[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pediatric-solid-tumor\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pediatric-solid-tumor":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,8,0,[8,52,84,109,136,164,196,221],{"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":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100379579","phase-1-metarrestin-ml-246-in-subjects-with-metastatic-solid-tumors-100379579",false,"NCT04222413","Metarrestin (ML-246) in Subjects With Metastatic Solid Tumors","First-in-Human Phase I Trial to Investigate the Safety, Tolerability, Pharmacokinetics, Biological and Clinical Activity of Metarrestin (ML-246) in Subjects With Metastatic Solid Tumors","* INCLUSION CRITERIA:\n* Adult (\\>= 18 years) subjects with:\n\n  * histologically or cytologically confirmed solid tumors (Phase IA).\n\nOR\n\n--histologically or cytologically confirmed pancreatic, colorectal, or breast cancer (Phase IB)\n\nOR\n\n* Pediatric (\\>=12 and \\\u003C 18 years) subjects with histologically or cytologically confirmed solid tumors other than rhabdomyosarcoma (RMS) including embryonal, alveolar, spindle cell\u002Fsclerosing and pleomorphic subtypes of RMS (Phase IB).\n* Subjects must have disease that:\n\n  * is not amenable to potentially curative resection,\n  * spread at least to one other organ system other than primary tumor or recurred after removal of primary tumor\n  * has site measurable per RECIST 1.1\n  * progressed on or after at least one line of standard systemic chemotherapy (Phase IA and IB1)\n  * have no standard therapy option available (Phase IB2)\n* Patients must have recovered from any acute toxicity related to prior therapy or surgery or disease to a grade 1 or less.\n* Performance status\n\n  --Karnofsky \\>= 70% (for patients \\>= 16 years old), Lansky \\>= 70% (for patients \\\u003C16 years old)\n* Adequate hematological function defined by:\n\n  * absolute neutrophil count (ANC) \\>= 1.0 x 10(9)\u002FL,\n  * transfusion-independent platelet count \\>= 100 x 10(9)\u002FL,\n  * Hgb \\>= 9 g\u002F dL (patients who have received \\\u003C= 2 PRBC transfusions within 48 hours are eligible)\n* Adequate coagulation as defined by:\n\n  --INR\\\u003C1.5 (or \\\u003C 3.0 if subjects are currently taking anticoagulated medications) Note: increase of the upper limit of INR is restricted only to subjects who are receiving anticoagulation for medical reasons (DVT\u002FPE prophylaxis, treatment for a thromboembolic event) and have increased INR because of these medications. Patients who have an elevated INR due to compromised liver function or any other medical conditions remain excluded\n* Adequate hepatic function defined by:\n\n  * a total bilirubin level \\\u003C= 1.5 x ULN, (total bilirubin \\\u003C= 2.0 x ULN in case of prior diagnosis of Gilbert syndrome)\n  * an AST level \\\u003C= 3xULN\n  * an ALT level \\\u003C= 3 xULN\n* Adequate renal function defined by:\n\n  * Creatinine OR Measured or calculated creatinine clearance (CrCl) (eGFR may also be used in place of CrCl)\n\n    ---\\\u003C 1.5x institution upper limit of normal OR\n\n    ---\\>= 45 mL\u002Fmin\u002F1.73 m\\^2 for participant with creatinine levels \\>= 1.5 X institutional ULN\n  * Creatinine clearance (CrCl) or eGFR should be calculated per institutional standard.\n* The effects of the study treatment on the developing human fetus are unknown; thus, individuals of childbearing potential and individuals who can father children must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior\n\nto study entry, for the duration of study therapy and up to 120 days after the last dose of the study drug.\n\n* Nursing participants must be willing to discontinue nursing at the time of the study treatment initiation.\n* Weight \\>= 35 kg.\n* Ability of subject or parent\u002Fguardian to understand and the willingness to sign a written informed consent document.\n* Subjects must have lesion(s) accessible for biopsy (other than used for measurement of disease) and be willing to undergo mandatory study biopsies (Cohort IB1 only).\n* Ability to swallow oral capsules.\n\nEXCLUSION CRITERIA:\n\n* Anticancer treatment within designated period before treatment initiation including:\n\n  * minor surgical procedure (such as biliary stenting) within 14 days. Note: if liver function tests after biliary stenting or renal function tests after ureteral stenting return to normal, within 5 days after biliary or ureteral stenting;\n  * major surgical procedure or curative radiation treatment within 28 days;\n  * palliative radiation treatment within 14 days;\n  * chemotherapy or experimental drug treatment with published half-life known to be 72 hours or less within 14 days;\n  * experimental drug treatment with unpublished or half-life greater than 72 hours within 28 days;\n  * chemotherapy regimen containing an alkylating antineoplastic agent (cyclophosphamide, chlorambucil, melphalan, or ifosfamide), alkylating-like (platinumbased chemotherapeutic drugs, platinum analogues), and non-classical alkylating agent (dacarbazine, temozolomide) within 28 days.\n* Patients receiving any medications or substances that are moderate and strong inhibitors or inducers of CYP3A4 and are not able to safely stop these medications are excluded from this study; patients must stop strong CYP3A4 inhibiting\u002Finducing medications within 5 published half-lives and moderate within 3 published half-lives prior to the treatment initiation.\n\nNote: dihydropyridine calcium - channel blockers are permitted for management of underling disease\n\n* Subjects with cardiomyopathy diagnosed within 6 months prior to treatment initiation including but not limited to the following:\n\n  * hypertrophic cardiomyopathy\n  * arrhythmogenic right ventricular cardiomyopathy\n  * abnormal ejection fraction (echocardiogram \\[ECHO\\]) \\\u003C= 53% (if a range is given then the upper value of the range will be used)\n  * previous moderate or severe impairment of left ventricular systolic function (LVEF \\\u003C45%)\n  * severe valvular heart disease\n  * atrial fibrillation with a ventricular rate \\>100 bpm on EKG at rest\n  * Fridericia's corrected QT interval (QTcF) \\>= 480 msec (adults) or \\>= 460 msec (pediatric subjects, aged 12 to \\\u003C18 years) or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome.\n* HIV, HCV, HBV positive patients on antiviral drugs are excluded due to the absence of previous experience with concurrent use of antiviral medications and the investigational drug product to be evaluated in the current study and possible for adverse pharmacokinetic and\u002For pharmacodynamic interactions.\n* Previous malignant disease (other than the target malignancy to be investigated in this trial) within the last 3 years. Note: subjects with a history of cervical carcinoma in situ, superficial or non-invasive bladder cancer, or basal cell or squamous cell carcinoma in situ previously treated with curative intent are NOT excluded.\n* Rapidly progressive disease which, in the opinion of the Investigator, may predispose to inability to tolerate treatment or trial procedures.\n* Subjects with central nervous system (CNS) metastases or CNS disorders known to increase possible neurotoxicity of metarrestin in case of compromised blood-brain barrier (e.g. recent stroke (\\\u003C3 months of treatment initiation), infectious causes).