[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pineoblastoma\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pineoblastoma":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,49,85,114,151,189],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"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":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100530974","phase-1-immunotherapy-for-malignant-pediatric-brain-tumors-employing-adoptive-cellular-therapy-impact-100530974",false,"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.","ALL","1 Year","30 Years",{"count":20,"type":21},12,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","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.",[27,28,29,30,31,32],"Medulloblastoma, Childhood","Atypical Teratoid\u002FRhabdoid Tumor of CNS","Embryonal Tumor With Multilayered Rosettes","Pineoblastoma","Embryonal Brain Tumor Not Otherwise Specified","Ependymoma",[34,35],"Cellular Therapy","Pediatric Brain Tumors","RECRUITING","2026-06-15",{"date":39,"type":40},"2026-06-17","ACTUAL",{"date":42,"type":40},"2024-09-20",{"date":44,"type":21},"2032-12-29",{"name":46,"class":47},"Children's National Research Institute","OTHER",1,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":56,"enrollmentInfo":57,"targetDuration":4,"studyType":22,"phases":59,"briefSummary":61,"conditions":62,"keywords":74,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":76,"lastUpdatePostDateStruct":77,"startDateStruct":79,"completionDateStruct":81,"leadSponsor":83,"locationsCount":20},"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":58,"type":21},15,[60],"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.",[63,64,65,27,66,67,30,68,29,69,70,71,72,73],"CNS Embryonal Tumor","CNS, Medulloblastoma","Atypical Teratoid Rhabdoid Tumor","Medulloblastoma, Group 3","Medulloblastoma, Group 4","Neuroblastoma","Embryonal Tumor With Abundant Neuropil and True Rosettes","Ependymoblastoma","Medulloepithelioma","CNS Embryonal Tumor With Rhabdoid Features","CNS Embryonal Tumor, Nos",[75],"HR-EBT","2026-06-05",{"date":78,"type":40},"2026-06-08",{"date":80,"type":40},"2025-09-23",{"date":82,"type":21},"2032-12-31",{"name":84,"class":47},"C17 Council",{"id":86,"slug":87,"hasResults":11,"nctId":88,"briefTitle":89,"officialTitle":90,"acronym":91,"eligibilityCriteria":92,"healthyVolunteers":11,"sex":16,"minAge":93,"maxAge":94,"enrollmentInfo":95,"targetDuration":4,"studyType":22,"phases":97,"briefSummary":98,"conditions":99,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":106,"lastUpdatePostDateStruct":107,"startDateStruct":109,"completionDateStruct":111,"leadSponsor":113,"locationsCount":48},"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.","3 Years","21 Years",{"count":96,"type":21},18,[60],"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.",[100,101,102,30,103,104,105],"CNS Malignancies","Medulloblastoma Recurrent","ATRT Recurrent","Embryonal Neoplasm","Ependymoma Recurrent","High Grade Gliomas","2026-04-24",{"date":108,"type":40},"2026-04-29",{"date":110,"type":40},"2025-12-16",{"date":112,"type":21},"2028-05-01",{"name":46,"class":47},{"id":115,"slug":116,"hasResults":11,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":4,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":16,"minAge":121,"maxAge":94,"enrollmentInfo":122,"targetDuration":4,"studyType":22,"phases":124,"briefSummary":125,"conditions":126,"keywords":136,"overallStatus":140,"whyStopped":4,"lastUpdateSubmitDate":141,"lastUpdatePostDateStruct":142,"startDateStruct":144,"completionDateStruct":146,"leadSponsor":148,"locationsCount":150},"100622970","phase-1-b7-h3cd28zcart-in-cns-neoplasms-100622970","NCT07390539","B7-H3.CD28Z.CART in CNS Neoplasms","A Phase 1\u002F1b Study of Autologous b7-h3 Chimeric Antigen Receptor t Cells (b7-h3.cd28z.Cart) in Children and Young Adults With Recurrent or Progressive Cns Neoplasms Expressing b7-h3 Target","Pre-screening Inclusion Criteria:\n\n* Participants must have histologically and\u002For molecularly confirmed CNS embryonal tumor (see eligible tumor types below), OR ependymoma that is recurrent\u002Fprogressive following standard of care treatment.