[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"muscular-dystrophies-duchenne-becker-myotonic-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:muscular-dystrophies-duchenne-becker-myotonic-dystrophy":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,69,125],{"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":42,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":4},"100053565","phase-3-a-phase-3-study-to-evaluate-the-safety-and-efficacy-of-aoc-1044-also-referred-to-as-delpacibart-zotadirsen-in-participants-with-dmd-with-gene-mutations-amenable-to-exon-44-skipping-100053565",false,"NCT07587242","A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping","A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (Delpacibart Zotadirsen) for the Treatment of DMD With Gene Mutations Amenable to Exon 44 Skipping","SAFARI44","Key Inclusion Criteria:\n\n* Ambulatory males with clinical and genetic diagnosis of DMD\n* Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping\n* 7 to 16 years of age at time of consent\n* TTR and NSAA assessment completed within the protocol specified parameters at Screening\n* On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.\n\nKey Exclusion Criteria:\n\n* Previous treatment cell or gene therapy.\n* Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).\n* Lab values outside of the protocol specified range at Screening\n* If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:\n* Less than 1 month, for growth hormone and\u002For testosterone\n* Less than 6 months for givinostat","MALE","7 Years","16 Years",{"count":21,"type":22},70,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping",[28,29,30,31,32,33,34,35,36,37,38,39,40,41],"Muscular Dystrophies","Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)","Muscular Disorders, Atrophic","Muscular Disease","Musculoskeletal Diseases","Neuromuscular Diseases (NMD)","Nervous System Diseases","Genetic Diseases","X-Linked","Hereditary","Neonatal Disease","Duchene Muscular Dystrophy","Congenital","DMD",[43,44,45,46,47,48,49,50,15,51,52,53,54,55,56,41],"AOC","AOC 1044","AOC 1044-CS3","AOC 1044-CS1","AOC 1044-CS2","EXPLORE44","EXPLORE44-OLE","SAFARI","SAFARI 44","Avidity","Avidity Biosciences","Exon Skipping Therapy","Avidity Biosciences Inc., A Novartis Company","del-zota","NOT_YET_RECRUITING","2026-07-10",{"date":60,"type":61},"2026-07-13","ACTUAL",{"date":63,"type":22},"2026-06",{"date":65,"type":22},"2030-07",{"name":67,"class":68},"Avidity Biosciences, Inc.","INDUSTRY",{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":75,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":17,"minAge":77,"maxAge":78,"enrollmentInfo":79,"targetDuration":4,"studyType":23,"phases":81,"briefSummary":82,"conditions":83,"keywords":93,"overallStatus":116,"whyStopped":4,"lastUpdateSubmitDate":117,"lastUpdatePostDateStruct":118,"startDateStruct":120,"completionDateStruct":121,"leadSponsor":123,"locationsCount":124},"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","4 Years","18 Years",{"count":80,"type":22},90,[25],"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.",[84,85,86,41,28,87,88,89,29,90,91,92,33],"Duchenne Muscular Dystrophy (DMD)","Muscular Dystrophy, Duchenne","Muscular Dystrophy (DMD)","Muscular Dystrophy in Children","Muscular Dystrophy, Duchenne Type","Muscular Dystrophy, Duchenne and Becker Types","Genetic Disease, Inborn","Genetic Disease, X-Linked","Congenital, Hereditary, and Neonatal Diseases and Abnormalities",[94,41,95,96,97,98,99,100,101,102,75,103,104,105,106,107,108,109,110,111,112,113,114,115],"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":119,"type":61},"2026-05-27",{"date":63,"type":22},{"date":122,"type":22},"2032-10",{"name":98,"class":68},1,{"id":126,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":127,"targetDuration":4,"studyType":23,"phases":128,"briefSummary":26,"conditions":129,"keywords":130,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":131,"lastUpdatePostDateStruct":132,"startDateStruct":134,"completionDateStruct":135,"leadSponsor":136,"locationsCount":4},"100638788",{"count":21,"type":22},[25],[28,29,30,31,32,33,34,35,36,37,38,39,40,41],[43,44,45,46,47,48,49,50,15,51,52,53,54,55,56,41],"2026-05-08",{"date":133,"type":61},"2026-05-14",{"date":63,"type":22},{"date":65,"type":22},{"name":67,"class":68}]