\n* Significant acute or chronic infections including tuberculosis with presence of clinical symptoms or physical findings.\n* Patients with a history of any seizures or increased risk of seizures on screening EEGs defined by 1) interictal epileptiform discharges, 2) temporal intermittent rhythmic delta activity (TIRDA), or 3) electrographic or clinical seizures on EEG.\n* Clinically relevant diseases (for example, inflammatory bowel disease) and \u002F or uncontrolled medical conditions, which, in the opinion of the Investigator, might impair the subject's tolerance or ability to participate in the trial.\n* Patients with previous gastric bypass, patients receiving nutrition via feeding tubes or parenterally, or patients with malabsorptive conditions (damage to the intestine from infection, inflammation, trauma, or surgery, celiac disease, Crohn's disease, chronic pancreatitis, or cystic fibrosis resulting malabsorption). Patients with refractory nausea and vomiting. Note: patients with gastric banding are allowed.\n* Pregnant individuals.","ALL","12 Years","120 Years",{"count":20,"type":21},116,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","Background:\n\nMetastasis is the spread of cancer from one organ to a nonadjacent organ. It causes 90% of cancer deaths. No treatment specifically prevents or reduces metastasis. Researchers hope a new drug can help. It stops cancer cells from growing and spreading further and possibly shrink cancer lesions in distant organs.\n\nObjective:\n\nTo find a safe dose of metarrestin and to see if this dose shrinks tumors.\n\nEligibility:\n\nAdults age 18 and older with pancreatic cancer, breast cancer, or a solid tumor that has not been cured by standard therapies. Also, children age 12-17 with a solid tumor (other than a muscle tumor) with no standard therapy options.\n\nDesign:\n\nParticipants will be screened with:\n\n* blood tests\n* physical exam\n* documentation of disease confirmation or tumor biopsy\n* electrocardiogram to evaluate the heart\n* review of their medicines and their ability to do their normal activities\n\nParticipants will take metarrestin by mouth until they cannot tolerate it or stop to benefit from it. They will keep a medicine diary.\n\nParticipants will visit the Clinical Center. During the first month there are two brief hospital stays required with visits weekly or every other week thereafter. They will repeat some of the screening tests. They will fill out questionnaires. They will have tests of their cognitive function. They will have an electroencephalogram to record brain activity. They will have a computed tomography (CT) scan or magnetic resonance imaging (MRI). A CT is a series of X-rays of the body. An MRI uses magnets and radio waves to take pictures of the body.\n\nAdult participants may have tumor biopsies.\n\nParticipants will have a follow-up visit 30 days after treatment ends. Then they will have follow-up phone calls or emails every 6 months for the rest of their life or until the study ends.\n\n...",[27,28,29,30,31,32],"Advanced Solid Tumors","Metastatic Pancreatic Cancer","Pediatric Solid Tumor","Advanced Breast Cancer","Malignant Peripheral Nerve Sheath Tumor","Colorectal Neoplasms",[34,35,36,37,38],"First-In-Class Investigational Agent","Peri-Nucleolar Compartment (PNC)","effective therapies against metastasis","Oral Administration","Maximum Recommended Starting Dose","RECRUITING","2026-07-01",{"date":42,"type":43},"2026-07-02","ACTUAL",{"date":45,"type":43},"2020-10-27",{"date":47,"type":21},"2028-12-31",{"name":49,"class":50},"National Cancer Institute (NCI)","NIH",2,{"id":53,"slug":54,"hasResults":11,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":4,"eligibilityCriteria":58,"healthyVolunteers":59,"sex":16,"minAge":60,"maxAge":4,"enrollmentInfo":61,"targetDuration":4,"studyType":63,"phases":4,"briefSummary":64,"conditions":65,"keywords":70,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":83},"100342530","natural-history-and-biospecimen-acquisition-for-children-and-adults-with-rare-solid-tumors-100342530","NCT03739827","Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors","Natural History and Biospecimen Acquisition Study for Children and Adults With Rare Solid Tumors","* INCLUSION CRITERIA:\n* Cohort 1: Participants with a diagnosis of a rare solid tumor (fewer than 15 cases in 100,000 people per year). There are no age restrictions beyond the neonatal period (4 weeks).\n\nOR\n\n-Cohort 2: Participants without a rare tumor who have a germline genetic variant that predisposes to a rare solid tumor\n\nOR\n\n-Cohort 3: Relatives of participants with diagnosis of rare solid tumors who do NOT have a known germline variant that predisposes to a rare solid tumor\n\nOR\n\n* Cohort 4: Parent\u002Fguardian of child participating in a focus group if not already enrolled on the study.\n* Ability of subject or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.\n\nEXCLUSION CRITERIA:\n\nNone",true,"4 Weeks",{"count":62,"type":21},10000,"OBSERVATIONAL","Background:\n\nApproximately 150 cases of cancer per one million per year are considered rare cancers. While all tumors originate from genetic changes, a small percentage of these tumors are familial. Researchers want to study these changes in biological samples from people with rare tumors in order to learn more about how these tumors develop. The information obtained from this study may lead to improved screening, preventive guidelines, and treatments.\n\nObjective:\n\nTo better understand rare cancers and hereditary cancer syndromes.\n\nEligibility:\n\nPeople who have a rare tumor, a family history of a rare tumor, a hereditary cancer syndrome, or a mutation that leads to rare tumors.\n\nDesign:\n\nParticipants will be screened with questions about their medical history and\u002For that of their family members. They will give a saliva sample.\n\nParticipants who have a tumor will have their medical records and tests reviewed. They will answer questions about their wellbeing and needs. They may provide a tumor tissue sample.\n\nParticipants may also have:\n\n* Physical exam\n* Clinical photography\n* Blood, urine, saliva, and stool samples taken\n* Consultation with specialists\n* A scan that produces a picture of the body. Either one that uses a small amount of radiation, or one that uses a magnetic field.\n* Genetic testing\u002Fgenetic counseling.\n\nParticipants will be contacted once a year. They will answer updated questions about their medical and family history.\n\nParticipants will be asked to contact the study team if there are changes in their tumors.\n\nParticipants may be invited to join focus groups for people with the same diagnosis of rare tumors.\n\nParticipants may be invited to participate in other NIH protocols.