\n\n  --Eligible CNS embryonal tumor types include:\n  * Medulloblastoma\n  * Atypical Teratoid Rhabdoid Tumor (ATRT)\n  * Embryonal Tumor with Multilayered Rosettes (ETMR) Pineoblastoma\n  * Other CNS embryonal tumor types, at the discretion of the study chair (or designee)\n* Participants must have adequate pre-trial tumor material available to determine B7- H3 expression status. Tumor tissue from the most recent resection or biopsy of recurrent disease is preferred. If unavailable, tumor tissue from prior recurrences or from time of initial diagnosis is acceptable. Biopsies will not be performed for participation in this research trial or for research purposes.\n* Pre-screening IHC Consent: All participants ≥ 18 years of age must be able to give informed consent. For participants \\\u003C18 years of age, their legal authorized representative (LAR) (i.e. parent or guardian) must give informed consent. Pediatric participants will be included in age-appropriate discussion and written assent will be obtained for those ≥10 years of age, where appropriate. If a minor becomes of age during participation of this study, they will be asked to reconsent as an adult.\n\nInclusion Criteria:\n\n* Participants must have histologically and\u002For molecularly confirmed CNS embryonal tumor (see eligible tumor types below), OR ependymoma that is recurrent\u002Fprogressive following standard of care treatment.\n\n  --Eligible CNS embryonal tumor types include:\n  * Medulloblastoma\n  * Atypical Teratoid Rhabdoid Tumor (ATRT)\n  * Embryonal Tumor with Multilayered Rosettes (ETMR)\n  * Pineoblastoma\n  * Other CNS embryonal tumor types, at the discretion of the study chair (or designee)\n* B7-H3 expression: Demonstration of B7-H3 expression with H score greater than 100 by immunohistochemistry (IHC) is required.\n* Age: greater than or equal to two (2) years of age and less than or equal to 21 years of age. The first participant treated at each dose level within each stratum (Standard Risk and High Risk) will be ≥ 6 years of age when feasible.\n* Disease status: Participants must have evaluable disease in the central nervous system to be eligible. Evaluable disease includes either measurable OR non-measurable disease, defined as follows:\n\n  --Measurable disease (contrast-enhancing or non-enhancing tumor)\n  * Clearly defined lesional margins with two perpendicular diameters of at least 10mm, OR\n  * At least two times (in both perpendicular diameters) the MRI slice thickness, plus the interslice gap\n\n    --Non-measurable disease (tumor that is too small to be accurately measured)\n  * Lesion that is measurable in only one perpendicular dimension, OR\n  * Lesion that is less than 10mm in at least one perpendicular dimension, OR\n  * Lesion that is less than two times the MRI slice thickness, plus the interslice gap\n  * Note: Leptomeningeal (LM) disease is considered non-measurable but evaluable.\n* Performance status: Karnofsky performance status ≥60% for participants ≥16 years of age and Lansky performance status ≥60% for participants \\\u003C16 years of age (see APPENDIX A PERFORMANCE STATUS CRITERIA). NOTE: Participants with neurologic deficits must have a stable neurologic exam for seven (7) days prior to enrollment. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n* Life expectancy of greater than 12 weeks\n* Prior therapy: Participants must have received prior standard of care therapy, including maximal safe surgical resection, radiation therapy and\u002For standard chemotherapy, and is recovered from all acute treatment-related toxicities (defined as ≤ Grade 1 or stable) from all prior therapy before entering this study There is no upper limit to the number of prior therapies allowed, but must have received all standard curative options for their tumor type.