[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Stephan Grupp MD PhD\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":188},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,47,69,90,113,138,164],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100628692","phase-1-cart123-cells-with-or-without-ruxolitinib-in-relapsedrefractory-acute-myeloid-leukemia-100628692",false,"NCT07464951","CART123 Cells With or Without Ruxolitinib in Relapsed\u002FRefractory Acute Myeloid Leukemia","Phase 1 Trial of Autologous CD123-Directed CAR T-Cells (CART123) as Monotherapy or in Combination With Ruxolitinib in Relapsed\u002FRefractory Acute Myeloid Leukemia","CART123","Inclusion Criteria:\n\n* 1\\. Age at time of consent: Cohort A: 0-29 years. Cohort B: 1-29 years (Note: the first subject at each dose level of Cohort B must be ≥12 years old)\n* 2\\. Subjects with AML in second or greater relapse, post-transplant relapse, or with chemotherapy-refractory disease. Specifically:\n\n  1. Second or greater relapse defined as flow cytometric confirmation of myeloid leukemia of at least 0.1% after second documented complete remission; OR\n  2. Any detectable disease post-allogeneic transplant with flow cytometric confirmation of myeloid leukemia of at least 0.1%; OR\n  3. Refractory disease, defined as: Persistent bone marrow involvement with \\>5% blasts after two courses of induction chemotherapy for patients at initial presentation, \\>5% bone marrow blasts after one course of induction chemotherapy for patients who have relapsed after previously achieving a CR, and \\>5% bone marrow blasts after one course of AML-directed chemotherapy for those with myeloid lineage switch.\n* 3\\. Subjects must have an identified stem cell donor with the ability to proceed rapidly to transplant following CART123 treatment if indicated.\n* 4\\. Adequate organ function defined as:\n\n  1. Serum creatinine based on age\u002Fgender.\n  2. Adequate liver function: ALT ≤ 500 U\u002FL, Bilirubin ≤3x the upper limit of normal, and ALT and\u002For bilirubin results that exceed this range are acceptable if, in the opinion of the physician-investigator (or as confirmed by liver biopsy), the abnormalities are directly related to AML infiltration of the liver.\n  3. Must have a minimum level of pulmonary reserve defined as ≤Grade 1 dyspnea and \\\u003CGrade 3 hypoxia; DLCO ≥ 40% (corrected for anemia if necessary) if PFTs are clinically appropriate as determined by the treating investigator.\n  4. Left Ventricular Shortening Fraction (LVSF) ≥ 28% or Ejection Fraction (LVEF) ≥ 45% confirmed by echocardiogram or another scan.\n* 5\\. Adequate performance status defined as Lansky or Karnofsky performance score ≥ 50.\n* 6\\. Subjects of reproductive potential must agree to use acceptable birth control methods.\n\nExclusion Criteria:\n\n* 1\\. Active hepatitis B or active hepatitis C\n* 2\\. HIV infection\n* 3\\. Active acute or chronic GVHD requiring systemic therapy\n* 4\\. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n* 5\\. CNS disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.\n* 6\\. Pregnant or nursing (lactating) subjects.\n* 7\\. Uncontrolled active infection","ALL","0 Years","29 Years",{"count":21,"type":22},30,"ESTIMATED","INTERVENTIONAL",[25],"PHASE1","This study is designed to evaluate the safety and effectiveness of CART123 cells either alone or when combined with ruxolitinib in pediatric and young adult subjects with relapsed or refractory AML. Subjects will be enrolled into one of two treatment cohorts: subjects who will receive CART123 alone (Cohort A) or subjects who will receive CART123 in combination with ruxolitinib (Cohort B).",[28],"Acute Myeloid Leukemia (AML)",[30,31,15,32,33],"CART","AML","Leukemia","Ruxolitinib","RECRUITING","2026-05-22",{"date":37,"type":38},"2026-05-27","ACTUAL",{"date":40,"type":38},"2026-05-14",{"date":42,"type":22},"2030-05-14",{"name":44,"class":45},"Stephan Grupp MD