[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"ependymoma-recurrent\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:ependymoma-recurrent":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,55,122,149],{"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":17,"maxAge":130,"enrollmentInfo":131,"targetDuration":4,"studyType":22,"phases":133,"briefSummary":135,"conditions":136,"keywords":4,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":141,"lastUpdatePostDateStruct":142,"startDateStruct":144,"completionDateStruct":146,"leadSponsor":148,"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.","21 Years",{"count":132,"type":21},18,[134],"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.",[137,29,138,139,140,30,28],"CNS Malignancies","ATRT Recurrent","Pineoblastoma","Embryonal Neoplasm","2026-04-24",{"date":143,"type":47},"2026-04-29",{"date":145,"type":47},"2025-12-16",{"date":147,"type":21},"2028-05-01",{"name":53,"class":54},{"id":150,"slug":151,"hasResults":11,"nctId":152,"briefTitle":153,"officialTitle":154,"acronym":155,"eligibilityCriteria":156,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":157,"enrollmentInfo":158,"targetDuration":4,"studyType":22,"phases":160,"briefSummary":161,"conditions":162,"keywords":164,"overallStatus":111,"whyStopped":4,"lastUpdateSubmitDate":172,"lastUpdatePostDateStruct":173,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":181},"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":159,"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.",[29,30,138,163],"Rare CNS Tumor Recurrent",[70,86,165,166,167,168,169,170,171],"ATRT","Relapse","Children","antiangiogenic","metronomic","intraventricular","Rare CNS tumor","2026-02-19",{"date":174,"type":47},"2026-02-23",{"date":176,"type":47},"2014-04",{"date":178,"type":21},"2030-04",{"name":180,"class":54},"Medical University of Vienna",22]