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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 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PhD",{"facility":293,"status":50,"city":294,"state":36,"zip":295,"country":296,"countryCode":297,"cosmosGeoPoint":298,"geoPoint":302,"contacts":303},"Great Ormond Street Hospital","London","WC1N 3JH","United Kingdom","UK",{"type":57,"coordinates":299},[300,301],-0.12574,51.50853,{"lat":301,"lon":300},[304,308],{"name":305,"role":44,"phone":306,"phoneExt":36,"email":307},"Lucinda Furtado","+44 (0)20 7762 6932","Lucinda.furtado@gosh.nhs.uk",{"name":309,"role":69,"phone":36,"phoneExt":36,"email":36},"Francesco Muntoni, MD",{"type":311,"investigatorFullName":36,"investigatorTitle":36,"investigatorAffiliation":36,"oldNameTitle":36,"oldOrganization":36},"SPONSOR","100526737","phase-1-a-study-of-sgt-003-gene-therapy-in-duchenne-muscular-dystrophy-inspire-duchenne-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":323,"type":324},60,"ESTIMATED","INTERVENTIONAL",[327,328],"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.",[331],"Duchenne Muscular Dystrophy",[333,334],"DMD","Gene Therapy","2026-06-17",{"date":337,"type":338},"2026-06-22","ACTUAL",{"date":340,"type":338},"2024-05-06",{"date":342,"type":324},"2031-05-06",{"name":5,"class":6},15]