PhD","OTHER",1,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":54,"targetDuration":4,"studyType":23,"phases":56,"briefSummary":58,"conditions":59,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":62,"startDateStruct":64,"completionDateStruct":66,"leadSponsor":68,"locationsCount":46},"100476170","phase-1-autologous-hucart19-t-cells-manufactured-using-the-clinimacs-prodigy-platform-for-pediatric-b-all-hucart19-prodigy-100476170","NCT05480449","Autologous HuCART19 T Cells Manufactured Using the CliniMACS Prodigy Platform for Pediatric B-ALL (huCART19 Prodigy)","Phase 1\u002F2b Trial of Autologous Humanized CD19-Directed Chimeric Antigen Receptor T-Cells Manufactured Using the CliniMACS Prodigy Platform for the Treatment of Pediatric B Cell Acute Lymphoblastic Leukemia (B-ALL)","Inclusion Criteria:\n\n1. Signed Informed Informed Consent\n2. Subjects with documented CD19+ ALL or Lly:\n\n   a. Cohort A: Subjects with relapsed or refractory ALL or Lly who have not previously received CAR T-cell Therapy: i. 2nd or greater relapse (marrow or extramedullary) OR ii. Any relapse after allogeneic HSCT and ≥4 months from HSCT at enrollment OR iii. Refractory disease defined as having not achieved an MRD-negative (by multiparameter flow cytometry) or CSF-negative CR after ≥2 chemotherapy regimens\u002Fcycles of frontline therapy, or 1 cycle of reinduction therapy for subjects in first relapse OR iv. Newly diagnosed NCI high-risk B-ALL with induction failure, defined as a M3 bone marrow (≥25%) blasts at the end of induction chemotherapy OR v. First bone marrow relapse of B-ALL at \\\u003C36 months after initial diagnosis OR vi. First or greater CNS relapse of B-ALL vii. Ineligible for allogeneic HSCT because of at least one of the following:\n\n1\\. Comorbid disease 2. Other contraindications to HSCT conditioning regimen 3. Lack of suitable donor 4. Prior HSCT 5. Declines HSCT as the therapeutic option after documented discussion, with expected outcomes, and the role of HSCT with a BMT physician not a part of the study team.\n\nb. Cohort B: Subjects with poor response to prior B cell directed engineered cell therapy, defined as any one of the following: i. Partial response or no response to prior cell therapy ii. CD19+ relapse after prior cell therapy, defined as bone marrow blasts \\> 0.01% by multiparameter flow cytometry or evidence of extramedullary disease iii. Demonstrated early (approximately 6 months from infusion) B cell recovery suggesting loss of engineered cells\n\n3\\. Subjects with prior or current history of CNS3 disease will be eligible if Central Nervous System (CNS) disease is responsive to therapy.\n\n4\\. Documentation of CD19 tumor expression in bone marrow, peripheral blood, cerebrospinal fluid (CSF), or tumor tissue by flow cytometry. If the subject has received CD19-directed therapy, flow cytometry should be obtained after this therapy to demonstrate CD19 expression.\n\n5\\. Age 0-29 years\n\n6\\. Adequate organ function.\n\na. Serum creatinine based on age\u002Fgender b. Adequate liver function: i. ALT within 5x ULN in the absence of ALL infiltration of the liver ii. Bilirubin ≤3x the upper limit of normal iii. ALT and\u002For bilirubin results that exceed this range are acceptable if, in the opinion of the physician-investigator (or as confirmed by liver biopsy), the abnormalities are directly related to ALL infiltration of the liver.\n\nc. Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and \\\u003C Grade 3 hypoxia; DLCO ≥ 40% (corrected for anemia if necessary) if PFTs are clinically appropriate as determined by the investigator.\n\nd. Left Ventricular Shortening Fraction (LVSF) ≥28% or Ejection Fraction (LVEF) ≥45% confirmed by echocardiogram or another scan. In cases where quantitative assessment of LVSF\u002FLVEF is not possible, a statement by the cardiologist that the ECHO shows qualitatively normal ventricular function will suffice.\n\n7\\. Adequate performance status defined as Lanksy or Karnofsky performance score ≥50\n\n8\\. Subjects of reproductive potential must agree to use acceptable birth control methods.