[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100526737":3},{"organization":4,"armGroups":7,"interventions":30,"overallOfficials":37,"centralContacts":42,"locations":47,"responsibleParty":306,"collaborators":36,"id":308,"slug":36,"hasResults":309,"nctId":310,"briefTitle":311,"officialTitle":312,"acronym":36,"eligibilityCriteria":313,"healthyVolunteers":309,"sex":314,"minAge":315,"maxAge":316,"enrollmentInfo":317,"targetDuration":36,"studyType":320,"phases":321,"briefSummary":324,"conditions":325,"keywords":327,"overallStatus":50,"whyStopped":36,"lastUpdateSubmitDate":330,"lastUpdatePostDateStruct":331,"startDateStruct":334,"completionDateStruct":336,"leadSponsor":338,"locationsCount":339},{"fullName":5,"class":6},"Solid Biosciences Inc.","INDUSTRY",[8,14,18,22,26],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort 1: SGT-003","EXPERIMENTAL","All ambulatory participants from age 4 to \\\u003C 7 years will receive a single IV infusion of SGT-003 on Day 1.",[13],"Genetic: SGT-003",{"label":15,"type":10,"description":16,"interventionNames":17},"Cohort 2: SGT-003","All ambulatory participants from age 7 to \\\u003C 12 years will receive a single IV infusion of SGT-003 on Day 1.",[13],{"label":19,"type":10,"description":20,"interventionNames":21},"Cohort 3: SGT-003","All participants from age 0 to \\\u003C 4 years will receive a single IV infusion of SGT-003 on Day 1.",[13],{"label":23,"type":10,"description":24,"interventionNames":25},"Cohort 4: SGT-003","All ambulatory participants from age 12 to \\\u003C 18 years will receive a single IV infusion of SGT-003 on Day 1.",[13],{"label":27,"type":10,"description":28,"interventionNames":29},"Cohort 5: SGT-003","All non-ambulatory participants from age 10 to \\\u003C 18 years will receive a single IV infusion of SGT-003 on Day 1.",[13],[31],{"type":32,"name":33,"description":34,"armGroupLabels":35,"otherNames":36},"GENETIC","SGT-003","Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)",[9,15,19,23,27],null,[38],{"name":39,"affiliation":40,"role":41},"Solid Bio Clinical Trials","Solid Biosciences","STUDY_DIRECTOR",[43],{"name":39,"role":44,"phone":45,"phoneExt":36,"email":46},"CONTACT","617-337-4680","clinicaltrials@solidbio.com",[48,69,86,102,118,135,152,169,186,203,220,237,254,272,289],{"facility":49,"status":50,"city":51,"state":52,"zip":53,"country":54,"cosmosGeoPoint":55,"geoPoint":60,"contacts":61},"Arkansas Children's Hospital","RECRUITING","Little Rock","Arkansas","72202","United States",{"type":56,"coordinates":57},"Point",[58,59],-92.28959,34.74648,{"lat":59,"lon":58},[62,66],{"name":63,"role":44,"phone":64,"phoneExt":36,"email":65},"Amber Kellogg","501-364-3389","aveerapandiyan@uams.edu",{"name":67,"role":68,"phone":36,"phoneExt":36,"email":36},"Aravindhan Veerapandiyan, MD","PRINCIPAL_INVESTIGATOR",{"facility":70,"status":50,"city":71,"state":72,"zip":73,"country":54,"cosmosGeoPoint":74,"geoPoint":78,"contacts":79},"University of California, Los Angeles Medical Center","Los Angeles","California","90095",{"type":56,"coordinates":75},[76,77],-118.24368,34.05223,{"lat":77,"lon":76},[80,84],{"name":81,"role":44,"phone":82,"phoneExt":36,"email":83},"Merve Kaleli","310-825-3264","mkaleli@mednet.ucla.edu",{"name":85,"role":68,"phone":36,"phoneExt":36,"email":36},"Perry Shieh, MD, PhD",{"facility":87,"status":50,"city":88,"state":72,"zip":89,"country":54,"cosmosGeoPoint":90,"geoPoint":94,"contacts":95},"University of California, Davis","Sacramento","95817",{"type":56,"coordinates":91},[92,93],-121.4944,38.58157,{"lat":93,"lon":92},[96,100],{"name":97,"role":44,"phone":98,"phoneExt":36,"email":99},"Neuromuscular Research Lab","916-734-5057","NMRL@ucdavis.edu",{"name":101,"role":68,"phone":36,"phoneExt":36,"email":36},"Craig