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

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.

NCT Number	NCT07160634
Lead Sponsor	Solid Biosciences Inc.
Conditions	Duchenne Muscular Dystrophy
Enrollment	80 participants
Start Date	2025-10-22
Primary Completion	2029-01 (estimated)
Study Completion	2034-01 (estimated)
Updated on ClinicalTrials.gov	2026-08-13