ClinicaliQ Trial Snapshot
- A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) — Recruiting • Phase I / Phase II • Rare Diseases • NCT06138639.
- Single intravenous gene therapy infusion (SGT-003) tested for safety and effectiveness in children with Duchenne muscular dystrophy across age groups.
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What This Trial Is Studying
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 < 7 years of age. Cohort 2 will include participants 7 to < 12 years of age. Cohort 3 will include participants 0 to < 4 years of age. Cohort 4 will include participants 12 to < 18 years of age. Cohort 5 will include participants 10…