McDonald, MD",{"facility":103,"status":50,"city":104,"state":72,"zip":105,"country":54,"cosmosGeoPoint":106,"geoPoint":110,"contacts":111},"University of California","San Diego","92037",{"type":56,"coordinates":107},[108,109],-117.16472,32.71571,{"lat":109,"lon":108},[112,116],{"name":113,"role":44,"phone":114,"phoneExt":36,"email":115},"Gabriella Penner","858-382-3061","gpenner@health.ucsd.edu",{"name":117,"role":68,"phone":36,"phoneExt":36,"email":36},"Chamindra Laverty, MD",{"facility":119,"status":50,"city":120,"state":121,"zip":122,"country":54,"cosmosGeoPoint":123,"geoPoint":127,"contacts":128},"Rare Disease Research","Atlanta","Georgia","30329",{"type":56,"coordinates":124},[125,126],-84.38798,33.749,{"lat":126,"lon":125},[129,133],{"name":130,"role":44,"phone":131,"phoneExt":36,"email":132},"Neona Ni","470-666-1884","neona.ni@rarediseaseresearch.com",{"name":134,"role":68,"phone":36,"phoneExt":36,"email":36},"Renata Shih, MD",{"facility":136,"status":50,"city":137,"state":138,"zip":139,"country":54,"cosmosGeoPoint":140,"geoPoint":144,"contacts":145},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611-2605",{"type":56,"coordinates":141},[142,143],-87.65005,41.85003,{"lat":143,"lon":142},[146,150],{"name":147,"role":44,"phone":148,"phoneExt":36,"email":149},"Alka Maheshwari","312-227-3813","amaheshwari@luriechildrens.org",{"name":151,"role":44,"phone":36,"phoneExt":36,"email":36},"Nancy Kuntz, MD",{"facility":153,"status":50,"city":154,"state":155,"zip":156,"country":54,"cosmosGeoPoint":157,"geoPoint":161,"contacts":162},"Washington University in St. Louis","St Louis","Missouri","63110",{"type":56,"coordinates":158},[159,160],-90.19789,38.62727,{"lat":160,"lon":159},[163,167],{"name":164,"role":44,"phone":165,"phoneExt":36,"email":166},"Natalie Goedeker","314-362-4919","neuromusclepediatricresearch@wustl.edu",{"name":168,"role":68,"phone":36,"phoneExt":36,"email":36},"Craig Zaidman, MD",{"facility":170,"status":50,"city":171,"state":172,"zip":173,"country":54,"cosmosGeoPoint":174,"geoPoint":178,"contacts":179},"Nationwide Children's Hospital","Columbus","Ohio","43215",{"type":56,"coordinates":175},[176,177],-82.99879,39.96118,{"lat":177,"lon":176},[180,184],{"name":181,"role":44,"phone":182,"phoneExt":36,"email":183},"Destany McCain","614-722-6961","dbs001@nationwidechildrens.org",{"name":185,"role":68,"phone":36,"phoneExt":36,"email":36},"Kevin Flanigan, MD",{"facility":187,"status":50,"city":188,"state":189,"zip":190,"country":54,"cosmosGeoPoint":191,"geoPoint":195,"contacts":196},"Oregon Health and Sciences University","Portland","Oregon","97239",{"type":56,"coordinates":192},[193,194],-122.67621,45.52345,{"lat":194,"lon":193},[197,201],{"name":198,"role":44,"phone":199,"phoneExt":36,"email":200},"Beata Dyar","503-494-8216","dyar@ohsu.edu",{"name":202,"role":68,"phone":36,"phoneExt":36,"email":36},"Erika Finanger, MD",{"facility":204,"status":50,"city":205,"state":206,"zip":207,"country":54,"cosmosGeoPoint":208,"geoPoint":212,"contacts":213},"Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":56,"coordinates":209},[210,211],-75.16362,39.95238,{"lat":211,"lon":210},[214,218],{"name":215,"role":44,"phone":216,"phoneExt":36,"email":217},"Brandt Parlanti","215-850-7769","parlantib@chop.edu",{"name":219,"role":68,"phone":36,"phoneExt":36,"email":36},"John Brandsema, MD",{"facility":221,"status":50,"city":222,"state":223,"zip":224,"country":54,"cosmosGeoPoint":225,"geoPoint":229,"contacts":230},"Children's Hospital of the King's Daughters","Norfolk","Virginia","23510",{"type":56,"coordinates":226},[227,228],-76.28522,36.84681,{"lat":228,"lon":227},[231,235],{"name":232,"role":44,"phone":233,"phoneExt":36,"email":234},"E.Ann