\n\nExclusion Criteria:\n\n1. Active hepatitis B or active hepatitis C\n2. HIV infection\n3. Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.\n4. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n5. CNS disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.\n6. Subjects who are pregnant or nursing.\n7. Uncontrolled active infection.\n8. History of seizure disorder that requires ongoing anti-epileptic therapy.\n9. If the subject has received previous CAR T cell therapies, history of grade 3 or higher ICANS following administration of a CAR T cell product.",{"count":55,"type":22},115,[25,57],"PHASE2","This study will determine the safety and efficacy of moving to a second-generation manufacturing process using the CliniMACS Prodigy platform to manufacture huCART19 cells for patients with B cell Acute Lymphoblastic Leukemia (B-ALL).",[60,61],"B Cell Acute Lymphoblastic Leukemia (B-ALL)","B Lineage Lymphoblastic Lymphoma",{"date":63,"type":38},"2026-05-26",{"date":65,"type":38},"2022-09-20",{"date":67,"type":22},"2029-09-20",{"name":44,"class":45},{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":4,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":19,"enrollmentInfo":76,"targetDuration":4,"studyType":23,"phases":78,"briefSummary":79,"conditions":80,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":85,"completionDateStruct":87,"leadSponsor":89,"locationsCount":46},"100491057","phase-1-co-administration-of-cart22-65s-and-hucart19-for-b-all-100491057","NCT05674175","Co-administration of CART22-65s and huCART19 for B-ALL","Use of Autologous Anti-CD22 CAR T Cells (CART22-65s) Co-administered With Humanized Anti-CD19 CAR T Cells (huCART19) in Children and Young Adults With Relapsed or Refractory B-ALL","Inclusion Criteria:\n\n1. Signed informed consent form\n2. Patients with documented CD19+ and\u002For CD22+ ALL\u002FLLy:\n\n   1. Cohort A: Patients with relapsed or refractory ALL\u002FLLy:\n   2. Cohort B: Patients with poor response to prior B cell directed engineered cell therapy\n3. Patients with prior or current history of Central Nervous System 3 disease will be eligible if Central Nervous System disease is responsive to therapy\n4. Documentation of CD19 and\u002For CD22 tumor expression in bone marrow, peripheral blood, Cerebrospinal fluid, or tumor tissue by flow cytometry at the time of last detectable disease. If the patient has experienced a relapse after CD19-directed and\u002For CD22-directed therapy, flow cytometry should be evaluated after this therapy to demonstrate CD19 and\u002For CD22 expression.\n5. Age 0-29 years\n6. Adequate organ function\n7. Adequate performance status defined as Lanksy or Karnofsky performance score ≥50.\n8. Subjects of reproductive potential must agree to use acceptable birth control methods.\n\nExclusion Criteria:\n\n1. Active hepatitis B or active hepatitis C\n2. HIV infection\n3. Active acute or chronic Graft Vs. Host Disease requiring systemic therapy\n4. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n5. Central nervous system disease that is progressive on therapy, or with Central nervous system parenchymal lesions that might increase the risk of central nervous system toxicity.\n6. Pregnant or nursing (lactating) women\n7. Uncontrolled active infection",{"count":77,"type":22},93,[25,57],"This study will evaluate the safety and efficacy of administering two CAR T cell products, huCART19 and CART22-65s, in children with advanced B cell Acute Lymphoblastic Leukemia (B-ALL).",[81,61],"B-cell Acute Lymphoblastic Leukemia","2026-03-24",{"date":84,"type":38},"2026-03-27",{"date":86,"type":38},"2023-01-25",{"date":88,"type":22},"2029-07-01",{"name":44,"class":45},{"id":91,"slug":92,"hasResults":11,"nctId":93,"briefTitle":94,"officialTitle":95,"acronym":4,"eligibilityCriteria":96,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":97,"targetDuration":4,"studyType":23,"phases":99,"briefSummary":100,"conditions":101,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":105,"lastUpdatePostDateStruct":106,"startDateStruct":108,"completionDateStruct":110,"leadSponsor":112,"locationsCount":46},"100383762","phase-2-orphan-indications-for-cd19-redirected-autologous-t-cells-100383762","NCT04276870","Orphan Indications for CD19 Redirected Autologous T Cells","CD19-Directed Chimeric Antigen Receptor CD19 Redirected Autologous T Cells (CART19) for Orphan Indications of Pediatric B Cell Acute Lymphoblastic Leukemia (B ALL)","Inclusion Criteria:\n\n1. Signed informed consent form must be obtained prior to any study procedure.