Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

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.

Timeline

Start

2017-12-06

Primary completion

2026-10-15

Completion

2026-10-15

Publications

• Boehler JF, Brown KJ, Ricotti V, Morris CA. N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy. Skelet Muscle. 2024 Jan 16;14(1):2. doi: 10.1186/s13395-023-00334-y.

Drugs

Indications