[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Avidity Biosciences, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":165},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,69,102,153],{"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":4,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":17,"minAge":76,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":77,"phases":4,"briefSummary":78,"conditions":79,"keywords":85,"overallStatus":96,"whyStopped":4,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":100,"locationsCount":101},"100612219","managed-access-program-for-del-zota-in-participants-with-dmd-mutations-amenable-to-exon-44-skipping-100612219","NCT07250737","Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping","Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping","Key Inclusion Criteria\n\nRollover Participants\n\n* Completed Study EXPLORE44-OLE Treatment Period (through W102)\n* No significant tolerability issues with AOC 1044\n\nNew (Non-Rollover) Participants\n\n* Permanently residing in the US and have a US primary health care provider\n* Documented dystrophin gene mutation that is amenable to exon 44 skipping\n* Age 2 or older at the time of consent\n* If previously treated with gene therapy for DMD, treatment and associated immunosuppressive regimen was more than 12 months before consent and in the opinion of the prescriber, participant has had an unsatisfactory treatment response\n\nKey Exclusion Criteria\n\nRollover Participants\n\n• Prescence of any new condition or worsening of existing condition that could affect participant's safety or ability to comply with the program requirements\n\nNew (Non-Rollover) Participants\n\n* Recently treated with or on a clinical study for another investigation drug\n* Serious respiratory or cardiac dysfunction, or nearing end of life\n* Screening laboratory parameters do not meet protocol requirements\n* History of multiple drug allergies or to any component of AOC 1044\n* Participants who discontinued early from the treatment period of EXPLORE44 or EXPLORE44-OLE","2 Years","EXPANDED_ACCESS","The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.",[30,80,32,81,34,82,83,28,84],"Muscular Diseases","Neuromuscular Diseases","Genetic Diseases, Inborn","Genetic Diseases, X-Linked","Muscular Dystrophy, Duchenne",[52,53,44,46,47,48,49,86,87,41,88,89,90,91,92,93,94,95],"Del-zota","delpacibart zotadirsen","exon skipping therapy","dystrophin","managed access","expanded access","pre-approval access","compassionate use","MAP","EAP","AVAILABLE","2026-06-26",{"date":99,"type":61},"2026-06-29",{"name":67,"class":68},14,{"id":103,"slug":104,"hasResults":11,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":108,"eligibilityCriteria":109,"healthyVolunteers":11,"sex":110,"minAge":19,"maxAge":111,"enrollmentInfo":112,"targetDuration":4,"studyType":23,"phases":114,"briefSummary":107,"conditions":115,"keywords":133,"overallStatus":143,"whyStopped":4,"lastUpdateSubmitDate":144,"lastUpdatePostDateStruct":145,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":152},"100595880","phase-3-a-study-to-evaluate-del-brax-also-referred-to-as-aoc-1020-in-participants-with-fshd-100595880","NCT07038200","A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD","A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)","FORTITUDE-3","Inclusion Criteria:\n\n* Clinical and genetic diagnosis of FSHD1 or FSHD2\n* Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening\n* Adequate muscle strength based on QMT composite score\n\nExclusion Criteria:\n\n* Breastfeeding, pregnancy, or intent to become pregnant during the study\n* Unwilling or unable to comply with contraceptive requirements\n* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study\n* Blood Pressure \\> 140\u002F90 mmHg at Screening\n* Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer\n* Treatment with an oligonucleotide within 9 months of Screening","ALL","70 Years",{"count":113,"type":22},200,[25],[116,117,118,119,120,121,122,123,124,125,126,127,128,129,130,131,132],"Facioscapulohumeral Muscular Dystrophy","FSHD","FSHD - Facioscapulohumeral Muscular Dystrophy","FSHD1","FSHD2","Fascioscapulohumeral Muscular Dystrophy","Fascioscapulohumeral Muscular Dystrophy Type 1","Fascioscapulohumeral Muscular Dystrophy Type 2","Facioscapulohumeral Muscular Dystrophy 1","Facioscapulohumeral Dystrophy","Facio-Scapulo-Humeral Dystrophy","Facioscapulohumeral Muscular Dystrophy 2","Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)","FSH Muscular Dystrophy","Landouzy Dejerine Dystrophy","Landouzy-Dejerine Muscular Dystrophy","Landouzy-Dejerine Syndrome",[52,53,134,135,136,137,138,139,108,140,141,142],"del-brax","del brax","delbrax","AOC1020","AOC 1020","delpacibart braxlosiran","FORTITUDE Phase 3","FORTITUDE","FORTITUDE 3","RECRUITING","2026-05-27",{"date":146,"type":61},"2026-05-29",{"date":148,"type":61},"2025-06-10",{"date":150,"type":22},"2028-07",{"name":67,"class":68},46,{"id":154,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":155,"targetDuration":4,"studyType":23,"phases":156,"briefSummary":26,"conditions":157,"keywords":158,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":159,"lastUpdatePostDateStruct":160,"startDateStruct":162,"completionDateStruct":163,"leadSponsor":164,"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":161,"type":61},"2026-05-14",{"date":63,"type":22},{"date":65,"type":22},{"name":67,"class":68},""]