\n2. Male and female patients with documented CD19+ B-ALL\n\n   a.Cohort A \\& B: Patients, regardless their response to initial or relapsed B ALL therapy, with the following characteristics: i.Cohort A: Subjects with confirmation of a hypodiploid karyotype (chromosome number fewer than 45) ii.Cohort B: Subjects with cytogenetic confirmation of the chromosomal translocation t(17;19) (Cohort B) b.Cohort C: Infants w\u002F newly diagnosed KMT2A rearranged B-ALL classified as very high risk by the following criteria: i.Age \\\u003C 3 months at diagnosis ii.Age \\\u003C 6 months and WBC \\> 300,000x109\u002FL at diagnosis or a poor prednisone response in induction iii.MRD positive \\> 0.01 (or PCR \\> 104) after 2 courses of standard infant ALL therapy.\n\n   c.Cohort D: Subjects in a first or greater CNS relapse, prior to therapy with cranial XRT or HSCT for the current relapse\n3. Documentation of CD19 tumor expression in bone marrow, peripheral blood, CSF, or tumor tissue.\n4. Age 0 to 29 years\n5. Adequate organ function defined as:\n\n   1. A serum creatinine based on age\u002Fgender as follows:\n\n      Maximum Serum Creatinine (mg\u002FdL) Age Male Female 0 to \\\u003C 2 years 0.6 0.6 2 to \\\u003C 6 years 0.8 0.8 6 to \\\u003C 10 years 1.0 1.0 10 to \\\u003C 13 years 1.2 1.2 13 to \\\u003C 16 years 1.5 1.4\n\n      ≥ 16 years 1.7 1.4\n   2. Adequate liver function:\n\n   i.ALT≤ 5 x ULN; ALT ii.Total bilirubin ≤ 3 x ULN iii.ALT and\u002For bilirubin results that exceed this range are acceptable if, in the opinion of the physician-investigator (or as confirmed by liver biopsy), the abnormalities are directly related to ALL infiltration of the liver.\n\n   c.Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and \\\u003C Grade 3 hypoxia; DLCO ≥ 40% (corrected for anemia) if PFTs are clinically appropriate as determined by the physician-investigator.\n\n   d.Left Ventricular Shortening Fraction (LVSF) ≥ 28%, or Left Ventricular Ejection Fraction (LVEF) ≥ 45% by echocardiogram. In cases where quanitative assessment of LVSF\u002FLVEF is not possible, a statement by the cardiologist that the ECHO shows qualititatively normal ventricular function wll suffice.\n6. Adequate performance status defined as Lansky or Karnofsky score ≥ 50\n7. Subjects of reproductive potential must agree to use acceptable birth control methods\n\nExclusion Criteria:\n\n1. For subjects with a CNS relapse, prior cranial XRT or BMT for the current relapse is an exclusion.\n2. Active hepatitis B or active hepatitis C.\n3. HIV Infection.\n4. Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.\n5. Concurrent use of systemic steroids at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n6. CNS3 disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.\n7. Pregnant or nursing (lactating) women.\n8. Uncontrolled active infection.",{"count":98,"type":22},133,[57],"This is an open-label, four-cohort, phase 2 study to determine the efficacy of CART19 in pediatric and young adult patientswith hypodiploid (Cohort A) or t(17;19) B-ALL (Cohort B), infants with very high risk KMT2A B-ALL (Cohort C), and in patients with central nervous system (CNS) relapse who did not receive cranial radiation (XRT) or bone marrow transplantation (BMT) (Cohort D).",[102,103,104],"Pediatric and Young Adult Patientswith Hypodiploid or t(17;19) B-ALL","Infants With Very High Risk KMT2A B-ALL","Patients With Central Nervous System Relapse Who Did Not Receive Cranial Radiation or Bone Marrow