\n\n\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\n\n\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\n\nRARE TUMOR LIST:\n\n1. Acinar cell carcinoma of the pancreas\n2. Adamantinoma\n3. Adenosqaumous carcinoma of the pancreas\n4. Adrenocortical carcinoma\n5. Alveolar soft part sarcoma\n6. Anaplastic Thyroid Cancer\n7. Angiosarcoma\n8. Atypical Teratoid Rhabdoid Tumor\u002FMRT\n9. Carcinoid\n10. Carcinoma of Unknown Primary\n11. Chondrosarcoma\n12. Chondromyxoid fibroma\n13. Chordoma\n14. Clear cell renal carcinoma\n15. Clear Cell Sarcoma\n16. Clear cell sarcoma of kidney\n17. Conventional chordoma\n18. Dedifferentiated chordoma\n19. Desmoid\n20. Desmoplastic small round cell tumor\n21. Epithelioid hemangioendothelioma\n22. Esthenioneuroblastoma\n23. Ewing Sarcoma\n24. Fibrolamellar carcinoma\n25. Fusion negative rhabdomyosarcoma\n26. Fusion positive renal cell carcinoma\n27. Fusion positive rhabdomyosarcoma\n28. Gastro-enteropancreatic neuroendocrine tumor\n29. Hepatoblastoma\n30. Hereditary Diffuse Gastric Cancer\n31. Inflammatory myofibroblastic tumor\n32. Kaposiform hemangioendothelioma\n33. Malignant ectomesenchymal tumor\n34. Malignant peripheral nerve sheath tumor\n35. Malignant triton tumor\n36. Medullary thyroid cancer\n37. Mixed acinar adenocarcinoma\n38. Mixed acinar neuroendocrine carcinoma\n39. Myxoid Liposarcoma\n40. Neuroblastoma\n41. Neuroendocrine tumors\n42. NUT midline carcinoma\n43. Osteosarcoma\n44. Pancreas ductal adenocarcinoma with squamous features\n45. Pancreatic acinar cell carcinoma\n46. Papillary renal cell carcinoma\n47. Paraganglioma\n48. Parosteal Osteosarcoma\n49. Periosteal Osteosarcoma\n50. Peripheral nerve sheath tumor\n51. Peripheral primitive neuroectodermal tumor\n52. Pheochromocytoma\n53. Pituitary cancer\n54. Poorly differentiated chordoma\n55. Renal medullary carcinoma\n56. Rhabdomyosarcoma\n57. Round cell Liposarcoma\n58. Schwannoma\n59. Sclerosing Epithelioid Fibrosarcoma\n60. SDH deficient GIST\n61. SMARCB1 deficient tumors\n62. SMARCA4 deficient tumors\n63. Synovial sarcoma\n64. Undifferentiated Sarcoma\n\n    \\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\n\n    \\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*\\*",[66,67,29,68,69],"Malignant Solid Tumors","Other Neoplasms Solid Tumors","Refractory Solid Tumors","Solid Tumor",[71,72,73,74],"Service to the Patients","Knowledge about the Nature of Cancer","Cancer Evaluations","Natural History","2026-06-23",{"date":77,"type":43},"2026-06-24",{"date":79,"type":43},"2019-01-28",{"date":81,"type":21},"2032-03-31",{"name":49,"class":50},3,{"id":85,"slug":86,"hasResults":11,"nctId":87,"briefTitle":88,"officialTitle":88,"acronym":4,"eligibilityCriteria":89,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":91,"enrollmentInfo":92,"targetDuration":4,"studyType":22,"phases":94,"briefSummary":95,"conditions":96,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":108},"100479361","phase-1-targeting-pediatric-brain-tumors-and-relapsedrefractory-solid-tumors-with-sodium-glucose-cotransporter-2-inhibitors-sglt2i-100479361","NCT05521984","Targeting Pediatric Brain Tumors and Relapsed\u002FRefractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)","Inclusion Criteria:\n\n* Diagnosis of a recurrent primary brain tumor with no curative therapy available OR diagnosis of relapsed\u002Frefractory solid tumor with no curative option and has trialed past a second line of therapy.\n* Measurable disease per the following:\n\n  * For patients with brain tumors: measurable disease pediatric Response Assessment in Neuro-Oncology Criteria (RANO) criteria\n  * For patients with solid tumors: measurable disease using response evaluation criteria in solid tumors (RECIST 1.1). Includes patients with diagnoses of relapsed or refractory sarcomas, neuroblastoma, and Wilms tumor. Other rare solid tumors can be discussed with study chair.\n* Life expectancy \\> 12 weeks.\n* Prior treatment with radiation alone, chemotherapy alone or combined radiation and chemotherapy is allowed.\n* Patient is between 6 and 29 years old (inclusive)\n* Patient is capable of swallowing whole pills\n* Normal bone marrow and organ function as defined below:\n\n  * Leukocytes ≥ 3,000\u002FmcL\n  * Absolute neutrophil count ≥ 1,500\u002Fmcl\n  * Platelets ≥ 100,000\u002Fmcl\n  * Total bilirubin ≤ 1.5 x IULN\n  * AST(SGOT)\u002FALT(SGPT) ≤ 3.0 x IULN\n  * Creatinine ≤ IULN OR creatinine clearance ≥ 60 mL\u002Fmin\u002F1.73 m\\^2 for patients with creatinine levels above institutional normal\n  * Normal room air oxygenation must be documented. If room air oxygen saturation is less than 97%, a diffusion capacity of carbon monoxide (DLCO) of greater than 80%, must be demonstrated.\n* Karnofsky or Lansky performance score of ≥ 60\n* Patients of childbearing potential and their partners must agree to use two forms of acceptable contraception (including one barrier method) prior to study entry and for the duration of study participation. Should a female patient or partner of a male patient become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.\n* Ability to understand and willingness to sign an IRB approved written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants. All patients and\u002For their parents or legal guardians must sign an IRB approved written informed consent document.\n\nExclusion Criteria:\n\n* Current or previous treatment with SGLT2i or thiazolidinedione.\n* Current use of high dose dexamethasone (exceeding 4 mg\u002Fday). Seven days prior to start of dapagliflozin, patients receiving dexamethasone must be on a stable or decreasing dose (≤ 0.1 mg\u002Fkg\u002Fday or maximum 4 mg\u002Fday). Note that it is preferred that patients not be on dexamethasone during the study.\n* A history of other malignancy with the exceptions of malignancies for which all treatment was completed at least 2 years before registration with no evidence of disease and locally treated skin squamous or basal cell carcinoma.\n* Type 1 diabetes or current insulin treatment.\n* History of stroke or transient ischemic attack (in the last 5 years).\n* HbA1c \\> 8.5%. The rationale is that this is the level that would require addition of insulin. However, insulin use is excluded in this study due to the increased risk of ketoacidosis.\n* Currently receiving any other investigational agents.\n* A history of allergic reactions attributed to compounds of similar chemical or biologic composition to dapagliflozin or other agents used in the study.\n* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, peripheral arterial disease, ketoacidosis, severe kidney disease (estimated glomerular filtration rate eGFR \\\u003C 30 mL\u002Fmin\u002F1.73m\\^2), symptomatic hypotension, and chronic\u002Ffrequent urinary tract infections or yeast infections.\n* Pregnant and\u002For breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days prior to first dose of dapagliflozin.