[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100410604":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":24,"centralContacts":28,"locations":35,"responsibleParty":142,"collaborators":144,"id":147,"slug":148,"hasResults":149,"nctId":150,"briefTitle":151,"officialTitle":152,"acronym":153,"eligibilityCriteria":154,"healthyVolunteers":149,"sex":155,"minAge":156,"maxAge":33,"enrollmentInfo":157,"targetDuration":33,"studyType":160,"phases":161,"briefSummary":163,"conditions":164,"keywords":166,"overallStatus":38,"whyStopped":33,"lastUpdateSubmitDate":174,"lastUpdatePostDateStruct":175,"startDateStruct":178,"completionDateStruct":180,"leadSponsor":182,"locationsCount":183},{"fullName":5,"class":6},"Sarepta Therapeutics, Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Delandistrogene Moxeparvovec","EXPERIMENTAL","Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.",[13],"Genetic: delandistrogene moxeparvovec",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"GENETIC","delandistrogene moxeparvovec","Single IV infusion of delandistrogene moxeparvovec",[9],[21,22,23],"SRP-9001","delandistrogene moxeparvovec-rokl","ELEVIDYS",[25],{"name":26,"affiliation":5,"role":27},"Medical Director","STUDY_DIRECTOR",[29],{"name":30,"role":31,"phone":32,"phoneExt":33,"email":34},"Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4","CONTACT","1-888-SAREPTA (1-888-727-3782)",null,"SareptAlly@Sarepta.com",[36,57,72,84,100,111,127],{"facility":37,"status":38,"city":39,"state":40,"zip":41,"country":42,"countryCode":43,"cosmosGeoPoint":44,"geoPoint":49,"contacts":50},"Arkansas Children's Hospital","RECRUITING","Little Rock","Arkansas","72202","United States","US",{"type":45,"coordinates":46},"Point",[47,48],-92.28959,34.74648,{"lat":48,"lon":47},[51,54],{"name":33,"role":31,"phone":52,"phoneExt":33,"email":53},"501-364-1868","KurakuVV@archildrens.org",{"name":55,"role":56,"phone":33,"phoneExt":33,"email":33},"Aravindhan Veerapandiyan, MD","PRINCIPAL_INVESTIGATOR",{"facility":58,"status":38,"city":59,"state":60,"zip":61,"country":42,"countryCode":43,"cosmosGeoPoint":62,"geoPoint":66,"contacts":67},"Stanford University","Palo Alto","California","94304",{"type":45,"coordinates":63},[64,65],-122.14302,37.44188,{"lat":65,"lon":64},[68,70],{"name":33,"role":31,"phone":33,"phoneExt":33,"email":69},"neuromuscularresearch@stanford.edu",{"name":71,"role":56,"phone":33,"phoneExt":33,"email":33},"John Day, MD",{"facility":73,"status":38,"city":74,"state":60,"zip":75,"country":42,"countryCode":43,"cosmosGeoPoint":76,"geoPoint":80,"contacts":81},"University of California, Davis","Sacramento","95616",{"type":45,"coordinates":77},[78,79],-121.4944,38.58157,{"lat":79,"lon":78},[82],{"name":83,"role":56,"phone":33,"phoneExt":33,"email":33},"Craig McDonald, MD",{"facility":85,"status":38,"city":86,"state":87,"zip":88,"country":42,"countryCode":43,"cosmosGeoPoint":89,"geoPoint":93,"contacts":94},"Washington University in St. Louis","St Louis","Missouri","63110",{"type":45,"coordinates":90},[91,92],-90.19789,38.62727,{"lat":92,"lon":91},[95,98],{"name":33,"role":31,"phone":96,"phoneExt":33,"email":97},"314-362-6981","NeuromusclePediatricResearch@wustl.edu",{"name":99,"role":56,"phone":33,"phoneExt":33,"email":33},"Craig Zaidman, MD",{"facility":101,"status":102,"city":103,"state":104,"zip":105,"country":42,"countryCode":43,"cosmosGeoPoint":106,"geoPoint":110,"contacts":33},"Nationwide Children's