\n* Participants must meet the following washouts prior to enrollment:\n\n  * Radiation therapy - Participants must have had their last fraction of:\n\n    ---Craniospinal irradiation, whole brain radiation therapy, or radiation therapy to \\>50% of the pelvis or spine \\>28 days prior to enrollment\n\n    ---Focal irradiation (small port) \\>14 days prior to enrollment\n  * At least 14 days since any prior cytotoxic chemotherapy\n  * At least 7 days since any biologic antineoplastics, tyrosine kinase inhibitor, targeted agent\n  * At least 21 days or 5 half-lives (whichever is shorter) since any investigational antineoplastic or disease-directed agent (but at least 28 days from prior investigational antineoplastic vaccine therapy)\n  * At least 21 days since any monoclonal antibody therapy\n  * At least 90 days since any systemic inhibitor\u002Fstimulatory immune checkpoint therapy\n  * At least 28 days from prior autologous stem cell transplantation, with no ongoing toxicities\n  * At least 14 days after peg-filgrastim and 7 days for hematopoietic growth factor support\n* Steroid use: Must not require concurrent systemic steroid therapy, although physiologic corticosteroid replacement therapy for management of pituitary\u002Fadrenal insufficiency and\u002For topical administration (e.g. inhaled or dermatologic) is allowed. Use of topical, ocular, intranasal, or inhaled corticosteroids are permitted per PI\n\ndiscretion.\n\n* Participants must have adequate organ function, as defined below\n\n  --Adequate bone marrow function\n  * Hemoglobin ≥ 8 g\u002FdL\n  * Absolute neutrophil count (ANC) ≥ 1000 cells\u002FuL\n  * Absolute lymphocyte count (ALC) ≥ 150 cells\u002FuL\n  * Platelets ≥100,000\u002FuL (unsupported, defined as no platelet transfusion within 4 days)\n* Adequate renal function defined as creatinine within normal limits for age OR creatinine clearance (as estimated by Cockcroft Gault Equation for participants ≥ 18yo and Bedside Schwartz for participants \\\u003C18yo) ≥70mL\u002Fmin\n\n  * Maximum Serum Creatinine mg\u002FDL ---6 months to 1 year Male 0.5 Female 0.5 ---1 to \\\u003C 2 years Male 0.6 Female 0.6 ---2 to \\\u003C 6 years Male 0.8 Female 0.8\n\n    * 6 to \\\u003C 10 years Male 1 Female 1\n    * 10 to \\\u003C 13 years Male 1.2 Female 1.2\n    * 13 years to \\\u003C 16 years Male 1.5 Female 1.4\n\n      * 16 years Male 1.7 Female 1.4\n* Adequate hepatic function\n\n  * Serum ALT\u002FAST ≤3.0 upper limit of normal (ULN)\n  * Total bilirubin ≤1.5mg\u002FdL, except in subjects with confirmed Gilbert's syndrome\n* Adequate cardiac function\n\n  --Ejection fraction ≥50% or fractional shortening ≥28%, measured by echocardiography\n* Adequate pulmonary function\n\n  * No evidence of dyspnea at rest\n  * Pulse oximetry \\>92% whilst breathing room air\n* Adequate neurologic function\n\n  * Participants with seizure disorders on anticonvulsants may be enrolled if seizures are well controlled (no seizure activity within 7 days prior to enrollment)\n  * Nervous system disorders (CTCAE v6.0) resulting from prior therapy must be ≤ Grade 2, with the exception of decreased tendon reflex (DTR; any Grade eligible). Participants with neurological deficits should be stable for a minimum of 7 days prior to enrollment. (A baseline detailed neurological exam should clearly document the neurological status of the participant prior at enrollment).\n* Females of childbearing potential must have a negative serum or urine pregnancy test (females who have undergone surgical sterilization are not considered to be of childbearing potential)\n* Participants of childbearing or child-fathering potential must be willing to practice birth control from the time of enrollment on this study and for one year after receiving the preparative lymphodepletion regimen, or for as long as B7- H3.CD28Z.CART cells are detectable in peripheral blood or CSF, whichever is later.\n* Participant or parent of participant or legally recognized representative must be able to sign a written informed consent document. Pediatric participants will be included in age-appropriate discussion and written assent will be obtained for those ≥10 years of age, where appropriate.