[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100605293":3},{"organization":4,"armGroups":7,"interventions":19,"overallOfficials":25,"centralContacts":30,"locations":36,"responsibleParty":119,"collaborators":25,"id":121,"slug":25,"hasResults":122,"nctId":123,"briefTitle":124,"officialTitle":125,"acronym":25,"eligibilityCriteria":126,"healthyVolunteers":122,"sex":127,"minAge":128,"maxAge":129,"enrollmentInfo":130,"targetDuration":25,"studyType":133,"phases":134,"briefSummary":136,"conditions":137,"keywords":139,"overallStatus":84,"whyStopped":25,"lastUpdateSubmitDate":143,"lastUpdatePostDateStruct":144,"startDateStruct":147,"completionDateStruct":149,"leadSponsor":151,"locationsCount":152},{"fullName":5,"class":6},"Solid Biosciences Inc.","INDUSTRY",[8,15],{"label":9,"type":10,"description":11,"interventionNames":12},"SGT-003 followed by Placebo","EXPERIMENTAL","Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2.",[13,14],"Drug: SGT-003","Drug: Placebo",{"label":16,"type":10,"description":17,"interventionNames":18},"Placebo followed by SGT-003","Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2.",[13,14],[20,26],{"type":21,"name":22,"description":23,"armGroupLabels":24,"otherNames":25},"DRUG","SGT-003","Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)",[16,9],null,{"type":21,"name":27,"description":28,"armGroupLabels":29,"otherNames":25},"Placebo","IV infusion",[16,9],[31],{"name":32,"role":33,"phone":34,"phoneExt":25,"email":35},"Solid Bio Clinical Trials","CONTACT","6173374680","clinicaltrials@solidbio.com",[37,54,68,82,101],{"facility":38,"status":39,"city":40,"state":41,"zip":42,"country":43,"cosmosGeoPoint":44,"geoPoint":49,"contacts":50},"Arkansas Children's Hospital","NOT_YET_RECRUITING","Little Rock","Arkansas","72202","United States",{"type":45,"coordinates":46},"Point",[47,48],-92.28959,34.74648,{"lat":48,"lon":47},[51],{"name":52,"role":33,"phone":25,"phoneExt":25,"email":53},"Amber Kellogg, RN","KelloggA@archildrens.org",{"facility":55,"status":39,"city":56,"state":57,"zip":58,"country":43,"cosmosGeoPoint":59,"geoPoint":63,"contacts":64},"Neurology Rare Disease Center","Flower Mound","Texas","75082",{"type":45,"coordinates":60},[61,62],-97.09696,33.01457,{"lat":62,"lon":61},[65],{"name":66,"role":33,"phone":25,"phoneExt":25,"email":67},"Jennifer Avelar, CRC","Research@neuromdcenter.com",{"facility":69,"status":39,"city":70,"state":71,"zip":72,"country":43,"cosmosGeoPoint":73,"geoPoint":77,"contacts":78},"Children's Hospital of the King's Daughters","Norfolk","Virginia","23510",{"type":45,"coordinates":74},[75,76],-76.28522,36.84681,{"lat":76,"lon":75},[79],{"name":80,"role":33,"phone":25,"phoneExt":25,"email":81},"Jennifer Beachum, BSHS","Proud.research@chkd.org",{"facility":83,"status":84,"city":85,"state":86,"zip":25,"country":87,"cosmosGeoPoint":88,"geoPoint":92,"contacts":93},"The Children's Hospital of Westmead","RECRUITING","Sydney","New South Wales","Australia",{"type":45,"coordinates":89},[90,91],151.20732,-33.86785,{"lat":91,"lon":90},[94,98],{"name":95,"role":33,"phone":96,"phoneExt":25,"email":97},"CHW CRC clinical trials team","(02) 7825 1387","SCHN-ClinicalTrials@health.nsw.gov.au",{"name":99,"role":100,"phone":25,"phoneExt":25,"email":25},"Michelle Lorentzos, MD","PRINCIPAL_INVESTIGATOR",{"facility":102,"status":84,"city":103,"state":104,"zip":25,"country":105,"cosmosGeoPoint":106,"geoPoint":110,"contacts":111},"BC Children's Hospital","Vancouver","British Columbia","Canada",{"type":45,"coordinates":107},[108,109],-123.11934,49.24966,{"lat":109,"lon":108},[112,117],{"name":113,"role":33,"phone":114,"phoneExt":115,"email":116},"Nela Martic","604-875-2345","6549","nmartic3@cw.bc.ca",{"name":118,"role":100,"phone":25,"phoneExt":25,"email":25},"Kathryn Selby, MD",{"type":120,"investigatorFullName":25,"investigatorTitle":25,"investigatorAffiliation":25,"oldNameTitle":25,"oldOrganization":25},"SPONSOR","100605293",false,"NCT07160634","A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)","A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n* Participant is ambulatory.\n* Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.\n* Negative for antibodies against adeno-associated virus.\n* On a stable daily oral regimen of at least 0.5 mg\u002Fkg\u002Fday prednisone or 0.75 milligrams per kilogram per day (mg\u002Fkg\u002Fday) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice.\n* Meet 10-meter walk\u002Frun time criteria.\n* Meet time to rise from supine criteria.\n* Participant has bodyweight ≤50 kg.\n\nExclusion Criteria:\n\n* Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy.\n* Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment.\n* Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report.\n\nOther Inclusion\u002FExclusion criteria to be applied as per protocol.","MALE","7 Years","11 Years",{"count":131,"type":132},80,"ESTIMATED","INTERVENTIONAL",[135],"PHASE3","This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.",[138],"Duchenne Muscular Dystrophy",[22,140,141,142],"Duchenne Muscular Dystrophy (DMD)","adeno-associated virus (AAV)","IMPACT DUCHENNE","2026-06-01",{"date":145,"type":146},"2026-06-02","ACTUAL",{"date":148,"type":146},"2025-10-22",{"date":150,"type":132},"2034-01",{"name":5,"class":6},5]