Walker","757-668-6543","Proud.Research@CHKD.org",{"name":236,"role":68,"phone":36,"phoneExt":36,"email":36},"Crystal Proud, 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MD",{"facility":273,"status":50,"city":274,"state":36,"zip":275,"country":276,"cosmosGeoPoint":277,"geoPoint":281,"contacts":282},"Fondazione Policlinico Universitario Agostino Gemelli IRCCS","Rome","00168","Italy",{"type":56,"coordinates":278},[279,280],12.51133,41.89193,{"lat":280,"lon":279},[283,287],{"name":284,"role":44,"phone":285,"phoneExt":36,"email":286},"Lorenzo Stivala","+39 063-015-6330","lorenzo.stivala@policlinicogemelli.it",{"name":288,"role":68,"phone":36,"phoneExt":36,"email":36},"Eugenio Mercuri, MD, PhD",{"facility":290,"status":50,"city":291,"state":36,"zip":292,"country":293,"cosmosGeoPoint":294,"geoPoint":298,"contacts":299},"Great Ormond Street Hospital","London","WC1N 3JH","United Kingdom",{"type":56,"coordinates":295},[296,297],-0.12574,51.50853,{"lat":297,"lon":296},[300,304],{"name":301,"role":44,"phone":302,"phoneExt":36,"email":303},"Lucinda Furtado","+44 (0)20 7762 6932","Lucinda.furtado@gosh.nhs.uk",{"name":305,"role":68,"phone":36,"phoneExt":36,"email":36},"Francesco Muntoni, MD",{"type":307,"investigatorFullName":36,"investigatorTitle":36,"investigatorAffiliation":36,"oldNameTitle":36,"oldOrganization":36},"SPONSOR","100526737",false,"NCT06138639","A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)","A Phase 1\u002F2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)","Inclusion Criteria:\n\n* Cohort 1: 4 to \\\u003C7 years of age\n* Cohort 2: 7 to \\\u003C12 years of age\n* Cohort 3: 0 to \\\u003C 4 years of age\n* Cohort 4: 12 to \\\u003C 18 years of age\n* Cohort 5: 10 to \\\u003C 18 years of age\n* Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk\u002Frun test in \\\u003C 30 seconds:\n\n  * Cohorts 1, 2, and 4: Ambulatory\n  * Cohort 3: Either ambulatory or non-ambulatory\n  * Cohort 5: Non-ambulatory, but having been previously ambulatory by history\n* Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and\u002For a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.\n* Negative for AAV antibodies.\n* Steroid regimen:\n\n  * Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg\u002Fkg\u002Fday of prednisone or 0.75 mg\u002Fkg\u002Fday of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice.\n  * Cohort 3: N\u002FA\n* Meet 10-meter walk\u002Frun time criteria\n* Meet time to rise from supine criteria\n* Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria\n* Participant has body weight: ≤ 90 kg\n\nExclusion Criteria:\n\n* Treatment with dystrophin modifying drugs within 3 months prior to screening.\n* Current or prior treatment with an approved or investigational gene transfer drug.\n* Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.\n* Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.\n\nOther inclusion or exclusion criteria apply.","MALE","0 Years","17 Years",{"count":318,"type":319},60,"ESTIMATED","INTERVENTIONAL",[322,323],"PHASE1","PHASE2","This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \\\u003C 7 years of age. Cohort 2 will include participants 7 to \\\u003C 12 years of age. Cohort 3 will include participants 0 to \\\u003C 4 years of age. Cohort 4 will include participants 12 to \\\u003C 18 years of age. Cohort 5 will include participants 10 to \\\u003C 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.",[326],"Duchenne Muscular Dystrophy",[328,329],"DMD","Gene Therapy","2026-06-17",{"date":332,"type":333},"2026-06-22","ACTUAL",{"date":335,"type":333},"2024-05-06",{"date":337,"type":319},"2031-05-06",{"name":5,"class":6},15]