[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"embryonal-tumor-with-multilayered-rosettes\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:embryonal-tumor-with-multilayered-rosettes":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,49,72,103,138,163],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":34,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100551831","phase-1-eflornithine-dfmo-and-amxt-1501-for-neuroblastoma-cns-tumors-and-sarcomas-100551831",false,"NCT06465199","Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas","A Phase I\u002FII Study Using Eflornithine (DFMO) and AMXT 1501 for Relapsed and Refractory Neuroblastoma, CNS Tumors, and Sarcomas","Inclusion Criteria:\n\n1. Age:\n\n   All participants : Must be a maximum of 26 years of age at diagnosis\n\n   Age at enrollment by Phase:\n   1. Safety Run-in (Dose level 1)-The first three (3) participants enrolled will be ≥ 12 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the next three (3) participants enrolled who will be ≥6 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the Phase I.\n   2. Phase I and II: ≤ 26 years of age at diagnosis.\n2. Pathology\n\n   All participants must have a confirmed pathologic diagnosis of tumor type (except for DIPG):\n   * Relapsed\u002Frefractory Neuroblastoma (NB)\n   * Relapsed\u002Frefractory Embryonal tumor with multilayer rosettes (ETMR)\n   * Relapsed\u002Frefractory Atypical teratoid rhabdoid tumor (ATRT)\n   * Newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)- radiologic diagnosis acceptable\n   * Relapsed\u002Frefractory Ewing Sarcoma (EWS)\n   * Relapsed\u002Frefractory Osteosarcoma (OST)\n3. Tumor assessment:\n\n   Disease staging must be performed at baseline during the 28 day screening period prior to first dose of study drug.\n4. Disease Status:\n\n   Relapsed or Refractory Neuroblastoma Relapsed disease defined as: High-risk neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy, with or without radiation, surgery, and immunotherapy, or according to a standard high-risk treatment\u002Fneuroblastoma protocol).\n\n   Refractory disease defined as: High-risk neuroblastoma that 1) failed to achieve CR after at least 4 cycles of aggressive multi-drug induction chemotherapy with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment\u002Fneuroblastoma protocol, or 2) progression during upfront therapy or 3) with disease remaining after standard immunotherapy.\n\n   Eligible NB participants may have active disease or no active disease.\n\n   NB participants with no active disease need to meet the following criteria:\n\n   Timing from prior therapy: Enrollment (first dose of study drug) no later than 60 days from most recent therapy.\n\n   NB participants with active disease need to meet the following criteria:\n   * Received at least one recent treatment for their relapse\u002Frefractory disease and is stable (SD) or better on this treatment.\n   * Participants must not have disease in any organs (including lungs, liver, or brain).\n\n   Relapsed or refractory ETMR\u002FATRT Participants that have relapsed following standard of care therapy or having progressed during standard of care therapy and non-responsive\u002Fprogressive to accepted curative therapy, including up-front chemotherapy and radiation and\u002For high-dose chemotherapy with stem cell rescue.\n\n   ETMR\u002FATRT participants with no active disease need to meet the following criteria:\n\n   Timing from prior therapy: Enrollment (first dose of study drug) no later than 60 days from most recent therapy.\n\n   ETMR\u002FATRT participants with active disease need to meet the following criteria:\n\n   • Received at least one recent treatment for their relapse\u002Frefractory disease and is stable (SD) or better on this treatment.\n\n   Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) Participants with DIPG to start greater than 30 days, and no longer than 60 days, after standard of care radiation therapy.