\n* Patients with HIV are eligible unless their CD4+ T-cell counts are \\\u003C 350 cells\u002FmcL or they have a history of AIDS-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective ART according to DHHS treatment guidelines is recommended.","6 Years","21 Years",{"count":93,"type":21},20,[24],"This is a pilot phase Ib study of the safety of dapagliflozin (in addition to standard of care treatment) for the treatment of pediatric patients with recurrent brain tumors and relapsed\u002Frefractory solid tumors. The primary hypothesis is that dapagliflozin is well-tolerated and safe to use in this patient population. The investigators also hypothesize that dapagliflozin will be efficacious as an adjunct to front-line chemotherapy assessed by decreased tumor markers mediated by its pleiotropic metabolic effects.",[97,29],"Pediatric Brain Tumor","2026-06-07",{"date":100,"type":43},"2026-06-10",{"date":102,"type":43},"2023-04-03",{"date":104,"type":21},"2031-06-30",{"name":106,"class":107},"Washington University School of Medicine","OTHER",1,{"id":110,"slug":111,"hasResults":11,"nctId":112,"briefTitle":113,"officialTitle":114,"acronym":115,"eligibilityCriteria":116,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":117,"enrollmentInfo":118,"targetDuration":4,"studyType":63,"phases":4,"briefSummary":120,"conditions":121,"keywords":123,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":127,"lastUpdatePostDateStruct":128,"startDateStruct":130,"completionDateStruct":132,"leadSponsor":134,"locationsCount":108},"100450916","multiomic-approach-to-radioresistance-of-ependymomas-in-children-and-adolescents-100450916","NCT05151718","Multiomic Approach to Radioresistance of Ependymomas in Children and Adolescents","Ependymomics: Multiomic Approach to Radioresistance of Ependymomas in Children and Adolescents","EPENDYMOMICS","Inclusion Criteria:\n\n* children with intracranial ependymoma\n* treated\n* included in PEPPI study, pediaRT or in SIOP II Ependymoma french program\n\nExclusion Criteria:\n\n* NA","25 Years",{"count":119,"type":21},370,"Treatment of childhood ependymoma, the second most frequent pediatric brain tumor, is based on surgery and radiation therapy. However, 50% relapse, mainly locally. Progress in imaging, molecular biology and radiotherapy ballistics has led us to propose the EPENDYMOMICS project, a multi-omics approach using artificial intelligence to detect the predictive characteristics of relapse, and to define innovative radiotherapy targets using multimodal imaging. We previously reported that the relapse sites are mainly located in the high-dose radiotherapy zone and that there appear to be prognostic factors for relapse based on anatomical and functional MRI abnormalities by diffusion and perfusion. In addition, recent studies in molecular biology have identified significant prognostic factors. The challenge now is to use and correlate all these findings in larger cohorts to tackle the radio-resistance of this disease.\n\nOur objective is to collate in a single database called NETSPARE (Network to Structure and Share Pediatric data to Accelerate Research on Ependymoma) the clinical, histological, biological, imaging and radiotherapy data from two consecutive studies that included 370 children and adolescents with ependymoma since 2000 in France. The EPENDYMOMICS project will comprise a clinical research team, three imaging research teams, two histopathology teams, and a biostatistics team working on NETSPARE. Our goal is to obtain a radiogenomic signature of our data, which will be validated with the English external cohort of 200 patients that is currently being analyzed. The perspective is to optimize the indications and volumes of irradiation that could in the future be used in a European translational research trial to tackle radioresistance.",[122,29],"Ependymoma of Brain",[124,125,126],"childhood ependymoma","radio-resistance","radiogenomic signature","2026-06-02",{"date":129,"type":43},"2026-06-04",{"date":131,"type":43},"2021-09-30",{"date":133,"type":21},"2026-09-30",{"name":135,"class":107},"Institut Claudius Regaud",{"id":137,"slug":138,"hasResults":11,"nctId":139,"briefTitle":140,"officialTitle":141,"acronym":4,"eligibilityCriteria":142,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":143,"enrollmentInfo":144,"targetDuration":4,"studyType":22,"phases":146,"briefSummary":148,"conditions":149,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":155,"lastUpdatePostDateStruct":156,"startDateStruct":158,"completionDateStruct":160,"leadSponsor":162,"locationsCount":51},"100475241","phase-1-safety-and-efficacy-of-cyclophosphamide-sorafenib-bevacizumab-and-atezolizumab-in-pediatric-solid-tumor-patients-100475241","NCT05468359","Safety and Efficacy of Cyclophosphamide, Sorafenib, Bevacizumab, and Atezolizumab in Pediatric Solid Tumor Patients","ANGIO-A: Safety and Tolerability of Oral Cyclophosphamide and Sorafenib With Intravenous Bevacizumab With the Addition of Atezolizumab in Pediatric Solid Tumor Patients","Inclusion Criteria:\n\n* Age: Patients must be \\\u003C 30 years at the time of enrollment on study.\n* Willingness to enroll on the St. Jude Molecular Analysis of Solid Tumors (MAST) study.\n* Diagnosis\n* Part 1: Patients with refractory or recurrent (relapsed) solid tumors accessible by biopsy for which there is no standard therapy are eligible.\n* Part 2: Patients with one of the following diagnoses:\n* Biopsy accessible refractory or recurrent (relapsed) hepatocellular carcinoma\n* Biopsy accessible refractory or recurrent (relapsed)or FL-HCC, DSRCT or non-CNS MRT.\n* Performance level: Karnofsky \\> 50 for patients \\> 16 years of age and Lansky \\> 50 for patients \\\u003C 16 years of age (See Appendix III). Note: 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* Disease status: Patients must tumors that are unresectable and have either measurable or evaluable disease that is accessible by biopsy\n* Organ function: Must have adequate organ and bone marrow function as defined by the following parameters:\n* Patients with solid tumor not metastatic to bone marrow:\n\n  * Peripheral absolute neutrophil count (ANC) \\>1,000\u002Fmm3\n  * Platelet count \\> 75,000\u002Fmm3 (no transfusion within 7 days of enrollment)\n  * Hemoglobin \\> 8 g\u002FdL (with or without support)\n* Patients with solid tumor metastatic to bone marrow will be eligible for study but not evaluable for hematologic toxicity. These patients must not be known to be refractory to red cell or platelet transfusions. At least 2 of every cohort of 3 patients must be evaluable for hematologic toxicity. If dose limiting hematologic toxicity is observed at any dose level, all subsequent patients enrolled must be evaluable for hematologic toxicity.\n* Adequate renal function defined as serum creatinine based on age as shown in Table 1, or creatinine clearance or radioisotope GFR 50 ml\u002Fmin\u002F1.73m2 (GFR 40 ml\u002Fmin\u002F1.73m2 if \\\u003C 2 years of age).\n* Adequate hepatic function defined as total bilirubin \\\u003C 5x upper limit of normal (ULN) and AST\u002FALT \\\u003C 3 x ULN for age.\n* Adequate cardiac function defined as shortening fraction \\> 28% OR ejection fraction of ≥ 47% by echocardiogram.\n* Adequate blood clotting defined as PT\u002FPTT \\\u003C 1.2 x ULN without factor replacement products for 7 days\n* Females of childbearing potential and males able to father a child must be willing to practice acceptable methods of birth control to prevent pregnancy during the study and for at least 5 months after last dose of therapy.\n* Patients must have fully recovered from the acute toxic effects of chemotherapy, immunotherapy, surgery, or radiotherapy prior to entering this study:\n* Myelosuppressive chemotherapy: Patient has not received myelosuppressive chemotherapy within 1 weeks of enrollment onto this study (within 2 weeks of estimated therapy start date) (4 weeks if prior nitrosourea).\n* Hematopoietic growth factors: At least 7 days must have elapsed since the completion of therapy with a growth factor. At least 14 days must have elapsed after receiving pegfilgrastim.\n* Biologic (anti-neoplastic agent): At least 7 days must have elapsed since completion of therapy with a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur.\n* Monoclonal antibodies: At least 14 days (at least 21 days from therapy start date) must have elapsed since the completion of therapy with a monoclonal antibody.