[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100624357":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":28,"locations":37,"responsibleParty":58,"collaborators":62,"id":64,"slug":65,"hasResults":66,"nctId":67,"briefTitle":68,"officialTitle":69,"acronym":20,"eligibilityCriteria":70,"healthyVolunteers":66,"sex":71,"minAge":20,"maxAge":20,"enrollmentInfo":72,"targetDuration":20,"studyType":75,"phases":76,"briefSummary":79,"conditions":80,"keywords":89,"overallStatus":96,"whyStopped":20,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":106},{"fullName":5,"class":6},"Stanford University","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Intervention - in utero hematopoietic stem cell transplantation","EXPERIMENTAL","Single dose in utero hematopoietic stem cell transplantation (IUHSCT) in fetuses with Fanconi anemia during 19 - 28 weeks gestation. The cellular product is: Semi-allogeneic, Related, Maternal Bone Marrow-Derived, Miltenyi CliniMACS Plus enriched CD34+ hematopoietic stem cells administered in utero at a dose of 1 x 10\\^7-10\\^9 cells\u002Fkg fetal weight with equal to or less than 1% CD3+ T cells (equivalent to 10\\^5-10\\^7 T cells\u002Fkg fetal weight) in a final volume of 2-5ml suspended in 5% human serum albumin in Normosol buffer (Hospira, Inc.).",[13],"Biological: IUHSCT for FA-affected fetuses",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"BIOLOGICAL","IUHSCT for FA-affected fetuses","Single-dose IUHSCT Administration of Semi-allogeneic, Related, Maternal Bone Marrow-Derived, Miltenyi CliniMACS Plus Enriched CD34+ Hematopoietic Stem Cells Administered in Utero via fetal injection during 19 - 28 weeks gestation.",[9],null,[22,25],{"name":23,"affiliation":5,"role":24},"Yair Blumenfeld, MD","PRINCIPAL_INVESTIGATOR",{"name":26,"affiliation":27,"role":24},"Tippi MacKenzie, MD","University of California, San Francisco",[29,34],{"name":30,"role":31,"phone":32,"phoneExt":20,"email":33},"Agnieszka Czechowicz, MD, PhD","CONTACT","650-497-2218","bmf@stanfordchildrens.org",{"name":23,"role":31,"phone":35,"phoneExt":20,"email":36},"650-725-5720","mfmresearch@stanford.edu",[38,50],{"facility":27,"status":20,"city":39,"state":40,"zip":41,"country":42,"countryCode":43,"cosmosGeoPoint":44,"geoPoint":49,"contacts":20},"San Francisco","California","94143","United States","US",{"type":45,"coordinates":46},"Point",[47,48],-122.41942,37.77493,{"lat":48,"lon":47},{"facility":5,"status":20,"city":51,"state":40,"zip":52,"country":42,"countryCode":43,"cosmosGeoPoint":53,"geoPoint":57,"contacts":20},"Stanford","94305",{"type":45,"coordinates":54},[55,56],-122.16608,37.42411,{"lat":56,"lon":55},{"type":59,"investigatorFullName":60,"investigatorTitle":61,"investigatorAffiliation":5,"oldNameTitle":20,"oldOrganization":20},"SPONSOR_INVESTIGATOR","Agnieszka Czechowicz","Assistant Professor of Pediatrics",[63],{"name":27,"class":6},"100624357","phase-1-prenatal-transplantation-for-fetuses-with-fanconi-anemia-100624357",false,"NCT07408583","Prenatal Transplantation for Fetuses With Fanconi Anemia","A Phase I\u002FII, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fanconi Anemia in Affected Fetuses","Inclusion Criteria:\n\n* Male or female fetuses from 19\\^0\u002F7 - 28\\^0\u002F7 weeks gestational age at time of transplant.\n* Diagnosed with FA by either chorionic villus sampling (CVS), or amniocentesis, or cordocentesis with abnormal fetal chromosomal breakage studies and\u002For FANC gene mutations when combined with at least one of the following: 1) abnormal chromosomal breakage result consistent with an FA diagnosis, 2) family history of a 1st degree relative with confirmed FA, or 3) congenital anomalies consistent with the diagnosis of FA on fetal ultrasound.\n* Parents must consent to fetal autopsy in the event of a fetal demise.\n* Adequate bone marrow harvest from maternal participant is a condition for inclusion.\n\nExclusion Criteria:\n\n* Fetal Participant Exclusion Criteria: Major anatomic or genetic anomalies that contributes a significant morbidity or mortality risk, and\u002For echocardiogram or ultrasound findings that indicate a high risk of fetal demise after fetal intervention. Fetuses with a normal chromosomal breakage study that determines they are likely FA negative.\n* Maternal Subject Exclusion Criteria: Maternal participants will be excluded if they have one or more morbidities that would preclude bone marrow harvest and fetal intervention including, but not limited to, morbid obesity with a body mass index greater than 40, significant maternal cardiac disease, mirror syndrome, clinically symptomatic maternal anemia, Preterm premature rupture of membranes (PPROM), Active Preterm labor (PTL), opioid use disorder, current use of anticoagulants.","ALL",{"count":73,"type":74},12,"ESTIMATED","INTERVENTIONAL",[77,78],"PHASE1","PHASE2","The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.",[81,82,83,84,85,86,87,88],"Fanconi Anemia","Anemia, Hypoplastic, Congenital","Congenital Bone Marrow Failure Syndromes","Bone Marrow Failure Disorders","Genetic Diseases, Inborn","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","DNA Repair-Deficiency Disorders","Cancer Predisposition Syndrome",[90,91,92,93,94,95],"cell transplants","grafts","stem cells","bone marrow","Fanconi anemia","prenatal","NOT_YET_RECRUITING","2026-06-12",{"date":99,"type":100},"2026-06-16","ACTUAL",{"date":102,"type":74},"2028-01",{"date":104,"type":74},"2033-07",{"name":60,"class":6},2]