[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"congenital-hereditary-and-neonatal-diseases-and-abnormalities\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:congenital-hereditary-and-neonatal-diseases-and-abnormalities":31},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,53],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":24,"conditions":25,"keywords":34,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":5},"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":18,"type":19},12,"ESTIMATED","INTERVENTIONAL",[22,23],"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.",[26,27,28,29,30,31,32,33],"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",[35,36,37,38,39,40],"cell transplants","grafts","stem cells","bone marrow","Fanconi anemia","prenatal","NOT_YET_RECRUITING","2026-06-12",{"date":44,"type":45},"2026-06-16","ACTUAL",{"date":47,"type":19},"2028-01",{"date":49,"type":19},"2033-07",{"name":51,"class":52},"Agnieszka Czechowicz","OTHER",{"id":54,"slug":55,"hasResults":11,"nctId":56,"briefTitle":57,"officialTitle":58,"acronym":59,"eligibilityCriteria":60,"healthyVolunteers":11,"sex":61,"minAge":62,"maxAge":63,"enrollmentInfo":64,"targetDuration":4,"studyType":20,"phases":66,"briefSummary":68,"conditions":69,"keywords":82,"overallStatus":105,"whyStopped":4,"lastUpdateSubmitDate":106,"lastUpdatePostDateStruct":107,"startDateStruct":109,"completionDateStruct":111,"leadSponsor":113,"locationsCount":115},"100638348","phase-3-efficacy-safety-and-tolerability-of-zeleciment-rostudirsen-dyne-251-administered-intravenously-every-4-weeks-in-ambulatory-participants-with-duchenne-muscular-dystrophy-forzetto-100638348","NCT07608432","Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping","FORZETTO","Inclusion Criteria:\n\n* Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .\n* Rise From Floor (RFF) time must be \\\u003C 10 seconds for both screening assessments .\n* Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)\n\nExclusion Criteria:\n\n* Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization\n* Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization\n* Any change in prophylaxis\u002Ftreatment for congestive heart failure (CHF) within 12 weeks prior to randomization\n* Receipt of eteplirsen within 1 week prior to randomization\n* Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization\n* Receipt of givinostat within 12 weeks prior to randomization\n* Receipt of gene therapy at any time\n\nNote: Other inclusion or exclusion criteria may apply","MALE","4 Years","18 Years",{"count":65,"type":19},90,[67],"PHASE3","The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.",[70,71,72,73,74,75,76,77,78,79,80,31,81],"Duchenne Muscular Dystrophy (DMD)","Muscular Dystrophy, Duchenne","Muscular Dystrophy (DMD)","DMD","Muscular Dystrophies","Muscular Dystrophy in Children","Muscular Dystrophy, Duchenne Type","Muscular Dystrophy, Duchenne and Becker Types","Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)","Genetic Disease, Inborn","Genetic Disease, X-Linked","Neuromuscular Diseases (NMD)",[83,73,84,85,86,87,88,89,90,91,59,92,93,94,95,96,97,98,99,100,101,102,103,104],"Ambulatory","Duchenne Muscular Dystrophy","Duchenne","Dyne","Dyne Therapeutics","DYNE-251","Dystrophy","Exon Skipping","Exon 51","Pediatric","PMO","Muscle Function","Muscular Dystropy, Duchenne","Rise From Floor","RFF","RFF Velocity","Rostudirsen","Time to rise","TTR","TTR Velocity","Zeleciment rostudirsen","Z-rostudirsen","RECRUITING","2026-05-20",{"date":108,"type":45},"2026-05-27",{"date":110,"type":19},"2026-06",{"date":112,"type":19},"2032-10",{"name":87,"class":114},"INDUSTRY",1]