Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Study Summary

This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed.

Conditions

Duchenne Muscular Dystrophy

Interventions / Drugs

SGT-001

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment12 participants

Primary Completion—

Lead SponsorSolid Biosciences Inc.

Data Retrieved2026-04-11 04:27:33.472517+00:00

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