\n* Radiotherapy: At least 1 week (2 weeks from estimated therapy start date) must have elapsed since any irradiation; at least 5 weeks (at least 6 weeks from estimated therapy start date) must have elapsed since craniospinal RT or substantial bone marrow irradiation.\n* Chemoembolization: at least 21 days (28 days from estimated therapy start date) must have elapsed since the completion of chemoembolization\n* Radioembolization: at least 21 days (28 days from estimated therapy start date) must have elapsed since the completion of radioembolization\n* Cardiac disease or hypertension: Patients must not have a history of myocardial - infarction, severe or unstable angina, or severe peripheral vascular disease. Hypertension must be well controlled on stable doses of medication for at least two weeks.\n* Female participant who is post-monarchal must have a negative urine or serum pregnancy test.\n* Life expectancy of at least 8 weeks\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding.\n* Currently receiving other investigational drugs.\n* Unwilling or unable to comply with the safety monitoring requirements of this protocol.\n* Tumor not safely accessible by biopsy\n* Inability or unwillingness of research participant or legal guardian \u002F representative to give written informed consent.\n* Surgical procedures and serious or non-healing wounds: patients with a documented, chronic non-healing wound, ulcer, or bone fracture or history of a major surgical procedure or significant traumatic injury within 28 days prior to beginning therapy are excluded due to preclinical evidence supporting the potential for delayed wound healing.\n* Minor surgical procedures for minimally invasive biopsies will be allowed. For minor surgeries, the wound must be healed, and 7 days elapsed since surgery. For procedures such as the placement of an indwelling IV catheter, it is recommended that bevacizumab be postponed for at least 24 hours after the procedure.\n* Thrombosis: Patients must not have a deep venous or arterial thrombosis (including pulmonary embolism) within the last three months prior to study entry and must not have a known thrombophilic condition (i.e., protein S, protein C or antithrombin III deficiency, Factor V Leiden, Factor II G20210A mutation, homocysteinemia or antiphospholipid antibody syndrome).","30 Years",{"count":145,"type":21},64,[24,147],"PHASE2","This is a phase I\u002FII study to evaluate the safety of combining intravenous (IV) atezolizumab and bevacizumab every three weeks, with daily oral cyclophosphamide and pharmacokinetic (PK)-guided sorafenib in children and adolescent and young adults (AYA) with relapsed or refractory solid malignancies (Part 1), and then evaluate the response rate of this combination in children, AYA with relapsed or refractory fibrolamellar carcinoma (FLC) and other rare solid malignancies (Part 2).\n\nPrimary Objectives Part 1\n\n* To establish the safety associated with the administration of the combination of cyclophosphamide, PK-guided sorafenib, bevacizumab and atezolizumab in children and AYA with relapsed or refractory solid tumors\n* To determine if sorafenib systemic exposure can be successfully targeted to an AUC between 20 and 55 hr·µg\u002FmL by Day 21 of cycle 1 in 60% of evaluable patients, when given in combination with cyclophosphamide, bevacizumab, and atezolizumab in children and AYA with relapsed or refractory solid tumors\n\nPart 2\n\n* To evaluate the response rate (CR+PR) of the combination of cyclophosphamide, PK-guided sorafenib, bevacizumab and atezolizumab in children and AYA with relapsed or refractory FLC following two cycles of therapy\n* To determine if the use of PK-guided sorafenib dosing to maintain a systemic exposure between 20 and 55 reduces the interpatient pharmacokinetic variability of sorafenib and the incidence of sorafenib- induced skin toxicities in children and AYA with relapsed or refractory FLC and other rare solid tumors\n\nParts 1 \\& 2\n\n* To determine if the combination of cyclophosphamide, PK-guided sorafenib and atezolizumab will result in increased intratumoral T-cell infiltration of CD8+C45RO+ cells between baseline and following two courses of therapy in pediatric children and AYA with relapsed or refractory solid tumors following two cycles of therapy\n* To characterize the pharmacokinetics of atezolizumab in combination with cyclophosphamide, PK-guided sorafenib and bevacizumab in children and AYA with relapsed or refractory solid tumors\n* To assess the feasibility of performing contrast enhanced ultrasound and explore the correlation between quantitative CEUS parameters and clinical response.\n\nSecondary Objectives\n\nPart 1\n\n• To describe the response rate (CR+PR) of the combination of cyclophosphamide, PK-guided sorafenib, bevacizumab and atezolizumab in children and AYA with relapsed or refractory solid tumors following two cycles of therapy\n\nPart 2\n\n• To describe the response rate (CR+PR) of the combination of cyclophosphamide, PK-guided sorafenib, bevacizumab and atezolizumab in children and AYA with relapsed or refractory FLC, HCC, desmoplastic small round cell tumor, malignant rhabdoid tumor, and other rare solid tumors following two cycles of therapy\n\nParts 1\\&2\n\n* To describe the number of children with liver tumors, initially judged unresectable at diagnosis, that can have their primary tumor resected after treatment with oral cyclophosphamide and sorafenib with intravenous bevacizumab and atezolizumab\n* To describe changes in immune cells in the peripheral blood at periodic times before and after treatment with this combination chemoimmunotherapy\n* To describe the PFS, EFS, and OS in patients treated with the combination of cyclophosphamide, PK-guided sorafenib, bevacizumab, and atezolizumab in patients with relapsed or refractory FLC, DSRCT, MRT, HCC and other rare solid tumors",[150,151,152,153,29,154],"Refractory Solid Tumor","Hepatocellular Carcinoma","Malignant Solid Tumor","Pediatric Cancer","Fibrolamellar Carcinoma","2026-05-18",{"date":157,"type":43},"2026-05-19",{"date":159,"type":43},"2022-11-07",{"date":161,"type":21},"2037-06",{"name":163,"class":107},"St. Jude Children's Research Hospital",{"id":165,"slug":166,"hasResults":11,"nctId":167,"briefTitle":168,"officialTitle":168,"acronym":4,"eligibilityCriteria":169,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":91,"enrollmentInfo":170,"targetDuration":4,"studyType":22,"phases":172,"briefSummary":173,"conditions":174,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":155,"lastUpdatePostDateStruct":190,"startDateStruct":191,"completionDateStruct":193,"leadSponsor":195,"locationsCount":108},"100431379","phase-1-b7-h3-specific-chimeric-antigen-receptor-autologous-t-cell-therapy-for-pediatric-patients-with-solid-tumors-3car-100431379","NCT04897321","B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)","Inclusion Criteria:\n\nProcurement and T-cell production eligibility\\*\n\n\\*a previously collected, autologous leukapheresis product can be used for T-cell production\n\n* Age ≤21 years old\n* B7-H3+ solid tumor with measurable disease; B7-H3 expression will be evaluated by standard immunohistochemistry (IHC) using a previously obtained biopsy; a tumor is considered B7-H3 positive with an H-score ≥100\n* Estimated