[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Porteus, Matthew, MD\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":68},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,45],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100422685","phase-1-depleted-donor-stem-cell-transplant-in-children-and-adults-with-fanconi-anemia-after-being-conditioned-with-a-regimen-containing-briquilimab-100422685",false,"NCT04784052","Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab","TCRαβ+ T-cell\u002FCD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 (Briquilimab) to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi Anemia","Inclusion Criteria:\n\nAll patients must have:\n\n1. Fanconi Anemia diagnosis as demonstrated by abnormal chromosome breakage studies with increased sensitivity to mitomycin-C (MMC) or diepoxybutane (DEB) and at least one mutation in a known Fanconi-associated gene\n2. Bone marrow failure (defined by reduction in at least one cell line on two separate occasions at least one month apart (e.g., platelet count of \\\u003C100,000 per cubic millimeter, hemoglobin \\\u003C9 gm\u002Fdl and\u002For absolute neutrophil count (ANC) of \\\u003C1000\u002Fmm)\n3. Age of ≥2 years\n4. Consenting ≥5\u002F10 HLA-matched related or unrelated donor available for apheresis\n5. Organ function defined as:\n\n   1. Serum Creatinine \\\u003C2.0 mg\u002FdL and corrected creatinine clearance\u002Fcystatin cL \\>60 mL\u002Fmin\u002F1.73m\\^2 without dialysis\n   2. Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and diffusing capacity of the lung for carbon monoxide (DLCO) corrected for hemoglobin and volume, \\>50% predicted by pulmonary function tests (PFTs)\n   3. For patients unable to cooperate for PFTs, criteria are no evidence of dyspnea at rest, no exercise intolerance, and no requirement for supplemental oxygen with spO2 \\>93%\n   4. Shortening fraction of ≥29% or ejection fraction of ≥45% by echocardiogram\n   5. Serum total bilirubin of \\\u003C4 x ULN\n   6. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\\u003C 5 x ULN\n   7. Prothrombin time international normalized ratio (PT INR) and partial thromboplastin time (PTT) \\\u003C1.5 x ULN\n6. Life expectancy of at least 2 years\n7. Patients of childbearing potential must be willing to use an effective contraceptive method for the duration of the peri-transplant conditioning through hematopoietic recovery\n8. Patients and\u002For parents or legal guardians must be able to provide written informed consent and authorize use and disclosure of personal health information in accordance with Health Insurance Portability and Accountability Act\n\nExclusion Criteria:\n\n1. Patients with available and consenting 10\u002F10 HLA-identical sibling donor for apheresis\n2. Patients with any acute or uncontrolled infections at the time of enrollment, including bacterial, fungal or viral\n3. Patients who are seropositive for HIV-I\u002FII or HTLV-I\u002FII.\n4. Patients receiving any other investigational agents or other biological, chemotherapy, or radiation therapy within 14 days of enrollment\n5. Patients with any active malignancies, myelodysplastic syndrome or other concerns for high-risk bone marrow disease\n6. Patients who received androgens in last 3 months\n7. Pregnant or lactating women\n8. Women who are nursing and do not wish to discontinue breastfeeding\n9. Lansky\u002FKarnofsky performance score \\\u003C50%.\n10. Any other medical condition or history that, in the opinion of the Principal Investigator, could pose a significant safety risk to the participant or jeopardize the integrity of the study\n11. Patients who, in the opinion of the Principal Investigator, may not be able to comply with the safety monitoring requirements of the study","ALL","2 Years",{"count":19,"type":20},18,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method).\n\nParticipants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.",[27],"Fanconi Anemia",[29,30,31],"Cell Transplants","Grafts","Stem Cells","RECRUITING","2026-01-23",{"date":35,"type":36},"2026-01-27","ACTUAL",{"date":38,"type":36},"2021-12-07",{"date":40,"type":20},"2028-12",{"name":42,"class":43},"Porteus, Matthew, MD","OTHER",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":52,"maxAge":53,"enrollmentInfo":54,"targetDuration":4,"studyType":21,"phases":56,"briefSummary":57,"conditions":58,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":44},"100411702","phase-1-stem-cell-transplant-from-donors-after-alpha-beta-cell-depletion-in-children-and-adults-with-t-allo10-cells-addback-100411702","NCT04640987","Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Adults With T-allo10 Cells Addback","Phase 1\u002F1b Study of T-allo10 Infusion After HLA-Partially Matched Related or Unrelated TCR αβ+ T-cell\u002F CD19+ B-cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation (αβ Depleted-HSCT) in Children and Young Adults Affected by Hematologic Malignancies","Inclusion Criteria prior to enrollment:\n\n* 1\\. Age \\> 1 months (with minimum weight of 10 Kg) and \\\u003C 45 years.\n* 2\\. Patients deemed eligible for allogeneic HSCT under the originating study, NCT 04249830\n* 3\\. Patients with life-threatening hematological malignancies for which HSCT has been recommended:\n\n  1. High-risk ALL in 1st CR, ALL in 2nd or subsequent CR;\n  2. High-risk AML in 1st CR, AML in 2nd or subsequent CR;\n  3. Myelodysplastic syndrome;\n  4. JMML (Juvenile myelomonocytic leukemia);\n  5. Non-Hodgkin lymphomas in 2nd or subsequent CR;\n  6. Other hematologic malignancies eligible for stem cell transplantation per institutional standard.\n* 4\\. All subjects ≥ 18 years of age must be able to give informed consent, or adults lacking capacity to consent must have a LAR available to provide consent. For subjects \\\u003C18 years old their LAR (i.e. parent or guardian) must give informed consent. Pediatric subjects will be included in age appropriate discussion and verbal assent will be obtained for those \\> 7 years of age, when appropriate.\n\nInclusion criteria prior to T-allo10 infusion:\n\n1. Patient already received αβdepleted-HSCT and has myeloid engraftment.\n2. Absence of active grade II aGvHD requiring \\>0.5 mg\u002FKg of steroids or any diagnosis of grade III\u002FIVaGvHD.\n\nExclusion Criteria prior to MNC collection for Tallo-10 manufacturing.:\n\n1. Not eligible to receive HSCT on NCT04249830\n2. Received another investigational agent within 30 days of enrollment.\n3. Pregnancy (positive serum or urine beta-HCG) within 7 days of MNC donation.\n4. Patient or donor is not willing or able to undergo an additional non-mobilized apheresis for collection of MNC prior to donation of cells for participation in NCT04249830.","1 Month","45 Years",{"count":55,"type":20},22,[23],"The purpose of this study is to determine the safety of a cell therapy, T-allo10, after αβdepleted-HSCT in the hopes that it will boost the adaptive immune reconstitution of the patient while sparing the risk of developing severe Graft-versus-Host Disease (GvHD).\n\nThe primary objective of Phase 1a is to determine the recommended Phase 2 dose (RP2D) administered after infusion of αβdepleted-HSCT in children and young adults with hematologic malignancies.\n\nA Phase 1b extension will occur after dose escalation, enrolling at the RP2D for the T-allo10 cells determined in the Phase 1 portion to evaluate the safety and efficacy of infusion of T-allo10 after receipt of αβdepleted-HSCT. Additionally, Phase 1b aims to explore improvements in immune reconstitution.\n\nAll participants on this study must be enrolled on another study: NCT04249830",[59],"Hematologic Diseases","2026-01-06",{"date":62,"type":36},"2026-01-08",{"date":64,"type":36},"2021-02-10",{"date":66,"type":20},"2029-03",{"name":42,"class":43},""]