\n\n   Participants with newly-diagnosed typical DIPG, defined as tumors with a pontine epicenter and diffuse involvement of the pons on at least 1 axial T2-weighted image, are eligible. No histologic confirmation is required. Participants with metastatic disease are not eligible. Participants with a biopsy and no evidence of H3K27m mutations are eligible as long as they meet radiographic criteria. Participants with H3K27m altered DMG outside of the brainstem are not eligible. Participants with progression or recurrence after initial standard of care radiation are ineligible.\n\n   Relapsed or refractory Ewing sarcoma and osteosarcoma Participants that have relapsed following standard of care therapy or having progressed during standard of care therapy. Standard of care therapy for Ewing sarcoma and osteosarcoma includes multi-agent chemotherapy with local control consisting of either surgery or radiation therapy.\n\n   EWS\u002FOST Participants with no active disease need to meet the following criteria:\n\n   Timing from prior therapy: Enrollment (first dose of study drug) no later than 60 days from most recent therapy.\n\n   EWS\u002FOST Participants with active disease need to meet the following criteria:\n\n   • Received at least one recent treatment for their relapse\u002Frefractory disease and is stable (SD) or better on this treatment.\n5. Participants must be able to swallow capsules.\n6. Participants with CNS disease currently taking steroids must have been on a stable dose of steroids for at least one week and must not have progressive hydrocephalus at enrollment.\n7. Participants must have fully recovered from the acute toxic effects of all prior anti- cancer chemotherapy and be within the following timelines:\n\n   1. Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea).\n   2. Small Molecule Inhibitor (anti-neoplastic agent): At least 7 days since the completion of therapy with a small molecule inhibitor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the Study Chair.\n   3. Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells except for anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.) which should be at least 2 weeks since prior treatment with a monoclonal antibody.\n   4. XRT: At least 14 days since the last treatment except for radiation delivered with palliative intent to a non-target site.\n\n      Note: Participants with DIPG will be required to have had up front standard of care radiation. As above, participants with DIPG must be between 30-60 days post initial up- front radiation therapy.\n   5. Stem Cell Transplant:\n\n      1. Allogeneic: No evidence of active graft vs. host disease\n      2. Allo\u002FAuto: ≥ 45 days must have elapsed since transplant.\n   6. MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.\n8. Participants must have a Lansky or Karnofsky Performance Scale score of \\>\u002F= 60\n9. Participants must have adequate organ function at the time of enrollment:\n\n   * Hematological: Hematological recovery as defined by ANC ≥750\u002FμL (unsupported- \\>24 hrs off G-CSF and 7 days off neulasta)\n   * Liver: Adequate liver function as defined by AST and ALT \\\u003C10x upper limit of normal\n   * Cardiac: all participants must have:\n\n     1. Normal serum Cardiac Troponin Concentration\n     2. Normal BNP (B-type natriuretic peptide) Level\n     3. A QTcF ≤ 470 msec (or EKG with no significant findings)\n     4. Normal ECHO defined as:\n\n     i. Shortening fraction of ≥ 27% by echocardiogram, or ii. Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram\n   * Renal: Participants must have adequate renal function defined as:\n\n     1. For participants \\\u003C 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Bedside Schwartz equation is: \\[(0.413) X (Height in cm)\\] \u002F SCr\n     2. For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL\u002Fmin\u002F1.73 m2) or via radioisotope GFR of ≥ 70 mL\u002Fmin\u002F1.73 m2. The Cockcroft and Gault formula is: \\[(140-age) x (Wt in kg) x (0.85 if female)\\] \u002F (72 x SCr)\n10. Participants of childbearing potential must have a negative pregnancy test. Participants of childbearing potential must agree to use an effective birth control method. Participants who are lactating must agree to stop breast-feeding.\n11. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all participants (or participants' legal representative).