\n\nExclusion Criteria:\n\n* Participants with bulky tumor are ineligible. Bulky tumor is defined as:\n\n  * Tumor with diameter of \\>5cm in one dimension on T2\u002FFLAIR sequence\n  * Tumor with evidence of clinically significant midline shift or uncal herniation\n  * Tumor that, in opinion of the site investigator, shows significant mass effect in either the brain or spine\n* Participants with clinical or radiological evidence of brain herniation.\n* Participants who have received other B7-H3 targeted cellular therapies. Other prior cellular therapies are eligible, including immune checkpoint inhibition and vaccine therapy. These prior therapies should be discussed with the study chair (or designee) prior to participant enrollment.\n* Concurrent illness\n\n  * Participants with any prior immunodeficiency or history of autoimmune disease requiring systemic steroids\u002F immunosuppressive medication\u002F disease-modifying agents within the last two (2) years.\n  * Uncontrolled (Grade 3) bacterial, viral, fungal, or other infection.\n  * Ongoing infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive). A history of hepatitis B or hepatitis C is permitted if the viral load is undetectable per quantitative PCR and\u002For nucleic acid testing.\n  * Evidence of severe or uncontrolled systemic disease (e.g. Grade 3 significant cardiac, pulmonary, hepatic, renal or other organ dysfunction) that in the judgement of the principal investigator is likely to interfere with assessment of safety or efficacy of the investigational regimen and its requirements.\n  * Known sensitivity or allergy to any of the agents\u002Freagents used in this study (i.e. DSMO, cyclophosphamide, fludarabine)\n  * History of severe hypersensitivity reaction to compounds of similar chemical or biologic compositions to any agent used in the study or in the manufacturing of cells.\n* Concomitant medications\n\n  * Current systemic corticosteroid therapy\n  * Note, physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency will be allowed.\n  * Participants who are receiving any other anti-cancer or investigational drug therapy are ineligible.\n  * Participants who have received the last vaccination of a live vaccine ≤ 30 days prior to the start of treatment are ineligible.\n  * Ongoing use of dietary supplements, alternative therapies or extreme diets, or any medication not approved by the study chair (or designee).\n* Any other condition which in the principal investigator's opinion makes the individual clinically unsuitable to participate in this trial, or which would jeopardize compliance with the protocol, or would make it difficult to interpret adverse events or study data.","2 Years",{"count":123,"type":21},70,[24],"The purpose of this research study is to test the safety and effectiveness of a cell therapy at different doses for children and young adults with recurrent or progressive brain tumors. Recurrent\u002Frecurred means a tumor that has gone away and then came back. This cell therapy is called B7- H3.CD28Z.CART, referred to as B7-H3 CAR T cells. B7-H3 is a protein that is over-expressed on many tumor cells, making it a good target for cancer cell therapy.\n\nThe names of the study investigational therapies involved in this study are:\n\n* Fludarabine (a type of chemotherapy)\n* Cyclophosphamide (a type of chemotherapy)\n* B7-H3 CAR T cells (a type of cellular therapy)",[127,128,129,130,131,132,27,133,101,32,134,29,30,135],"Central Nervous System Neoplasms","Brain Tumor","Brain Tumor, Recurrent","Brain Tumor, Pediatric","Brain Tumor Adult","Medulloblastoma","Medulloblastoma, Adult","Atypical Teratoid\u002FRhabdoid Tumor","Leptomeningeal Disease",[127,128,130,137,138,132,27,133,101,32,134,139,30,135],"Brain Tumor, Adult","Brain Tumor Recurrent","Embryonal Tumor with Multilayered Rosettes","NOT_YET_RECRUITING","2026-01-28",{"date":143,"type":40},"2026-02-05",{"date":145,"type":21},"2026-07",{"date":147,"type":21},"2032-08-31",{"name":149,"class":47},"Robbie