u003C18 years of age, their legal authorized representative (LAR) (i.e. parent or guardian) must give informed consent. Pediatric participants will be included in age-appropriate discussion and written assent will be obtained for those ≥10 years of age, where appropriate. If a minor becomes of age during participation of this study, they will be asked to reconsent as an adult.\n\nInclusion Criteria:\n\n* Participants must have histologically and\u002For molecularly confirmed CNS embryonal tumor (see eligible tumor types below), OR ependymoma that is recurrent\u002Fprogressive following standard of care treatment.\n\n  --Eligible CNS embryonal tumor types include:\n  * Medulloblastoma\n  * Atypical Teratoid Rhabdoid Tumor (ATRT)\n  * Embryonal Tumor with Multilayered Rosettes (ETMR)\n  * Pineoblastoma\n  * Other CNS embryonal tumor types, at the discretion of the study chair (or designee)\n* B7-H3 expression: Demonstration of B7-H3 expression with H score greater than 100 by immunohistochemistry (IHC) is required.\n* Age: greater than or equal to two (2) years of age and less than or equal to 21 years of age. The first participant treated at each dose level within each stratum (Standard Risk and High Risk) will be ≥ 6 years of age when feasible.\n* Disease status: Participants must have evaluable disease in the central nervous system to be eligible. Evaluable disease includes either measurable OR non-measurable disease, defined as follows:\n\n  --Measurable disease (contrast-enhancing or non-enhancing tumor)\n  * Clearly defined lesional margins with two perpendicular diameters of at least 10mm, OR\n  * At least two times (in both perpendicular diameters) the MRI slice thickness, plus the interslice gap\n\n    --Non-measurable disease (tumor that is too small to be accurately measured)\n  * Lesion that is measurable in only one perpendicular dimension, OR\n  * Lesion that is less than 10mm in at least one perpendicular dimension, OR\n  * Lesion that is less than two times the MRI slice thickness, plus the interslice gap\n  * Note: Leptomeningeal (LM) disease is considered non-measurable but evaluable.\n* Performance status: Karnofsky performance status ≥60% for participants ≥16 years of age and Lansky performance status ≥60% for participants \\\u003C16 years of age (see APPENDIX A PERFORMANCE STATUS CRITERIA). NOTE: Participants with neurologic deficits must have a stable neurologic exam for seven (7) days prior to enrollment. Participants 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* Life expectancy of greater than 12 weeks\n* Prior therapy: Participants must have received prior standard of care therapy, including maximal safe surgical resection, radiation therapy and\u002For standard chemotherapy, and is recovered from all acute treatment-related toxicities (defined as ≤ Grade 1 or stable) from all prior therapy before entering this study There is no upper limit to the number of prior therapies allowed, but must have received all standard curative options for their tumor type.\n* Participants must meet the following washouts prior to enrollment:\n\n  * Radiation therapy - Participants must have had their last fraction of:\n\n    ---Craniospinal irradiation, whole brain radiation therapy, or radiation therapy to \\>50% of the pelvis or spine \\>28 days prior to enrollment\n\n    ---Focal irradiation (small port) \\>14 days prior to enrollment\n  * At least 14 days since any prior cytotoxic chemotherapy\n  * At least 7 days since any biologic antineoplastics, tyrosine kinase inhibitor, targeted agent\n  * At least 21 days or 5 half-lives (whichever is shorter) since any investigational antineoplastic or disease-directed agent (but at least 28 days from prior investigational antineoplastic vaccine therapy)\n  * At least 21 days since any monoclonal antibody therapy\n  * At least 90 days since any systemic inhibitor\u002Fstimulatory immune checkpoint therapy\n  * At least 28 days from prior autologous stem cell transplantation, with no ongoing toxicities\n  * At least 14 days after peg-filgrastim and 7 days for hematopoietic growth factor support\n* Steroid use: Must not require concurrent systemic steroid therapy, although physiologic corticosteroid replacement therapy for management of pituitary\u002Fadrenal insufficiency and\u002For topical administration (e.g. inhaled or dermatologic) is allowed. Use of topical, ocular, intranasal, or inhaled corticosteroids are permitted per PI\n\ndiscretion.\n\n* Participants must have adequate organ function, as defined below\n\n  --Adequate bone marrow function\n  * Hemoglobin ≥ 8 g\u002FdL\n  * Absolute neutrophil count (ANC) ≥ 1000 cells\u002FuL\n  * Absolute lymphocyte count (ALC) ≥ 150 cells\u002FuL\n  * Platelets ≥100,000\u002FuL (unsupported, defined as no platelet transfusion within 4 days)\n* Adequate renal function defined as creatinine within normal limits for age OR creatinine clearance (as estimated by Cockcroft Gault Equation for participants ≥ 18yo and Bedside Schwartz for participants \\\u003C18yo) ≥70mL\u002Fmin\n\n  * Maximum Serum Creatinine mg\u002FDL ---6 months to 1 year Male 0.5 Female 0.5 ---1 to \\\u003C 2 years Male 0.6 Female 0.6 ---2 to \\\u003C 6 years Male 0.8 Female 0.8\n\n    * 6 to \\\u003C 10 years Male 1 Female 1\n    * 10 to \\\u003C 13 years Male 1.2 Female 1.2\n    * 13 years to \\\u003C 16 years Male 1.5 Female 1.4\n\n      * 16 years Male 1.7 Female 1.4\n* Adequate hepatic function\n\n  * Serum ALT\u002FAST ≤3.0 upper limit of normal (ULN)\n  * Total bilirubin ≤1.5mg\u002FdL, except in subjects with confirmed Gilbert's syndrome\n* Adequate cardiac function\n\n  --Ejection fraction ≥50% or fractional shortening ≥28%, measured by echocardiography\n* Adequate pulmonary function\n\n  * No evidence of dyspnea at rest\n  * Pulse oximetry \\>92% whilst breathing room air\n* Adequate neurologic function\n\n  * Participants with seizure disorders on anticonvulsants may be enrolled if seizures are well controlled (no seizure activity within 7 days prior to enrollment)\n  * Nervous system disorders (CTCAE v6.0) resulting from prior therapy must be ≤ Grade 2, with the exception of decreased tendon reflex (DTR; any Grade eligible). Participants with neurological deficits should be stable for a minimum of 7 days prior to enrollment. (A baseline detailed neurological exam should clearly document the neurological status of the participant prior at enrollment).