Transplantation","2026-03-04",{"date":107,"type":38},"2026-03-06",{"date":109,"type":38},"2020-03-12",{"date":111,"type":22},"2037-03-10",{"name":44,"class":45},{"id":114,"slug":115,"hasResults":11,"nctId":116,"briefTitle":117,"officialTitle":118,"acronym":119,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":121,"targetDuration":4,"studyType":23,"phases":123,"briefSummary":124,"conditions":125,"keywords":127,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":131,"startDateStruct":133,"completionDateStruct":135,"leadSponsor":137,"locationsCount":46},"100587899","phase-1-anti-cd7-car-t-cells-in-relapsedrefractory-t-cell-acute-lymphoblastic-leukemia-or-lymphoma-100587899","NCT06934382","Anti-CD7 CAR-T Cells in Relapsed\u002FRefractory T-Cell Acute Lymphoblastic Leukemia or Lymphoma","A Phase 1 Study of Allogeneic Anti-CD7 CAR-T Cells (BEAM-201) in Relapsed\u002FRefractory T-cell Acute Lymphoblastic Leukemia (T-ALL) or T-cell Lymphoblastic Lymphoma (T-LLy)","24CT015","Patients must meet all the following criteria to be eligible for enrollment into the study:\n\n1. Patients (ages ≥ 18 years) or parent\u002Flegal guardians (for patients ages \\\u003C 18 years) must provide signed, written informed consent according to local IRB and institutional requirements.\n2. Ages 0 to 29 years.\n3. T-ALL\u002FT-LLy in second or greater relapse, first relapse post-transplant, or chemotherapy-refractory disease. Specifically:\n\n   1. Second or greater relapse or post-transplant relapse, defined as:\n\n      * BM with ≥ 5% lymphoblasts by morphologic assessment or evidence of extramedullary disease after second documented CR; OR\n      * Flow cytometric confirmation of relapsed T-ALL of at least 0.1% after second CR documented to have been MRD negative \\\u003C 0.1%; OR\n      * Any detectable relapsed disease post-allogeneic HSCT with flow cytometric confirmation of T-ALL of at least 0.1%; OR\n      * Biopsy confirmed evidence of relapsed T-LLy after second CR; OR\n      * Any detectable disease post-allogeneic transplant with biopsy confirmed evidence of T-LLy\n   2. Refractory disease, defined as:\n\n      * Primary refractory T-ALL or T-LLy, defined as failure to achieve CR after induction chemotherapy, per investigator assessment and based on biopsy-or MRD-confirmed evidence of residual T-ALL or T-LLy; OR\n      * Relapsed, refractory disease, defined as \\> 0.1 % MRD or morphologic evidence of disease or evidence of residual T-LLy after 1 course of re-induction chemotherapy for patients who have relapsed after previously achieving a CR NOTE: Patients with mixed phenotype acute leukemia with T cell dominant phenotype may be enrolled if the aforementioned criteria are met.\n4. Documentation of CD7 expression on leukemic or T-LLy blasts (defined as at least 90% of blasts positive for CD7 by flow cytometry or immunohistochemistry).\n5. Patients with prior or current history of CNS3 disease will be eligible if CNS disease is responsive to therapy\n6. Eligible for myeloablative conditioning for and allogeneic HSCT based on the investigator's assessment with an available donor identified by a FACT accredited transplant center.\n7. Lansky Performance Status (ages \\\u003C 16 years at time of consent) or Karnofsky Performance Status (KPS) (ages ≥ 16 years at time of consent) score of ≥ 50.\n8. Patients of childbearing potential must have a negative urine or serum pregnancy test at screening.\n9. Adequate organ function defined as:\n\n   1. Adequate Serum creatinine based on age\u002Fgender\n   2. ALT ≤ 5x ULN in the absence of ALL infiltration of the liver\n   3. Bilirubin ≤ 3 × ULN for age Note: ALT and\u002For bilirubin results that exceed this range are acceptable if, in the opinion of the physician-investigator (or as confirmed by liver biopsy), the abnormalities are directly related to ALL infiltration of the liver.\n   4. Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and \\\u003CGrade 3 hypoxia; DLCO ≥40% (corrected for anemia if necessary) if PFTs are clinically appropriate as determined by the investigator.