[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100422685":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":59,"centralContacts":30,"locations":63,"responsibleParty":82,"collaborators":30,"id":85,"slug":86,"hasResults":87,"nctId":88,"briefTitle":89,"officialTitle":90,"acronym":30,"eligibilityCriteria":91,"healthyVolunteers":87,"sex":92,"minAge":93,"maxAge":30,"enrollmentInfo":94,"targetDuration":30,"studyType":97,"phases":98,"briefSummary":101,"conditions":102,"keywords":104,"overallStatus":65,"whyStopped":30,"lastUpdateSubmitDate":108,"lastUpdatePostDateStruct":109,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":118},{"fullName":5,"class":6},"Stanford University","OTHER",[8,20],{"label":9,"type":10,"description":11,"interventionNames":12},"Depleted Stem Cell Transplant with JSP-191 Conditioning","EXPERIMENTAL","Participants will receive an infusion of donor stem cells which have been depleted of αβ+T cells using the CliniMACS System device. Before the stem cell transplant, they will receive a reduced-intensity preparative regimen containing JSP191 in combination with rATG, cyclophosphamide, fludarabine and rituximab.",[13,14,15,16,17,18,19],"Drug: JSP191","Device: CliniMACS Prodigy System","Biological: Depleted Stem Cell Transplant","Biological: Rabbit Anti-Thymoglobulin (rATG)","Drug: Cyclophosphamide","Drug: Fludarabine","Drug: Rituximab",{"label":21,"type":10,"description":22,"interventionNames":23},"Depleted Stem Cell Transplant without JSP-191 Conditioning","Participants will receive an infusion of donor stem cells which have been depleted of αβ+T cells using the CliniMACS System device. Before the stem cell transplant, they will receive a reduced-intensity preparative regimen containing rATG, cyclophosphamide, fludarabine and rituximab.",[14,15,16,17,18,19],[25,31,36,41,45,51,55],{"type":26,"name":27,"description":28,"armGroupLabels":29,"otherNames":30},"DRUG","JSP191","Participants will receive a single IV dose at start of conditioning",[9],null,{"type":32,"name":33,"description":34,"armGroupLabels":35,"otherNames":30},"DEVICE","CliniMACS Prodigy System","The device used to remove the αβ+T cells from donor stem cell transplant before being given to the recipient",[9,21],{"type":37,"name":38,"description":39,"armGroupLabels":40,"otherNames":30},"BIOLOGICAL","Depleted Stem Cell Transplant","TCRαβ+ T-cell\u002FCD19+ B-cell depleted hematopoietic cells will be administered by IV after completion of conditioning regimen.",[9,21],{"type":37,"name":42,"description":43,"armGroupLabels":44,"otherNames":30},"Rabbit Anti-Thymoglobulin (rATG)","3 consecutive daily doses of rATG will be given by IV during conditioning",[9,21],{"type":26,"name":46,"description":47,"armGroupLabels":48,"otherNames":49},"Cyclophosphamide","4 consecutive daily doses of cyclophosphamid will be given by IV during conditioning",[9,21],[50],"Cytoxan",{"type":26,"name":52,"description":53,"armGroupLabels":54,"otherNames":30},"Fludarabine","4 consecutive daily doses of fludarabine will be given by IV during conditioning",[9,21],{"type":26,"name":56,"description":57,"armGroupLabels":58,"otherNames":30},"Rituximab","1 dose of rituximab will be given at the end of conditioning",[9,21],[60],{"name":61,"affiliation":5,"role":62},"Rajni Agarwal, MD","PRINCIPAL_INVESTIGATOR",[64],{"facility":5,"status":65,"city":66,"state":67,"zip":68,"country":69,"countryCode":70,"cosmosGeoPoint":71,"geoPoint":76,"contacts":77},"RECRUITING","Stanford","California","94305","United States","US",{"type":72,"coordinates":73},"Point",[74,75],-122.16608,37.42411,{"lat":75,"lon":74},[78],{"name":61,"role":79,"phone":80,"phoneExt":30,"email":81},"CONTACT","650-725-9250","scgt_clinical_trials_office@lists.stanford.edu",{"type":62,"investigatorFullName":83,"investigatorTitle":84,"investigatorAffiliation":5,"oldNameTitle":30,"oldOrganization":30},"Rajni Agarwal","Associate Professor of Pediatrics","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":95,"type":96},18,"ESTIMATED","INTERVENTIONAL",[99,100],"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.",[103],"Fanconi Anemia",[105,106,107],"Cell Transplants","Grafts","Stem Cells","2026-01-23",{"date":110,"type":111},"2026-01-27","ACTUAL",{"date":113,"type":111},"2021-12-07",{"date":115,"type":96},"2028-12",{"name":117,"class":6},"Porteus, Matthew, MD",1]