A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Start Date
10/22/2025
Completion Date
1/1/2034
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.
Eligibility Criteria
Age Range: 7 years to 11 years
Interventions
SGT-003
Placebo
Conditions
Locations
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United States
Neurology Rare Disease Center
Flower Mound, Texas 75082
United States
Children's Hospital of the King's Daughters
Norfolk, Virginia 23510
United States
The Children's Hospital of Westmead
Sydney, New South Wales
Australia
Alberta Children's Hospital
Calgary, Alberta T3B 6A8
Canada
BC Children's Hospital
Vancouver, British Columbia
Canada