NCT03368742

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Solid Biosciences Inc. · Industry
• Sex: Male
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
GENETIC	SGT-001	AAV9 vector containing muscle-specific promoter and microdystrophin construct

Timeline

• Start date: 2017-12-06
• Primary completion: 2026-10-15
• Completion: 2026-10-15
• First posted: 2017-12-11
• Last updated: 2026-03-03

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study