Majzner",2,{"id":152,"slug":153,"hasResults":11,"nctId":154,"briefTitle":155,"officialTitle":156,"acronym":4,"eligibilityCriteria":157,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":158,"targetDuration":4,"studyType":22,"phases":159,"briefSummary":160,"conditions":161,"keywords":170,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":180,"lastUpdatePostDateStruct":181,"startDateStruct":183,"completionDateStruct":185,"leadSponsor":187,"locationsCount":48},"100599631","phase-1-gpc2-car-t-cell-therapy-for-relapsed-or-refractory-medulloblastoma-in-children-and-young-adults-100599631","NCT07087002","GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults","Phase I Clinical Trial of GPC2 Chimeric Antigen Receptor T (GPC2-CAR T) Cells for Relapsed or Refractory Medulloblastoma in Children and Young Adults","Inclusion Criteria:\n\n1. Diagnosis: Histologically confirmed diagnosis of medulloblastoma or other primary CNS embryonal tumor according to 2021 CNS WHO Classification (5th edition)\n\n   * Other acceptable CNS embryonal tumors include:\n   * Embryonal Tumor with Multilayered Rosettes (ETMR)\n   * Pineoblastoma\n   * Atypical Teratoid\u002FRhabdoid Tumor (ATRT) of the CNS\n   * CNS neuroblastoma, FOXR2-activated\n   * CNS Embryonal Tumor NOS\n2. Recurrent\u002FRefractory Disease: History of relapsed and\u002For recurrent disease defined as tumor progression or recurrence following initial diagnosis and upfront treatment with curative intent, or failure to achieve disease control with standard curative-intent therapy.\n3. GPC2 Positive: H-score ≥ 100 by IHC staining performed on the (Prescreening Protocol IRB-78780, PI: Katherine Ryan, DO) at Stanford Clinical Anatomic Pathology Lab for GPC2 from a tumor sample any time since initial diagnosis.\n4. Evaluable Disease: Evaluable disease as per radiographic findings and\u002For positive cerebrospinal fluid cytology within 28 days of enrollment.\n5. Patients with VP shunts: Patients with pre-existing ventriculo-peritoneal (VP) shunt devices must have a programmable shunt device to enroll on this study. A VP shunt is not a requirement for this study.\n6. Prior therapy: No limit to the number of prior treatment regimens. Toxicities due to prior therapy must be stable or recovered to ≤ Grade 1 (except for clinically non-significant toxicities such as alopecia, nutritional support measures, electrolyte abnormalities, or those not impacting the investigator's ability to assess treatment emergent toxicities).\n\n   At time of enrollment, subjects are on track to meet the required therapy wash out period(s) prior to apheresis.\n\n   a. At least 6 weeks following craniospinal radiation therapy. i. At least 14 days wash-out needed following small volume radiotherapy (i.e., Stereotactic Radiosurgery (SRS)).\n\n   b. At least 21 days or 5 half-lives (whichever is shorter) must have elapsed since any prior systemic therapy, except for systemic inhibitory\u002Fstimulatory immune checkpoint therapy, which requires 5 half-lives.\n\n   c. At least 28 days following bevacizumab treatment. d. At least 30 days following any investigational drug. e. At least 12 weeks following systemic inhibitory or stimulatory immune checkpoint therapy.\n7. Age: ≥ 12 months to ≤ 30 years of age at time of enrollment The first 3 subjects treated with GPC2-CAR T cells must be ≥ 3 years old at time of infusion\n8. Performance Status: Subjects ≥ 16 years of age must have Karnofsky ≥ 60%. Subjects \\\u003C 16 years of age must have Lansky scale 60%; or ECOG performance status ≤ 2 (see Section 11.3).\n9. Normal Organ and Marrow Function \\[supportive care is allowed per institutional standards, i.e., filgrastim, transfusion\\]\n\n   1. Hemoglobin ≥ 8 g\u002FdL\n   2. Absolute Neutrophil Count (ANC) ≥ 1,000\u002FμL\n   3. Platelet count ≥ 75,000\u002FμL, with no platelet transfusion within 96 hours prior to enrollment\n   4. Absolute lymphocyte count (ALC) ≥ 150\u002FμL\n   5. PT\u002FINR, PTT ≤ 1.5 x ULN for age\n\n      Adequate renal, hepatic, cardiac, and pulmonary function defined as:\n   6. Serum creatinine \\\u003C 1.5 x ULN for age and gender, OR creatinine clearance or GFR (radioisotope or iothalamate) ≥ 70 mL\u002Fmin\u002F1.73 m2\n   7. Serum ALT or AST ≤ 3x ULN\n   8. Total bilirubin ≤ 1.5 mg\u002FdL, unless subject has Gilbert's Syndrome\n   9. Cardiac ejection fraction ≥ 45%\n   10. No evidence of physiologically significant pericardial effusion as determined by an ECHO\n   11. No clinically significant ECG findings\n   12. No clinically significant pleural effusion\n   13. Pulse oximetry ≥ 92% on room air, OR forced vital capacity ≥ 50% of predicted value\n10. Not Pregnant: Females of childbearing potential must have a negative pregnancy test.