A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Condition(s) studied
Investigational drug(s) / intervention(s)
SGT-003: Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
Placebo: IV infusion
Study 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
Primary outcome measure(s)
- Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 — Baseline, Day 540
Trial sites (7)
| Facility | City | Region | Status |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | Not Yet Recruiting |
| Neurology Rare Disease Center | Flower Mound | Texas | Not Yet Recruiting |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | Not Yet Recruiting |
| The Children's Hospital of Westmead | Sydney | New South Wales | Recruiting |
| Alberta Children's Hospital | Calgary | Alberta | Recruiting |
| BC Children's Hospital | Vancouver | British Columbia | Recruiting |
| The Hospital for Sick Children (SickKids) | Toronto | Ontario | Recruiting |
More Solid Biosciences Inc. trials in the USA
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07160634 on ClinicalTrials.gov ↗ ← All trials in the USA