\n\nExclusion Criteria:\n\n1. BSA of \\\u003C0.25 m2\n2. Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.\n3. Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.\n4. Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.\n5. Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.","ALL","26 Years",{"count":19,"type":20},289,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","The purpose of this study is to evaluate the investigational oral drug AMXT 1501 in combination with oral eflornithine (DFMO). An investigational drug is one that has not been approved by the U.S. Food \\& Drug Administration (FDA), or any other regulatory authorities around the world for use alone or in combination with any drug, for the condition or illness it is being used to treat.\n\nThe goals of this part of the study are:\n\n* Establish a recommended dose of AMXT 1501 in combination with DFMO\n* Test the safety and tolerability of AMXT 1501 in combination with DFMO\n* To determine the activity of study treatments chosen based on:\n* How each subject responds to the study treatment\n* How long a subject lives without their disease returning\u002Fprogressing",[27,28,29,30,31,32,33],"Atypical Teratoid\u002FRhabdoid Tumor","Embryonal Tumor With Multilayered Rosettes","Ewing Sarcoma","Diffuse Intrinsic Pontine Glioma","Osteosarcoma","Neuroblastoma","DIPG Brain Tumor",[35],"DFMO","RECRUITING","2026-06-30",{"date":39,"type":40},"2026-07-01","ACTUAL",{"date":42,"type":40},"2026-05-13",{"date":44,"type":20},"2035-05",{"name":46,"class":47},"Milton S. Hershey Medical Center","OTHER",12,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":28,"officialTitle":53,"acronym":54,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":56,"targetDuration":4,"studyType":21,"phases":58,"briefSummary":59,"conditions":60,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":71},"100582279","phase-2-embryonal-tumor-with-multilayered-rosettes-100582279","NCT06861244","PNOC031: Protocol for Embryonal Tumor With Multilayered Rosettes (ETMR)","PNOC031","The eligibility criteria listed below are interpreted literally and cannot be waived.\n\nInclusion Criteria:\n\n1. Participants must have either a molecularly or histologically confirmed embryonal tumor with multilayered rosettes.\n2. For enrollment, a confirmation of a minimum of 10-20 unstained formalin-fixed paraffin-embedded (FFPE) slides or 1 block (15-20 mg) with tumor content of 40% or greater is required. Anything less must be discussed and approved by the study chairs prior to enrollment.\n3. Prior Therapy:\n\n   1. Cohort 1 participants must not have received any prior tumor-directed therapy other than surgical resection.\n   2. Cohort 2 and 3 participants may receive tumor-directed therapy prior to enrollment. These participants must be discussed with study chairs prior to enrollment.\n4. Participants must not have received prior radiation for treatment of tumor.\n5. Participants of any age are eligible.\n6. Participants should begin induction chemotherapy within 28 days of the most recent definitive surgical procedure. Participants beginning therapy beyond 28 days from surgery, will need to discuss with study chairs.\n7. Cohort specific eligibility\n\n   1. Cohort 1: Gross-total resection, Eligible for early radiotherapy (please see age criteria below), and no evidence of metastatic disease.\n   2. Cohort 2: Gross-total resection, high dose chemotherapy (please see age criteria below), and no evidence of metastatic disease.\n   3. Cohort 3A: Metastatic or residual disease, and early radiotherapy.\n   4. Cohort 3B: Metastatic or residual disease, and high dose chemotherapy.\n   5. Radiotherapy Age Criteria (at the time of planned radiation): \\>12 months of age for participants with infratentorial tumor OR \\>15 months of age for participants with supratentorial tumor. For participants being treated on radiotherapy-containing arms, the legal parent\u002Fguardian or patient and the physician must be willing to allow the use of radiotherapy for treatment.\n8. Performance Score: 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.