\n11. Contraception: Subjects of child-bearing or child-fathering potential must be willing to practice birth control from the time of enrollment on this study and for four (4) months after receiving the preparative regimen or for as long as CAR T cells are detectable in peripheral blood.\n12. Must provide informed consent. All subjects ≥ 18 years of age must be able to give informed consent. For subjects \\\u003C18 years old or adults with limited decision-making capacity, their legal authorized representative (LAR) (i.e., parent or guardian) must give informed consent. Pediatric subjects will be included in age-appropriate discussion and assent will be obtained for those \\> 7 years of age, when appropriate. If a minor becomes of age during participation of this study, he\u002Fshe will be asked to reconsent as an adult.\n\nExclusion Criteria\n\n1. Any patient with metastatic disease OUTSIDE the CNS.\n2. Unwilling or unable, in the investigator's judgement, to have a CSF reservoir (Ommaya or Rickham) placed. Does not apply to subjects who have a pre-existing device suitable for ICV delivery of CAR T cells and ICP monitoring.\n3. Clinical evidence of active\u002Fon-going significant increased intracranial pressure (i.e., impending herniation) or uncontrolled seizures.\n4. Prior receipt of a chimeric antigen receptor (CAR)-based therapy.\n5. Currently receiving anticoagulation therapy.\n6. Known history of infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive).\n\n   EXCEPTION: A history of hepatitis B or hepatitis C is permitted if the viral load is undetectable per quantitative PCR and\u002For nucleic acid testing.\n7. Pregnancy or breastfeeding in a postpartum female.\n8. Known sensitivity or allergy to any agents\u002Freagents used in this study.\n9. History of prior other malignancy. EXCEPTION: Previously diagnosed and definitively treated more than 5 years prior to enrollment or whose prognosis is deemed good enough to not warrant surveillance.\n10. Primary immunodeficiency or history of autoimmune disease (e.g., Crohn's, rheumatoid arthritis, systemic lupus) resulting in end organ injury or requiring systemic immunosuppression\u002Fsystemic disease modifying agents within the last 2 years.\n11. History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 12 months of enrollment.\n12. Significant medical diseases or poorly controlled conditions that, in the judgement of the investigator, put the subject at an unacceptable risk of complications, including but not limited to: uncontrolled diabetes mellitus, chronic obstructive pulmonary disease, pulmonary fibrosis, clinically significant inflammatory disorders, immunodeficiency (e.g., HIV infection), immunocompromised for reasons other than malignancy (e.g., chronic corticosteroid therapy or other immunosuppressive therapy), renal failure including patients requiring dialysis, or clinically significant liver dysfunction.