life expectancy of \\>12 weeks\n* Karnofsky or Lansky (age-dependent) performance score ≥50\n* For females of child bearing age:\n* Not pregnant with negative serum pregnancy test within 7 days prior to enrollment\n* Not lactating with intent to breastfeed\n* Meets eligibility criteria to undergo autologous apheresis, or have previously undergone autologous apheresis\n\nExclusion Criteria:\n\n* Known primary immunodeficiency\n* Known HIV positivity\n* Severe intercurrent bacterial, viral or fungal infection (e.g. active hepatitis B or C infection or adenovirus infection)\n* History of hypersensitivity reactions to murine protein-containing products\n* Rapidly progressive disease (in the opinion of the study PIs)\n\nInclusion criteria\n\nTreatment eligibility\n\n* Age ≤21 years old\n* B7-H3+ solid tumor with measurable disease\n* Evidence of relapsed or refractory disease after standard first-line therapy\n* Estimated life expectancy of \\>8 weeks\n* Karnofsky or Lansky (age-dependent) performance score≥50\n* Echocardiogram with a ventricular ejection fraction\n* \\>40%; or shortening fraction ≥25%\n* Adequate renal function defined as creatinine clearance or radioisotope GFR 50 ml\u002Fmin\u002F1.73m2 (GFR 40 ml\u002Fmin\u002F1.73m2 if \\\u003C 2 years of age)\n* Adequate pulmonary function defined as pulse oximetry ≥92% on room air or forced vital capacity (FVC) ≥50% of predicted value\n* Total Bilirubin ≤3 times the upper limit of normal for age, except in subjects with Gilbert's syndrome\n* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤5 times the upper limit of normal for age\n* Hemoglobin≥ 7g\u002FdL (can be transfused)\n* Platelet count \\>50,000\u002FuL (can be transfused)\n* Absolute neutrophil count (ANC) ≥ 1000\u002FuL\n* Has recovered from all NCI CTAE grade III-IV, non-hematologic acute toxicities from prior therapy\n* For females of child bearing age:\n* Not pregnant with negative serum pregnancy test within 7 days prior to enrollment\n* Not lactating with intent to breastfeed\n* If sexually active, agreement to use birth control until 3 months after T-cell infusion. Male partners should use a condom.\n* Available autologous transduced T-cell product that has met GMP release criteria\n* Agreement to participate in long-term follow-up protocol for patients, who have received genetically modified cell products\n\nExclusion criteria\n\n* Known primary immunodeficiency\n* History of HIV infection\n* Severe, uncontrolled intercurrent bacterial, viral or fungal infection\n* History of hypersensitivity reactions to murine protein-containing products\n* Receiving systemic steroid therapy exceeding the equivalent of 0.5 mg\u002Fkg\u002Fday of methylprednisolone, in the 7 days prior to B7-H3-CAR T-cell infusion\n* Receiving systemic therapy in the 14 days prior to CAR T-cell infusion, which will interfere with the activity of the B7-H3-CAR product (in the opinion of the study PIs).\n* Rapidly progressing disease (in the opinion of the study PIs)",{"count":171,"type":21},48,[24],"3CAR is being done to investigate an immunotherapy for patients with solid tumors. It is a Phase I clinical trial evaluating the use of autologous T cells genetically engineered to express B7-H3-CARs for patients ≤ 21 years old, with relapsed\u002Frefractory B7-H3+ solid tumors. This study will evaluate the safety and maximum tolerated dose of B7-H3-CAR T cells.The purpose of this study is to find the maximum (highest) dose of B7-H3-CAR T cells that are safe to give to patients with B7-H3-positive solid tumors.\n\nPrimary objective\n\nTo determine the safety of one intravenous infusion of autologous, B7-H3-CAR T cells in patients (≤ 21 years) with recurrent\u002Frefractory B7-H3+ solid tumors after lymphodepleting chemotherapy\n\nSecondary objective\n\nTo evaluate the antitumor activity of B7-H3-CAR T cells\n\nExploratory objectives\n\n* To evaluate the tumor environment after treatment with B7-H3-CAR T cells\n* To assess the immunophenotype, clonal structure and endogenous repertoire of B7-H3-CAR T cells and unmodified T cells\n* To characterize the cytokine profile in the peripheral blood after treatment with B7-H3-CAR T cells",[29,175,176,177,178,179,180,181,182,183,184,185,186,187,188,189],"Osteosarcoma","Rhabdomyosarcoma","Neuroblastoma","Ewing Sarcoma","Wilms Tumor","Adrenocortical Cancer","Desmoplastic Small Round Cell Tumor","Germ Cell Cancer","Rhabdoid Tumor","Clear Cell Sarcoma","Hepatoblastoma","Melanoma","Carcinoma","Malignant Peripheral Nerve Sheath Tumors","Soft Tissue Sarcoma",{"date":157,"type":43},{"date":192,"type":43},"2022-07-06",{"date":194,"type":21},"2028-03-01",{"name":163,"class":107},{"id":197,"slug":198,"hasResults":11,"nctId":199,"briefTitle":200,"officialTitle":201,"acronym":4,"eligibilityCriteria":202,"healthyVolunteers":11,"sex":16,"minAge":203,"maxAge":143,"enrollmentInfo":204,"targetDuration":4,"studyType":22,"phases":206,"briefSummary":207,"conditions":208,"keywords":209,"overallStatus":211,"whyStopped":4,"lastUpdateSubmitDate":212,"lastUpdatePostDateStruct":213,"startDateStruct":215,"completionDateStruct":217,"leadSponsor":219,"locationsCount":108},"100620487","phase-1-b7-h3cd28zcart-in-solid-tumors-100620487","NCT07358260","B7-H3.CD28Z.CART in Solid Tumors","A Phase I, Open Label, Dose Escalation, Single Center Study of Autologous B7-H3.CD28Z.CART Cells in Children and Young Adults With Relapsed or Refractory Solid Tumors Expressing B7-H3","Inclusion Criteria:\n\nEligibility Criteria for Prescreening\n\nPurpose of prescreening is to establish B7-H3 expression by IHC performed at Boston Children's Hospital. This can be performed at any time prior to completing the protocol screening process. Participants who meet the following criteria, will be offered participation in the full screening process and protocol enrollment, if eligible:\n\n* Participants must have histologically confirmed diagnosis of a solid tumor that is relapsed or refractory for which standard curative measures do not exist or are no longer effective.\n* Participant must have adequate pre-trial tumor material available to determine B7-H3 status. Tumor tissue from the most recent resection or biopsy of recurrent disease is preferred. If unavailable, tumor tissue from prior recurrences or from the time of initial diagnosis is acceptable.\n* Age \\>=9 months and \\\u003C30 years.\n* Lansky\u002FKarnofsky performance status ≥50% (see Appendix A)\n* Life expectancy of greater than 12 weeks\n* Participants who are screened for this trial should be reasonably anticipated to meet the eligibility criteria for enrollment described in Section 3.2 if their tumor is B7-H3-positive.\n* Ability to understand and\u002For the willingness of their parent or legally authorized representative to sign a written informed consent document for prescreening.\n\nEligibility Criteria for Enrollment The following criteria are required for initial study enrollment. Once enrolled, participants will need to meet specific criteria prior to lymphocyte apheresis, prior to the receipt of lymphodepletion chemotherapy and B7-H3.CD28Z.CART cell infusion as outlined in the Treatment Section of the Protocol.\n\nLaboratory tests required for eligibility must be completed within 28 days prior to the date of registration. Disease evaluation is required only if needed for eligibility.\n\nThe screening window is 28 days.