A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Condition(s) studied
Investigational drug(s) / intervention(s)
SGT-003: Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Study summary
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
Eligibility
Primary outcome measure(s)
- Incidence of treatment-emergent adverse events (AEs) — Day 360
- Change from baseline in Microdystrophin Protein Levels — Day 90
Microdystrophin expression evaluation in muscle biopsies
Trial sites (15)
| Facility | City | Region | Status |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | Recruiting |
| University of California, Los Angeles Medical Center | Los Angeles | California | Recruiting |
| University of California, Davis | Sacramento | California | Recruiting |
| University of California | San Diego | California | Recruiting |
| Rare Disease Research | Atlanta | Georgia | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | Recruiting |
| Washington University in St. Louis | St Louis | Missouri | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | Recruiting |
| Oregon Health and Sciences University | Portland | Oregon | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | Recruiting |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | Recruiting |
| Seattle Children's Hospital | Seattle | Washington | Recruiting |
| The Hospital for Sick Children | Toronto | Ontario | Recruiting |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS | Rome | Italy | Recruiting |
| Great Ormond Street Hospital | London | United Kingdom | 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 NCT06138639 on ClinicalTrials.gov ↗ ← All trials in the USA