\n9. Organ Function Requirements:\n\n   1. Peripheral absolute neutrophil count (ANC) \\> 75,000\u002Fmm3\n   2. Platelet count \\> 75,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 72 hours prior to enrollment).\n10. Adequate Renal Function defined as:\n\n    a. Serum creatinine \\\u003C 1.5 x upper limit normal (ULN) based on age and gender.\n11. Adequate Liver Function defined as:\n\n    1. 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    2. alanine aminotransferase (ALT) \\\u003C 3 x ULN,\n    3. aspartate aminotransferase (AST) \\\u003C 3 x ULN,\n12. Adequate Neurologic Function defined as:\n\n    a. Participants with seizure disorder may be enrolled if well controlled. Participants on enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drugs.\n13. As chemotherapeutic agents used in this trial are known to be teratogenic, women and men of child-bearing potential 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 study therapy. 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.\n14. Participants must be enrolled on PNOC COMP prior to enrollment on PNOC031 if PNOC COMP is open to accrual at the enrolling institution.\n15. A legal parent\u002Fguardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.\n\nExclusion Criteria\n\n1. Cohort 1 only: Participants who have received any prior tumor-directed therapy other than surgical intervention\n2. Participants who are receiving any other tumor directed investigational agents.\n3. History of allergic reactions attributed to compounds of similar chemical or biologic composition to the agents used in study.\n4. Uncontrolled intercurrent illness.\n5. Women of childbearing potential must not be pregnant or breast-feeding.",{"count":57,"type":20},70,[24],"This is an open-label, comprehensive, iterative investigation of evaluating the use of induction chemotherapy, high-dose chemotherapy, and focal radiation therapy in children with newly diagnosed Embryonal Tumor With Multilayered Rosettes (ETMR).",[28,61],"Embryonal Tumor With Multilayered Rosettes, Nos","2026-06-26",{"date":64,"type":40},"2026-06-29",{"date":66,"type":40},"2025-03-06",{"date":68,"type":20},"2032-03-31",{"name":70,"class":47},"University of California, San Francisco",8,{"id":73,"slug":74,"hasResults":11,"nctId":75,"briefTitle":76,"officialTitle":76,"acronym":77,"eligibilityCriteria":78,"healthyVolunteers":11,"sex":16,"minAge":79,"maxAge":80,"enrollmentInfo":81,"targetDuration":4,"studyType":21,"phases":82,"briefSummary":83,"conditions":84,"keywords":90,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":102},"100530974","phase-1-immunotherapy-for-malignant-pediatric-brain-tumors-employing-adoptive-cellular-therapy-impact-100530974","NCT06193759","Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)","IMPACT","RECIPIENT SCREENING INCLUSION CRITERIA\n\n1. Diagnosis (select one group):\n\n   * Group A: New diagnosis of CNS embryonal tumors: medulloblastoma, embryonal tumor with multilayered rosettes, pineoblastoma, atypical teratoid\u002Frhabdoid tumor, and embryonal tumor, not otherwise specified (NOS).\n   * Group B: Radiographic evidence consistent with recurrent ependymoma, with planned or recent re-resection.\n2. Age:\n\n   * Group A: \\\u003C5 years of age at enrollment\n   * Group B: \\>1 year and \\\u003C30 years of age at enrollment\n3. Tissue:\n\n   o Group A: Availability of sufficient fresh or frozen tumor tissue (approximately 50 mg).\n\n   o Group B: Expectation of sufficient fresh or frozen tumor tissue, in the opinion of study PI or sub-I (based upon radiographic evidence of disease).\n4. Non-pregnant:\n\n   * Group A: N\u002FA\n   * Group B: For female of childbearing potential, must have negative pregnancy test.\n\n   Common to both groups:\n5. Karnofsky or Lansky score of ≥60%.\n6. Adequate organ function, defined below:\n\n   i. ANC ≥750\u002FµL. ii. Absolute lymphocyte count (ALC) \\>500\u002FμL. iii. Platelets ≥75K. iv. Bilirubin ≤3xULN. v. Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) \\\u003C5x upper limit of normal (ULN).\n\n   vi. Serum creatinine ≤1.0 mg\u002FdL or 1.5x ULN for age (whichever is higher). vii. Pulse oximetry \\>90% on room air.