\n\n* Participants must have histologically confirmed diagnosis of a solid tumor that is relapsed or refractory for which standard curative measures do not exist or are no longer effective.\n* Participants must have measurable or evaluable disease for dose escalation. For expansion phase, participants with neuroblastoma must have measurable or evaluable disease by INRC. Participants with other solid tumors must have measurable disease by RECIST1.1 for the expansion phase.\n* B7-H3 expression: Demonstration of B7-H3 expression with H score \\>100 by immunohistochemistry (IHC) is required by IHC performed at Boston Children's Hospital. Participants may choose to enroll on the prescreening portion, which allows for assessment of B7-H3 expression only, prior to enrollment on the full clinical trial as described in Section 3.1.\n* Age \\>=12 months and \\\u003C30 years.\n* Lansky\u002FKarnofsky performance status ≥50% (see APPENDIX A: PERFORMANCE STATUS SCALES\u002FSCORES)\n* Life expectancy of greater than 12 weeks\n* Prior therapy:\n\nAt enrollment these criteria do not apply to participants with available leukapheresis products; however, participants must meet all other eligibility criteria and meet criteria to start lymphodepleting chemotherapy as outlined in Section 5.4.1.\n\nParticipants must have received prior radiation therapy and\u002For chemotherapy and recovered from all acute treatment-related toxicities of prior therapy prior to entering this study. There is no upper limit to the number of prior therapies allowed. Participants must be:\n\n* At least 1 week post any small port radiation therapy; at least 6 weeks from large field or other substantial bone marrow irradiation (craniospinal, whole abdomen, total lung, total body irradiation, \\>50% marrow).\n* At least 2 weeks since any prior myelosuppressive chemotherapy\n* At least 28 days from other investigational antineoplastic or disease-directed agents\n* At least 7 days from most recent myeloid growth factor, at least 14 days must have elapsed after receipt of pegfilgrastim.\n* At least 7 days from prior biologic antineoplastics, tyrosine kinase inhibitor, targeted agent or metronomic non-myelosuppressive chemotherapy.\n* At least 21 days or 5 half-lives, whichever is shorter, post any treatment with monoclonal antibodies (including checkpoint inhibitors and bevacizumab)\n* At least 7 days from dinutuximab treatment\n* At least 8 weeks from prior cellular therapy or vaccine therapy with recovery of associated toxicities. If prior CAR T cells, need documented lack of persistence of prior product.\n* At least 6 weeks post 131I-MIBG therapy or other radioisotope therapy\n* At least 6 weeks post autologous stem cell therapy infusion following myeloablative conditioning\n* Participants can be eligible after autologous stem cell infusion without myelosuppressive therapy at any time as long as other criteria are met.\n* At least 12 weeks post allogeneic stem cell transplant with no evidence of GVHD or ongoing toxicities.\n* Steroid use: Corticosteroids at or below physiologic doses (replacement therapy for management of pituitary\u002Fadrenal insufficiency) is allowed and\u002For topical administration (e.g. inhaled or dermatologic) is allowed. Hydrocortisone for blood product premedication is allowed.\n\n  * Participants must have normal marrow function as defined below: Values must be without transfusions or platelet growth factor within 7 days. Participants with known marrow involvement are exempt from these requirements.\n* hemoglobin ≥7.0g\u002FdL\n* absolute neutrophil count ≥750\u002FmcL\n* platelets ≥75,000\u002FmcL\n\n  * Adequate renal function defined as creatinine below normal limits for age OR creatinine clearance (as estimated by Cockcroft Gault Equation for participants ≥ 18yo and Bedside Schwartz for participants \\\u003C18yo) ≥70mL\u002Fmin\u002F1.73m2\n* Maximu Serum Creatinine (mg\u002FdL)\n\n  * 6 months to 1 year: MALE = 0.5 FEMALE = 0.6\n  * 1 year \\\u003C 2 years: MALE = 0.6 FEMALE = 0.6\n  * 2 year \\\u003C 6 years: MALE = 0.8 FEMALE = 0.8\n  * 6 years \\\u003C 10 years: MALE = 1 FEMALE = 1\n  * 10 years \\\u003C 13 years: MALE = 1.2 FEMALE = 1.2\n  * 13 years \\\u003C 16 years: MALE = 1.5 FEMALE = 1.4\n  * ≥16 years: MALE = 1.7 FEMALE = 1.4\n\n    * Adequate hepatic function\n* Serum ALT\u002FAST \\\u003C3.0X ULN\n* Total bilirubin \\\u003C 3X ULN, except in participants with confirmed Gilbert's syndrome, where direct bilirubin must be \\\u003C3X ULN.\n\n  * Adequate cardiac function\n* Ejection fraction ≥50% or fractional shortening ≥28%, measured by echocardiography\n\n  * Adequate pulmonary function\n* No evidence of dyspnea at rest\n* No exercise intolerance due to pulmonary insufficiency\n* Pulse oximetry \\>92% while breathing room air\n\n  * Females of childbearing potential must have a negative serum or urine pregnancy test\n  * The effects of B7-H3.CD28z.CART on the developing human fetus are unknown. For this reason and because other chemotherapeutic agents used in the study 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 the study, and for 1 year after receiving the preparative lymphodepletion regimen or for as long as B7-H3.CD28z.CARTs are detectable in peripheral blood. 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, and for 1 year after receiving the preparative lymphodepletion regimen or for as long as B7-H3.CD28z.CARTs are detectable in peripheral blood.\n  * Ability to understand and\u002For the willingness of their parent or legally authorized representative to sign a written informed consent document. Pediatric patients will be included in the consent discussion as age-appropriate and will provide written informed assent as applicable per institutional standard.\n\nExclusion Criteria:\n\n* Participants who are receiving any other investigational agents.\n* Participants with known current brain metastases or leptomeningeal disease. Prior CNS metastatic disease is allowable if prior resection and\u002For radiation occurred at least 8 weeks prior to enrollment, without any intervening CNS metastasis, progression or recurrence, and participants are clinically stable as evidenced by no requirements for corticosteroids, no evolving neurologic deficits, and no progression of residual brain abnormalities.\n* Participants with any prior immunodeficiency or history of autoimmune disease requiring systemic steroids\u002F immunosuppressive medication\u002F disease modifying agents within the last 2 years\n* Prior solid organ transplant. Prior allogeneic or autologous stem cell transplant is permitted as outlined in Section 3.2.\n* Active or uncontrolled viral, bacterial or fungal infection. Participants may be receiving ongoing therapy for controlled infection.\n* Participants with a known additional malignancy other than non-melanomatous skin cancer or carcinoma in situ, unless not requiring active treatment and stable or disease-free for at least 3 years.\n* CNS disorder such as cerebrovascular ischemia\u002Fhemorrhage, dementia, cerebellar disease, or autoimmune disease with CNS involvement that in the judgement of the investigator may impair the ability to evaluate neurotoxicity.\n* History of severe hypersensitivity reaction to compounds of similar chemical or biologic compositions to any agents used in the study or in the manufacturing of cells.