NCT07160634

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: Solid Biosciences Inc. · Industry
• Sex: Male
• Age: 7 Years – 11 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	SGT-003	Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
DRUG	Placebo	IV infusion

Timeline

• Start date: 2025-10-22
• Primary completion: 2029-01-01
• Completion: 2034-01-01
• First posted: 2025-09-08
• Last updated: 2026-03-03

Locations

2 sites across 2 countries: Australia, Canada

Regulatory

• FDA-regulated drug study