\n7. The patient (if ≥18 years old), or the patient's parent(s)\u002Flegal guardian(s) (if the patient is a minor), is capable of providing informed consent.\n8. Patient deemed to be of sufficient size to undergo MNC apheresis for TSA-T generation (Groups A and B) and PBSC rescue (Group A only).\n9. Patient is a surgical candidate for placement of a Rickham reservoir in the opinion of study PI or medically licensed sub-I.\n\nRECIPIENT INCLUSION CRITERIA FOR PROCUREMENT\n\n1\\. Karnofsky or Lansky score of ≥60%. 2. Adequate organ function, defined below: i. ANC ≥750\u002FµL. ii. Absolute lymphocyte count (ALC) \\>500\u002FμL. iii. Platelets ≥75K. iv. Bilirubin ≤3xULN. v. Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) \\\u003C5x upper limit of normal (ULN).\n\nvi. Serum creatinine ≤1.0mg\u002FdL or 1.5x ULN for age (whichever is higher). vii. Pulse oximetry \\>90% on room air. 3. Non-pregnant:\n\n* Group A: N\u002FA\n* Group B: For female of childbearing potential (if applicable), must have negative pregnancy test.\n\nRECIPIENT INCLUSION CRITERIA FOR INITIAL TSA-T ADMINISTRATION AND FOR ADDITIONAL INFUSIONS\n\n1. Applicable to TSA-T infusion #1 only: Group B participants must have histopathologic confirmation of recurrent ependymoma.\n2. Karnofsky or Lansky score of ≥60%.\n3. Adequate organ function, defined as below:\n\n   i. Bilirubin ≤3x ULN. ii. AST and ALT ≤5x ULN. iii. Serum creatinine ≤1.0mg\u002FdL or 1.5x ULN for age (whichever is higher). iv. Pulse oximetry \\>90% on room air.\n4. Applicable to TSA-T Infusion #1 only: Adequate count recovery, as described below, from prior therapies:\n\n   i. Absolute Neutrophil Count (ANC) \\>1000\u002FμL ii. Absolute Lymphocyte Count (ALC) \\>500\u002FμL\n5. Patients must have received their last dose of:\n\n   a. Myelosuppressive chemotherapy (if applicable) ≥14 days prior to TSA-T infusion b. Focal radiation (if applicable) ≥14 days prior to TSA-T infusion c. Craniospinal irradiation (if applicable) ≥28 days prior to TSA-T infusion\n6. Patients must have recovered from all acute effects of prior surgical intervention\u002Fs.\n7. Group B female of childbearing potential or male capable of fathering a child (if applicable): Agree to use contraceptive measures during TSA-T treatment participation through 6 months following last administration of TSA-Ts\n8. Group B female of childbearing potential (if applicable), must have negative pregnancy test.\n9. Neurologic status: Patient must have a stable neurologic exam for 2 weeks, on a stable or decreasing dose of steroids, prior to administration of the first dose of TSA-T cells, and stability for 1 week prior to all subsequent infusions. The exams demonstrating stability must be performed by the study team, although these may occur via telemedicine if necessary. Patient must agree to a brief (\\\u003C72 hours) course of steroids if the PI or medically-licensed sub-I deems it clinically necessary in the context of clinical deterioration.\n10. Presence of a Rickham reservoir and catheter for intracerebroventricular administration of TSA-T therapy, placed \\>7 days prior to TSA-T infusion.\n11. For patients with programmable VP shunts: Able to tolerate the shunt being closed for at least 4 hours, in the opinion of study PI or medically licensed sub-I.\n\nEXCLUSION CRITERIA RECIPIENT SCREENING EXCLUSION CRITERIA\n\n1\\. Patients with uncontrolled infections. 2. Patients with known HIV infection. 3. Group A patients with medulloblastoma of the SHH subtype.\n\nRECIPIENT EXCLUSION CRITERIA FOR PROCUREMENT\n\n1\\. Patients with a fever above 38.0°C. 2. Patients with known HIV infection. 3. Prior immunotherapy with an investigational agent within the 28 days prior to planned date of procurement collection for TSA-T manufacturing.\n\n4\\. Patients who will be unable to tolerate the apheresis procedure, including inability to tolerate placement of apheresis line (if applicable), in the opinion of PI or medically licensed sub-I.\n\n5\\. Patients who have overly bulky tumors on imaging are ineligible. These include the following: i. Tumor with any evidence of herniation or significant midline shift. ii. Tumor with a significant brainstem component. iii. Patients who are deemed to have overly bulky tumor by the PI of the study.