Hospital","ACTIVE_NOT_RECRUITING","Columbus","Ohio","43205",{"type":45,"coordinates":107},[108,109],-82.99879,39.96118,{"lat":109,"lon":108},{"facility":112,"status":38,"city":113,"state":114,"zip":115,"country":42,"countryCode":43,"cosmosGeoPoint":116,"geoPoint":120,"contacts":121},"Neurology Rare Disease Center","Flower Mound","Texas","75028",{"type":45,"coordinates":117},[118,119],-97.09696,33.01457,{"lat":119,"lon":118},[122,125],{"name":33,"role":31,"phone":123,"phoneExt":33,"email":124},"972-999-1011","Research@neuromdcenter.com",{"name":126,"role":56,"phone":33,"phoneExt":33,"email":33},"Diana Castro, MD",{"facility":128,"status":38,"city":129,"state":130,"zip":131,"country":42,"countryCode":43,"cosmosGeoPoint":132,"geoPoint":136,"contacts":137},"Children's Hospital of The King's Daughters","Norfolk","Virginia","23507",{"type":45,"coordinates":133},[134,135],-76.28522,36.84681,{"lat":135,"lon":134},[138,140],{"name":33,"role":31,"phone":33,"phoneExt":33,"email":139},"Proud.Research@chkd.org",{"name":141,"role":56,"phone":33,"phoneExt":33,"email":33},"Crystal Proud, MD",{"type":143,"investigatorFullName":33,"investigatorTitle":33,"investigatorAffiliation":33,"oldNameTitle":33,"oldOrganization":33},"SPONSOR",[145],{"name":146,"class":6},"Hoffmann-La Roche","100410604","phase-1-a-gene-transfer-therapy-study-to-evaluate-the-safety-of-and-expression-from-delandistrogene-moxeparvovec-srp-9001-in-participants-with-duchenne-muscular-dystrophy-dmd---non-ambulatory-cohort-100410604",false,"NCT04626674","A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort","An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)","ENDEAVOR","Inclusion Criteria:\n\n* For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.\n* Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.\n* Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.\n* Cohort 1: Is ambulatory, and ≥4 to \\\u003C8 years of age at the time of Screening.\n* Cohort 2: Is ambulatory, and ≥8 to \\\u003C18 years of age at the time of Screening.\n* Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.\n* Cohort 4: Is ambulatory and ≥3 to \\\u003C4 years of age at the time of Screening.\n* Cohort 5a: Is ambulatory and ≥4 to \\\u003C9 years of age with time to rise from the floor ≤7 seconds at the screening visit.\n* Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.\n* Cohort 6: Is ambulatory, and ≥2 to \\\u003C3 years of age at the time of Screening.\n* Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.\n* Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.\n* Genetic mutation inclusion criteria vary by cohort.\n\nAll Cohorts:\n\n* Ability to cooperate with motor assessment testing.\n* rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.\n\nExclusion Criteria:\n\n* Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.\n* Has a concomitant illness, autoimmune disease, chronic drug treatment, and\u002For cognitive delay\u002Fimpairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.\n* Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.\n* Abnormality in protocol-specified diagnostic evaluations or laboratory tests.\n\nNote: Other inclusion\u002Fexclusion criteria apply.","MALE","2 Years",{"count":158,"type":159},83,"ESTIMATED","INTERVENTIONAL",[162],"PHASE1","Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.\n\nThis is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.",[165],"Duchenne Muscular Dystrophy",[165,167,168,169,170,171,172,173],"Gene-Delivery","Gene Therapy","DMD","Ambulatory Non-ambulatory","Pediatric","Adult","Dystrophin","2026-06-19",{"date":176,"type":177},"2026-06-24","ACTUAL",{"date":179,"type":177},"2020-11-23",{"date":181,"type":159},"2028-02-29",{"name":5,"class":6},7]