\n13. In the Investigator's judgment, the subject or parents\u002Fcaregivers (as required) will not be able to comply with the study procedures outlined in the study protocol including follow-up visits.",{"count":96,"type":21},[24],"This is a single-site, open-label Phase 1 clinical trial evaluating the feasibility, safety, and preliminary activity of autologous GPC2-targeted chimeric antigen receptor (CAR) T cells administered via intracerebroventricular (ICV) infusion in children and young adults with relapsed or refractory medulloblastoma or other eligible Central Nervous System (CNS) embryonal tumors.",[132,162,163,164,165,166,30,167,168,169],"Central Nervous System Embryonal Tumor","Refractory Medulloblastoma","Recurrent Medulloblastoma","Pediatric Brain Tumor","Embryonal Tumor With Multilayered Rosettes (ETMR)","Atypical Teratoid\u002FRhabdoid Tumor (ATRT) of the CNS","CNS Neuroblastoma","FOXR2-activated",[171,172,173,174,175,132,176,177,178,30,167,179,169],"GPC2-CAR T cells","Chimeric Antigen Receptor T cells","Intracerebroventricular CAR T","Pediatric CNS tumors","Immunotherapy","Refractory brain tumors","T cell therapy","Embryonal Tumor with Multilayered Rosettes (ETMR)","CNS neuroblastoma","2026-01-26",{"date":182,"type":40},"2026-01-27",{"date":184,"type":40},"2025-08-28",{"date":186,"type":21},"2027-08",{"name":188,"class":47},"Stanford University",{"id":190,"slug":191,"hasResults":11,"nctId":192,"briefTitle":193,"officialTitle":194,"acronym":4,"eligibilityCriteria":195,"healthyVolunteers":196,"sex":16,"minAge":197,"maxAge":198,"enrollmentInfo":199,"targetDuration":201,"studyType":202,"phases":4,"briefSummary":203,"conditions":204,"keywords":222,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":242,"lastUpdatePostDateStruct":243,"startDateStruct":245,"completionDateStruct":247,"leadSponsor":249,"locationsCount":48},"100315102","international-ppbdicer1-registry-100315102","NCT03382158","International PPB\u002FDICER1 Registry","International Pleuropulmonary Blastoma\u002FDICER1 Registry (for PPB, DICER1 and Associated Conditions)","Inclusion Criteria:\n\n1. Known or suspected PPB or related thoracic tumor\n2. Known or suspected sex-cord stromal tumor including Sertoli-Leydig cell tumor and gynandroblastoma (males or females)\n3. Other known or suspected DICER1-related condition including ovarian sarcoma, cystic nephroma, renal sarcoma, pineoblastoma, pituitary blastoma, nasal chondromesenchymal hamartoma, ciliary body medulloepithelioma and others\n4. Individuals with known or suspected DICER1 pathogenic variation regardless of whether they have an established DICER1-associated condition\n5. Informed consent by patient\u002F or parent\u002Fguardian (also, where appropriate: assent and HIPAA consent)\n\nExclusion criteria:\n\nAbsence of appropriate consent for Registry participation",true,"0 Minutes","100 Years",{"count":200,"type":21},3400,"10 Years","OBSERVATIONAL","Pleuropulmonary blastoma (PPB) is a rare malignant neoplasm of the lung presenting in early childhood. Type I PPB is a purely cystic lesion, Type II is a partially cystic, partially solid tumor, Type III is a completely solid tumor. Treatment of children with PPB is at the discretion of the treating institution. This study builds off of the 2009 study and will also seek to enroll individuals with DICER1-associated conditions, some of whom may present only with the DICER1 gene mutation, which will help the Registry understand how these tumors and conditions develop, their clinical course and the most effective treatments.",[205,206,207,208,209,30,210,211,212,213,68,214,215,216,217,218,219,220,221],"Pleuropulmonary Blastoma","Sertoli-Leydig Cell Tumor","DICER1 Syndrome","Cystic Nephroma","Wilms Tumor","Renal Sarcoma","Nodular Hyperplasia of Thyroid","Nasal Chondromesenchymal Hamartoma","Ciliary Body Medulloepithelioma","Pituitary Cancer","Embryonal Rhabdomyosarcoma","Ovarian Sarcoma","Gynandroblastoma","Thyroid Carcinoma","Embryonal Rhabdomyosarcoma of Vagina (Diagnosis)","Embryonal Rhabdomyosarcoma of Uterus (Diagnosis)","Embryonal Rhabdomyosarcoma of Cervix",[223,224,225,226,206,208,227,228,229,209,30,210,230,211,231,218,212,232,213,233,68,214,215,234,216,217,235,236,237,238,239,240,241],"pleuropulmonary blastoma","PPB","DICER1","SLCT","CN","DICER1 mutation","DICER1 syndrome","ASK","Thyroid Nodules","NCMH","CBME","ERMS","Peritoneal PPB","pPPB","multinodular goiter","PPB Type I","PPB Type II","PPB Type III","PPB Type Ir","2025-01-28",{"date":244,"type":40},"2025-01-30",{"date":246,"type":40},"2016-12-06",{"date":248,"type":21},"2035-12-06",{"name":250,"class":47},"Children's Hospitals and Clinics of Minnesota"]