\n\nIf, due to complications during apheresis or subsequent manufacturing, procurement is repeated at a later date using peripheral whole blood collection, exclusion criterion #4 does not apply.\n\nRECIPIENT EXCLUSION CRITERIA FOR INITIAL AND SUBSEQUENT TSA-T INFUSIONS\n\n1. Patients with progressive disease based on most recent evaluation (for subsequent infusions).\n\n   a. Patients with progressive disease based on most recent evaluation may receive initial TSA-T infusion but would be ineligible if the tumor is found to be progressive before subsequent infusions\n2. Patients with uncontrolled infections.\n3. Patients who have overly bulky tumors on imaging are ineligible. These include the following:\n\n   i. Tumor with any evidence of herniation or significant midline shift. ii. Tumor with a significant brainstem component. iii. Patients who are deemed to have overly bulky tumor by the PI of the study.\n4. Patients who received ATG, Campath or other immunosuppressive T cell monoclonal antibodies within 28 days of TSA-T infusion.\n5. Patients receiving steroids (e.g., dexamethasone) at a dose of \\>0.05 mg\u002Fkg\u002Fday.\n6. Patients who have non-programmable VP shunts.","1 Year","30 Years",{"count":48,"type":20},[23],"This is an open-label phase 1 safety and feasibility study that will employ multi-tumor antigen specific cytotoxic T lymphocytes (TSA-T) directed against proteogenomically determined personalized tumor-specific antigens (TSA) derived from a patient's primary brain tumor tissues. Young patients with embryonal central nervous system (CNS) malignancies typically are unable to receive irradiation due to significant adverse effects and are treated with intensive chemotherapy followed by autologous stem cell rescue; however, despite intensive therapy, many of these patients relapse. In this study, individualized TSA-T cells will be generated against proteogenomically determined tumor-specific antigens after standard of care treatment in children less than 5 years of age with embryonal brain tumors. Correlative biological studies will measure clinical anti-tumor, immunological and biomarker effects.",[85,86,28,87,88,89],"Medulloblastoma, Childhood","Atypical Teratoid\u002FRhabdoid Tumor of CNS","Pineoblastoma","Embryonal Brain Tumor Not Otherwise Specified","Ependymoma",[91,92],"Cellular Therapy","Pediatric Brain Tumors","2026-06-15",{"date":95,"type":40},"2026-06-17",{"date":97,"type":40},"2024-09-20",{"date":99,"type":20},"2032-12-29",{"name":101,"class":47},"Children's National Research Institute",1,{"id":104,"slug":105,"hasResults":11,"nctId":106,"briefTitle":107,"officialTitle":108,"acronym":4,"eligibilityCriteria":109,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":110,"enrollmentInfo":111,"targetDuration":4,"studyType":21,"phases":113,"briefSummary":115,"conditions":116,"keywords":127,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":129,"lastUpdatePostDateStruct":130,"startDateStruct":132,"completionDateStruct":134,"leadSponsor":136,"locationsCount":48},"100588487","early-phase-1-pilot-study-of-it-topotecan-and-maintenance-chemotherapy-for-hr-ebts-in-children--6-years-post-consolidation-100588487","NCT06942039","Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children \u003C 6 Years, Post Consolidation","A Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy in the Post-consolidation Setting for the Treatment of High-risk Embryonal Central Nervous System Tumours in Children Less Than 6 Years of Age","Inclusion Criteria:\n\n1. Tumor Tissue Sample\n2. Age: Patient must be aged ≥ 0 years to ≤ 6 years at the time of definitive confirmation of histologic diagnosis of eligible CNS tumor.\n3. Diagnoses. Participants must have Central nervous system (CNS) HR-EBT including atypical teratoid rhabdoid tumour (ATRT), group 3 and group 4 medulloblastoma (MB), pineoblastoma, CNS neuroblastoma, embryonal tumor with multi-layered rosettes (ETMR including embryonal tumor with abundant neuropil and true rosettes (ETANTR), ependymoblastoma and ETMR not otherwise specified), medulloepithelioma, CNS embryonal tumor with rhabdoid features (INI-1 intact) and CNS embryonal tumor, not otherwise specified. Metastatic disease included. Any extent of resection included.