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.
Interested in this trial?
Submit an InquiryInterventions
SGT-003DRUG
Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
PlaceboDRUG
IV infusion
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | United States |
| Neurology Rare Disease Center | Flower Mound | Texas | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | United States |
| The Children's Hospital of Westmead | Sydney | New South Wales | Australia |
| BC Children's Hospital | Vancouver | British Columbia | Canada |