\n* Females of childbearing potential must have a negative serum or urine pregnancy test (females who have undergone surgical sterilization are not considered to be of childbearing potential)\n* Participants of childbearing or child-fathering potential must be willing to practice birth control from the time of enrollment on this study and for one year after receiving the preparative lymphodepletion regimen, or for as long as B7- H3.CD28Z.CART cells are detectable in peripheral blood or CSF, whichever is later.\n* Participant or parent of participant or legally recognized representative must be able to sign a written informed consent document. Pediatric participants will be included in age-appropriate discussion and written assent will be obtained for those ≥10 years of age, where appropriate.\n\nExclusion Criteria:\n\n* Participants with bulky tumor are ineligible. Bulky tumor is defined as:\n\n  * Tumor with diameter of \\>5cm in one dimension on T2\u002FFLAIR sequence\n  * Tumor with evidence of clinically significant midline shift or uncal herniation\n  * Tumor that, in opinion of the site investigator, shows significant mass effect in either the brain or spine\n* Participants with clinical or radiological evidence of brain herniation.\n* Participants who have received other B7-H3 targeted cellular therapies. Other prior cellular therapies are eligible, including immune checkpoint inhibition and vaccine therapy. These prior therapies should be discussed with the study chair (or designee) prior to participant enrollment.\n* Concurrent illness\n\n  * Participants with any prior immunodeficiency or history of autoimmune disease requiring systemic steroids\u002F immunosuppressive medication\u002F disease-modifying agents within the last two (2) years.\n  * Uncontrolled (Grade 3) bacterial, viral, fungal, or other infection.\n  * Ongoing infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive). A history of hepatitis B or hepatitis C is permitted if the viral load is undetectable per quantitative PCR and\u002For nucleic acid testing.\n  * Evidence of severe or uncontrolled systemic disease (e.g. Grade 3 significant cardiac, pulmonary, hepatic, renal or other organ dysfunction) that in the judgement of the principal investigator is likely to interfere with assessment of safety or efficacy of the investigational regimen and its requirements.\n  * Known sensitivity or allergy to any of the agents\u002Freagents used in this study (i.e. DSMO, cyclophosphamide, fludarabine)\n  * History of severe hypersensitivity reaction to compounds of similar chemical or biologic compositions to any agent used in the study or in the manufacturing of cells.\n* Concomitant medications\n\n  * Current systemic corticosteroid therapy\n  * Note, physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency will be allowed.\n  * Participants who are receiving any other anti-cancer or investigational drug therapy are ineligible.\n  * Participants who have received the last vaccination of a live vaccine ≤ 30 days prior to the start of treatment are ineligible.\n  * Ongoing use of dietary supplements, alternative therapies or extreme diets, or any medication not approved by the study chair (or designee).\n* Any other condition which in the principal investigator's opinion makes the individual clinically unsuitable to participate in this trial, or which would jeopardize compliance with the protocol, or would make it difficult to interpret adverse events or study data.","2 Years",{"count":354,"type":21},70,[24],"The purpose of this research study is to test the safety and effectiveness of a cell therapy at different doses for children and young adults with recurrent or progressive brain tumors. Recurrent\u002Frecurred means a tumor that has gone away and then came back. This cell therapy is called B7- H3.CD28Z.CART, referred to as B7-H3 CAR T cells. B7-H3 is a protein that is over-expressed on many tumor cells, making it a good target for cancer cell therapy.\n\nThe names of the study investigational therapies involved in this study are:\n\n* Fludarabine (a type of chemotherapy)\n* Cyclophosphamide (a type of chemotherapy)\n* B7-H3 CAR T cells (a type of cellular therapy)",[358,359,191,360,361,70,136,362,29,86,363,364,240,365],"Central Nervous System Neoplasms","Brain Tumor","Brain Tumor, Pediatric","Brain Tumor Adult","Medulloblastoma, Adult","Atypical Teratoid\u002FRhabdoid Tumor","Embryonal Tumor With Multilayered Rosettes","Leptomeningeal Disease",[358,359,360,367,32,70,136,362,29,86,363,368,240,365],"Brain Tumor, Adult","Embryonal Tumor with Multilayered Rosettes","2026-01-28",{"date":371,"type":47},"2026-02-05",{"date":373,"type":21},"2026-07",{"date":375,"type":21},"2032-08-31",{"name":377,"class":54},"Robbie Majzner"]