\n* HIV\u002FHBV\u002FHCV infection: Participants are required to be negative for HIV Antibody or HIV viral load, negative for Hepatitis surface antigen (HbsAg) or viral load and negative for HCV antibody or HCV viral load. These participants are ineligible because of the potential for in vivo retroviral recombination events that could lead to replication-competent γ-retrovirus. A history of HIV, Hepatitis B, or Hepatitis C is permitted if the viral load is undetectable per quantitative PCR and\u002For nucleic acid testing.\n* Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n* Pregnant women are excluded from this study because the effects of B7-H3.CD28Z.CART cells on the developing fetus are unknown and because chemotherapeutic agents with the potential for teratogenic or abortifacient effects are used in this study. Because there is an unknown but potential risk for adverse events in nursing infants, secondary to treatment of the mother with B7-H3.CD28Z.CART cells and chemotherapy, breastfeeding should be discontinued if the mother is treated with T cells on this study (NOTE: breast milk cannot be stored for future use while the mother is being treated on study).","9 Months",{"count":205,"type":21},40,[24],"The goal of this research study is to test if a new cell therapy (B7-H3.CD28Z.CART \u002F B7-H3 CAR T cells) is safe and effective in treating children and young adults with solid cancers whose tumors have returned or stopped responding to standard treatments (relapsed or refractory) and have been identified with a B7-H3 marker.\n\nThe names of the treatment interventions used in this study are:\n\n* B7-H3.CD28Z.CART \u002F B7-H3 CAR T cells\n* Fludarabine\n* Cyclophosphamide",[29,177],[210,150,29,177],"Relapsed Solid Tumor","NOT_YET_RECRUITING","2026-01-14",{"date":214,"type":43},"2026-01-22",{"date":216,"type":21},"2026-06",{"date":218,"type":21},"2031-12-31",{"name":220,"class":107},"Robbie Majzner",{"id":222,"slug":223,"hasResults":11,"nctId":224,"briefTitle":225,"officialTitle":226,"acronym":4,"eligibilityCriteria":227,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":228,"enrollmentInfo":229,"targetDuration":4,"studyType":22,"phases":231,"briefSummary":232,"conditions":233,"keywords":234,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":237,"lastUpdatePostDateStruct":238,"startDateStruct":240,"completionDateStruct":242,"leadSponsor":244,"locationsCount":108},"100574502","phase-1-ugt1a1-genotype-drien-phase-i-study-of-irinotecan-in-vit-regimen-for-the-treatment-of-pediatric-rr-solid-tumors-100574502","NCT06760117","UGT1A1 Genotype-drien Phase I Study of Irinotecan in VIT Regimen for the Treatment of Pediatric R\u002FR Solid Tumors","Phase I Study of Irinotecan Dose Adjustment Guided by UGT1A1*6 Genotype in VIT Regimen for the Treatment of Relapsed and Refractory Childhood Solid Tumors","Inclusion Criteria:\n\n1. Age \\\u003C 18 years.\n2. Relapsed and refractory childhood solid tumors (pathologically confirmed). Definition of relapsed and refractory patients: 1. Patients who fail to achieve GPR or CR after first-line treatment are defined as refractory; 2. Patients who achieve CR after first-line treatment but relapse after more than 1 month are defined as relapsed.\n3. Must have undergone UGT1A1\\*6 genotype testing (provided free of charge by Jiangsu Hengrui Medicine Co., Ltd.), with results being wild type (T\u002FT).\n4. Patients must be at least 3 weeks post the last myelosuppressive chemotherapy and at least 6 months post hematopoietic stem cell transplantation, 2 weeks post local radiotherapy, 6 months post craniospinal or extensive pelvic radiotherapy, or 6 weeks post extensive bone marrow radiotherapy.\n5. Must have at least one measurable lesion as defined by RECIST criteria;\n6. Karnofsky score (for ages \\> 10, see Annex I) or Lansky score (for ages ≤ 10, see Annex II) ≥ 50 points;\n7. Expected survival time ≥ 6 months;\n8. Patients must have fully recovered from all acute toxic effects of previous anticancer chemotherapy: a) Bone marrow suppression chemotherapy: at least 21 days post the last bone marrow suppressive chemotherapy; b) Hematopoietic growth factors: at least 14 days post the last dose of long-acting growth factor or 7 days post the last dose of short-acting growth factor;\n9. For patients known not to involve the BM: a) Absolute neutrophil count (ANC) ≥ 1.0×10⁹\u002FL; b) Non-transfused platelet count ≥ 100.0×10⁹\u002FL; c) Hemoglobin ≥ 80 g\u002FL;\n10. Liver and kidney functions must meet the following criteria: a) Total bilirubin (conjugated + unconjugated) ≤ 1.5× upper limit of normal (ULN) for age; b) Aspartate aminotransferase (AST) ≤ 2.5×ULN (≤ 110 U\u002FL); c) Glomerular filtration rate or creatinine clearance ≥ 70 mL\u002Fmin\u002F1.73 m² or corresponding age-normal serum creatinine; d) Serum albumin ≥ 20 g\u002FL.\n11. Capable of adhering to outpatient treatment, laboratory monitoring, and necessary clinical visits during the study period;\n12. Parents\u002Fguardians of children or adolescent subjects must be able to understand, consent to, and sign the informed consent form (ICF) and applicable child consent forms before initiating any protocol-related procedures; if parents\u002Fguardians agree, subjects must be capable of expressing consent (when applicable).\n\nExclusion Criteria:\n\n1. Patients who have previously received chemotherapy with irinotecan combined with temozolomide and vincristine, or patients who have progressed after receiving treatment with irinotecan or temozolomide;\n2. Patients receiving P450 enzyme-inducing antiepileptic drugs (antiepileptic drugs affect the clearance of irinotecan);\n3. During the study period, patients must not receive any other chemotherapy, radiotherapy, or granulocyte colony-stimulating therapy;\n4. Patients positive for hepatitis B surface antigen;\n5. Patients infected with HIV or syphilis;\n6. Patients who have previously undergone organ transplantation;\n7. Uncontrolled active systemic bacterial, viral, or fungal infections; Clostridium difficile infection requiring treatment;\n8. Patients allergic to dacarbazine or cephalosporin drugs;\n9. Patients requiring high-dose dexamethasone treatment;\n10. Patients with a severe history of neurological or psychiatric disorders, including epilepsy or autism.","18 Years",{"count":230,"type":21},39,[24],"Irinotecan is a commonly used salvage chemotherapy drug for children with relapsed and refractory solid tumors. Common dose-limiting toxicities of irinotecan include abdominal pain and diarrhea. Studies have shown that patients with UGT1A16 gene mutations have a higher incidence of these side effects, thereby limiting the dosage of irinotecan. The combination of irinotecan with temozolomide and vincristine is a common salvage chemotherapy regimen for children with relapsed and refractory solid tumors. Currently, the recommended dose of irinotecan is 50mg\u002Fm², but there is still significant room for improvement in the efficacy of VIT for these children. Whether patients with wild-type UGT1A16 can further increase the dosage of irinotecan, thereby enhancing the efficacy of the VIT regimen, is the focus of our research.",[29],[235,236],"UGT1A1 gene","irinotecan","2024-12-30",{"date":239,"type":43},"2025-01-06",{"date":241,"type":43},"2022-01-01",{"date":243,"type":21},"2025-12",{"name":245,"class":107},"Sun Yat-sen University"]