\n   5. Cardiac echocardiography (ECHO) with left ventricular shortening fraction (LVSF) ≥ 30% or left ventricular ejection fraction (LVEF) ≥ 50%. In cases where quantitative assessment of LVSF\u002FLVEF is not possible, a statement by the cardiologist that the ECHO shows qualitatively normal ventricular function will suffice\n10. Patients who are sexually active and of reproductive potential must agree to use an acceptable form of highly effective contraception from consent to 12 months after BEAM 201 infusion.\n\n4.2 Exclusion Criteria\n\nPatients who meet any of the following criteria will be disqualified from entering the study:\n\n1. Active hepatitis B or active hepatitis C\n2. Active HTLV infection\n3. HIV infection\n4. Uncontrolled, active bacterial, viral, or fungal infection.\n5. CNS disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.\n6. Clinically active CNS dysfunction or known history of irreversible central neurological toxicity related to prior antileukemic therapy.\n7. Receipt of prior CD7 targeted therapy.\n8. Radiation therapy within 2 weeks prior to completion of screening, other than prophylaxis for CNS disease.\n9. Acute GVHD that is grade ≥ 2 and requiring systemic immunosuppression (corticosteroids), or chronic GVHD that is mild, moderate, or severe and requiring systemic immunosuppression (corticosteroids). Grade 1 acute GVHD not requiring immunosuppression is allowable.\n10. Undergone HSCT within 90 days prior to completion of screening (or donor leukocyte infusion, if received within 30 days prior to completion of screening).\n11. Any other condition that would make the patient ineligible for HSCT as determined by the investigator.\n12. Known primary immunodeficiency or BM failure syndrome.\n13. Atrial fibrillation\u002Fflutter (not including isolated episodes that responded to medical management)\n14. Clinically significant pericardial effusion\n15. Myocardial infarction within the last 12 months\n16. QT interval corrected for heart rate \\> 480 msec\n17. Cardiac dysfunction NYHA (New York Heart Association) III or IV\n18. Patients with an autoimmune disorder requiring systemic immunosuppressive therapy that cannot be safely withheld for 3 months.\n\n    Concurrent use of systemic corticosteroids for diagnoses unrelated to T-ALL\u002FT-LLy is prohibited, with exception of physiologic corticosteroid replacement therapy treatment for adrenal insufficiency.\n19. Pregnant or breastfeeding",{"count":122,"type":22},33,[25],"This will be a Phase 1, open-label study to evaluate the safety and efficacy of BEAM-201 in patients with R\u002FR T-ALL or T-LLy. BEAM-201 is an allogeneic anti-CD7 CART therapy.",[126],"T-Cell Acute Lymphoblastic Leukemia\u002FLymphoma",[30,128,129],"T-cell leukemia","T-cell lymphoma","2025-12-24",{"date":132,"type":38},"2025-12-26",{"date":134,"type":38},"2025-04-29",{"date":136,"type":22},"2031-05-30",{"name":44,"class":45},{"id":139,"slug":140,"hasResults":11,"nctId":141,"briefTitle":142,"officialTitle":143,"acronym":144,"eligibilityCriteria":145,"healthyVolunteers":11,"sex":17,"minAge":146,"maxAge":4,"enrollmentInfo":147,"targetDuration":4,"studyType":23,"phases":149,"briefSummary":150,"conditions":151,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":157,"startDateStruct":159,"completionDateStruct":161,"leadSponsor":163,"locationsCount":46},"100489256","phase-1-gpc2-car-t-cells-for-relapsed-or-refractory-neuroblastoma-and-metastatic-retinoblastoma-100489256","NCT05650749","GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma","Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma","GPC2","Neuroblastoma Inclusion Criteria:\n\n1. Patients must be ≥ 1 year of age\n2. Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment. Patients who were initially considered low- or intermediate-risk, but then reclassified as high-risk are also eligible.\n3. Patients must have a previously histologically confirmed diagnosis of neuroblastoma:\n\n   1. That is recurrent\u002Frelapsed or refractory\u002Fpersistent according to INRC AND\n   2. For which standard curative measures do not exist or are no longer effective.