\n4. Cranial and Spine MRI. A baseline MRI brain and spine with and without contrast is required for all patients. cranial MRI (with and without gadolinium) must be done pre-operatively. Post-operatively, cranial MRI (with and without gadolinium) must be done.\n5. Lumbar Puncture (LP) CSF for cytopathology (strongly recommended but not mandatory; if medically feasible). A baseline LP CSF cytology either pre-operatively or post-operatively at least 10 days after definitive surgery for all patients if medically feasible (This is not mandatory and will not make the patient ineligible).\n6. Life expectancy: Patients must have a life expectancy of greater than 8 weeks from diagnosis.\n7. Performance level: Patients must have a performance status corresponding of a Lansky score ≥ 50.\n8. Organ Function Requirements: Participants must have normal organ and marrow function as defined below:\n\nAdequate renal function defined as:\n\n\\- Creatinine clearance (12-24-hour urine collection) or radioisotope glomerular filtration rate (GFR) ≥ 60 ml\u002Fmin\u002F1.73m2\n\nAdequate cardiac function defined as:\n\n* Shortening fraction of ≥ 27% by echocardiogram, or\n* Ejection fraction of ≥ 47% by radionuclide angiogram.\n\nAdequate pulmonary function defined as:\n\n\\- No evidence of dyspnea at rest and a pulse oximetry \\> 94% on room air.\n\nAdequate Bone Marrow Function defined as:\n\n* Peripheral absolute neutrophil count (ANC) \\> 1000\u002FμL\n* Platelet Count \\> 100,000\u002FμL (without transfusion for 3 days)\n* Hemoglobin greater than 8 gm\u002FdL (may have received red blood cell (RBC) transfusions)\n\nAdequate liver function defined as:\n\n* Total bilirubin ≤ 1.5X upper limit of normal (ULN) within normal institutional limits for age (patients with documented Gilbert's Disease may be enrolled with Study Chair approval and total bilirubin ≤ 2.0 × ULN)\n* Alanine Aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 100 U\u002FL\n\nExclusion criteria:\n\n1. Patients who are receiving any other conventional anti-cancer agents or investigational agents.\n2. Patients who received previous therapy including radiotherapy or chemotherapy other than corticosteroids.\n3. Presence of another malignancy, except if the other primary malignancy is neither currently clinically significant nor requiring active intervention.\n4. Concomitant medications restrictions: Concurrent use of enzyme inducing anticonvulsants (e.g. phenytoin, phenobarbital, and carbamazepine), selected strong inhibitors of cytochrome P450 3A4 include azole antifungals, such as fluconazole, voriconazole, itraconazole, ketoconazole, and strong inducers include drugs such as rifampin, phenytoin, phenobarbitol, carbamazepine, and St. John's wort or CYP450 3A4 stimulators or inhibitors.\n5. Other uncontrollable medical disease: Patient has a severe and uncontrollable medical disease (i.e., uncontrolled diabetes, hyperglycemia, chronic renal disease or active uncontrolled infection), has chronic liver disease (i.e., chronic active hepatitis and cirrhosis), hypercholesterolemia (serum cholesterol \\>300 mg\u002FdL), intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, active hyperparathyroidism, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n6. Patients who have a known diagnosis of human immunodeficiency virus (HIV) infection, hepatitis B or C.\n7. Ineligible diagnoses for study entry by neuropathology: This includes sonic hedgehog (SHH) and wingless (WNT) MBs, all ependymomas, all choroid plexus carcinomas, all high grade glial and glio-neuronal tumors, all diffuse midline gliomas, all primary CNS germ cell tumors, all primary CNS sarcomas, all primary or metastatic CNS lymphomas and solid leukemic lesions (chloromas, granulocytic sarcomas).\n8. The participant or parent(s)\u002Fguardian(s) cannot comply with the study visit schedule and other protocol requirements, in the investigator's opinion.\n\nAll patients must meet inclusion\u002Fexclusion criteria prior to starting any protocol therapy.","6 Years",{"count":112,"type":20},15,[114],"EARLY_PHASE1","Pilot study to determine feasibility of adding intrathecal chemotherapy and maintenance therapy after high dose chemotherapy for treatment of newly diagnosed HR-EBTs in patients less than 6 years of age.",[117,118,119,85,120,121,87,32,28,122,123,124,125,126],"CNS Embryonal Tumor","CNS, Medulloblastoma","Atypical Teratoid Rhabdoid Tumor","Medulloblastoma, Group 3","Medulloblastoma, Group 4","Embryonal Tumor With Abundant Neuropil and True Rosettes","Ependymoblastoma","Medulloepithelioma","CNS Embryonal Tumor With Rhabdoid Features","CNS Embryonal Tumor, Nos",[128],"HR-EBT","2026-06-05",{"date":131,"type":40},"2026-06-08",{"date":133,"type":40},"2025-09-23",{"date":135,"type":20},"2032-12-31",{"name":137,"class":47},"C17 Council",{"id":139,"slug":140,"hasResults":11,"nctId":141,"briefTitle":142,"officialTitle":142,"acronym":4,"eligibilityCriteria":143,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":144,"targetDuration":146,"studyType":147,"phases":4,"briefSummary":148,"conditions":149,"keywords":150,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":154,"lastUpdatePostDateStruct":155,"startDateStruct":157,"completionDateStruct":159,"leadSponsor":161,"locationsCount":102},"100423502","etmr-one-an-international-registry-and-research-platform-for-children-with-embryonal-tumor-with-multilayer-rosettes-100423502","NCT04794686","ETMR One: An International Registry and Research Platform for Children With Embryonal Tumor With Multilayer Rosettes","Inclusion Criteria:\n\n1. Patients of any age\n2. Patients must have either a histologically confirmed primary intracranial CNS Embryonal Tumor with Multilayer Rosettes (as agreed upon by central review or local pathologist OR\n3. Patients must have tumor tissue that possesses C19MC amplification . Central Review is not required (but is strongly recommended) if the patient's tumor does not demonstrate C19MC amplification.\n4. Patients may be enrolled at point following diagnosis",{"count":145,"type":20},100,"10 Years","OBSERVATIONAL","This research is being done to find out more information about a brain tumor called Embryonal Tumor with Multilayer Rosettes (ETMR) by collecting medical information from children who have this disease.\n\nThe purpose of this research study is to create and maintain a research database for patients with ETMR. The database will include information about occurrence rates, patient information, tumor tissue information, and response to treatment. This will help advance our understanding of this rare disease.\n\nIn addition, this study will include obtaining survival data and evaluating therapeutic response to expert consensus therapy, and procuring patient tumor tissue.",[28],[151,152,153],"Embryonal","Multilayer Rosettes","CNS tumor","2026-05-29",{"date":156,"type":40},"2026-06-02",{"date":158,"type":40},"2020-07-12",{"date":160,"type":20},"2030-07-12",{"name":162,"class":47},"Hackensack Meridian Health",{"id":164,"slug":165,"hasResults":11,"nctId":166,"briefTitle":167,"officialTitle":168,"acronym":4,"eligibilityCriteria":169,"healthyVolunteers":11,"sex":16,"minAge":170,"maxAge":171,"enrollmentInfo":172,"targetDuration":4,"studyType":21,"phases":173,"briefSummary":174,"conditions":175,"keywords":185,"overallStatus":189,"whyStopped":4,"lastUpdateSubmitDate":190,"lastUpdatePostDateStruct":191,"startDateStruct":193,"completionDateStruct":195,"leadSponsor":197,"locationsCount":199},"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","21 Years",{"count":57,"type":20},[23],"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)",[176,177,178,179,180,181,85,182,183,89,27,28,87,184],"Central Nervous System Neoplasms","Brain Tumor","Brain Tumor, Recurrent","Brain Tumor, Pediatric","Brain Tumor Adult","Medulloblastoma","Medulloblastoma, Adult","Medulloblastoma Recurrent","Leptomeningeal Disease",[176,177,179,186,187,181,85,182,183,89,27,188,87,184],"Brain Tumor, Adult","Brain Tumor Recurrent","Embryonal Tumor with Multilayered Rosettes","NOT_YET_RECRUITING","2026-01-28",{"date":192,"type":40},"2026-02-05",{"date":194,"type":20},"2026-07",{"date":196,"type":20},"2032-08-31",{"name":198,"class":47},"Robbie Majzner",2]