\n   3. patients at first relapse are eligible as no known curative therapies exist for relapsed high-risk neuroblastoma.\n4. Patients must have evaluable or measurable disease at enrollment.\n5. In addition, patient must have experienced at least one of the following:\n\n   a. New disease site documented on at least one of the following: i. 123I-meta-iodobenzylguanidine (MIBG) or 18F-mFBG (meta-fluorobenzylguanidine) scan; OR ii. CT\u002FMRI; OR iii. FDG or Ga-68 Dotatate PET (in patients known to have MIBG non-avid tumor) and MRI findings consistent with tumor (i.e., bone lesions), OR iv. Biopsy confirmed neuroblastoma for any new or progressing lesion. b. Greater than 20% increase in a least one dimension of soft tissue mass documented by CT\u002FMRI and a minimum absolute increase of 5 mm in longest dimension in existing lesion(s). Previously irradiated lesions may be included.\n\n   c. Bone marrow biopsy shows progressive disease according to the revised INRC d. Stable persistent disease, such that response at the completion of upfront therapy or salvage therapy is less than partial response AND has a biopsy of at least one site showing viable neuroblastoma.\n\n   e. Responding persistent disease, defined as at least a partial response to frontline therapy (i.e., at least a partial response to frontline therapy but still has residual disease by MIBG scan, CT\u002FMRI, or bone marrow aspirations\u002Fbiopsies). Patients in this category are required to have histologic confirmation of viable neuroblastoma from at least one residual site (tumor seen on routine bone marrow morphology is sufficient).\n6. Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 60\n7. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age.\n8. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)\n9. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).\n10. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).\n11. Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.\n12. Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist.\n\nNeuroblastoma Exclusion Criteria:\n\n1. Patients with active hepatitis B or active hepatitis C.\n2. Patients with HIV infection.\n3. Patients with uncontrolled active infection.\n4. Patients with primary or acquired immunodeficiency disorder.\n5. Patients with a known hypersensitivity to DMSO.\n6. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n7. Patients with actively progressing CNS metastases, including parenchymal or leptomeningeal involvement. (Note: CNS imaging at screening is only required if the there is a clinical indication of suspected CNS metastasis)\n8. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and\u002For neurotoxicity.\n9. Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months prior to study entry or a history of myocarditis.\n10. Patients who have received any live vaccines within 30 days prior to enrollment.\n11. Patients who are pregnant or nursing (lactating).\n12. Patients who have a life expectancy \\\u003C 6 months at time of consent.\n\nRetinoblastoma Inclusion Criteria:\n\n1. Patient age ≥ 6 months.\n2. Patients must have metastatic retinoblastoma according to International\n\n   Retinoblastoma Staging System (IRSS) risk classification (4) at the time of study enrollment:\n\n   a. Retinoblastoma Cohort 1 (Extra-CNS metastasis) i. Stage IVa disease ii. Extra-CNS disease must be confirmed as retinoblastoma by histology (either at diagnosis or recurrence) iii. Measurable disease: Defined as \\> 1cm2 or biopsy-proven bone marrow disease iv. Prior treatment: Recurrent or refractory disease following treatment with COG ARET0321-like or equivalent regimen as part of upfront or recurrent therapy b. Retinoblastoma Cohort 2 (CNS disease) i. Stage IVb disease ii. Histologic confirmation is not required iii. CNS disease defined as measurable disease \\>1cm2, non-measurable, or CSF positivity alone c. Prior treatment: i. Stage IVb.1 and IVb.2: Recurrent after treatment with COG ARET0321-like or equivalent regimen as part of upfront or recurrent therapy i. Stage IVb.3: Prior treatment is not required (i.e., eligible at initial diagnosis or recurrence)\n3. Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 60\n4. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN .\n5. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)\n6. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).\n7. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).\n8. Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.\n9. Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist.\n\nRetinoblastoma Exclusion Criteria:\n\n1. Patients with active hepatitis B or active hepatitis C.\n2. Patients with HIV infection.\n3. Patients with uncontrolled active infection.\n4. Patients with primary or acquired immunodeficiency disorder.\n5. Patients with a known hypersensitivity to DMSO.\n6. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n7. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk to the subject.\n8. Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months prior to study entry or a history of myocarditis.\n9. Patients who have received any live vaccines within 30 days prior to enrollment.\n10. Patients who are pregnant or nursing (lactating).\n11. Patients who have a life expectancy \\\u003C 6 months at time of consent.\n12. Retinoblastoma Cohort 1 (Extra-CNS disease):\n\n    1. Concurrent CNS disease (they may be eligible for Retinoblastoma Cohort 2)\n    2. Stage III disease (orbital or lymph node regional extension without other hematogenous metastases).\n13. Retinoblastoma Cohort 2 (CNS disease):\n\n    1. \"Bulky\" disease (\\>5 cm in diameter) within or compressing the brainstem or thalamus. Note: Tumors touching the brainstem\u002Fthalamus without evidence of compression and\u002For tumors in other CNS locations do not have a maximal size criterion.\n    2. If evidence of clinically significant increased intracranial pressure at time of relapse, patient must demonstrate improvement by time of enrollment.","1 Year",{"count":148,"type":22},45,[25],"This is a first in human dose escalation trial to determine the safety of administering GPC2 CAR T cells in patients with advanced neuroblastoma or retinoblastoma.",[152,153,154,155,156],"Refractory Neuroblastoma","Relapsed Neuroblastoma","High-risk Neuroblastoma","Retinoblastoma","Metastatic Retinoblastoma",{"date":158,"type":38},"2025-12-29",{"date":160,"type":38},"2023-05-23",{"date":162,"type":22},"2030-01-30",{"name":44,"class":45},{"id":165,"slug":166,"hasResults":11,"nctId":167,"briefTitle":168,"officialTitle":169,"acronym":4,"eligibilityCriteria":170,"healthyVolunteers":11,"sex":17,"minAge":171,"maxAge":172,"enrollmentInfo":173,"targetDuration":4,"studyType":23,"phases":175,"briefSummary":176,"conditions":177,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":180,"lastUpdatePostDateStruct":181,"startDateStruct":183,"completionDateStruct":185,"leadSponsor":187,"locationsCount":46},"100441512","phase-2-autologous-stem-cell-transplant-asct-for-autoimmune-diseases-100441512","NCT05029336","Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases","Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases","Inclusion Criteria:\n\n1. Age 8 ≤ 25 years at time of enrollment.\n2. Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria\n3. Adequate organ function status\n4. No active, untreated infections.\n\nExclusion Criteria:\n\n1. Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant\n2. Pregnancy\n3. Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)\n4. Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy","8 Years","25 Years",{"count":174,"type":22},20,[57],"A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for \"immune reset.\" This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach.",[178,179],"Systemic Lupus Erythematosus","Systemic Sclerosis","2025-10-09",{"date":182,"type":38},"2025-10-14",{"date":184,"type":22},"2026-03",{"date":186,"type":22},"2031-05",{"name":44,"class":45},""]