Condition: Duchenne Muscular Dystrophy · Sponsor: Solid Biosciences Inc.
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.
This description comes directly from the study's public registry record.
Solid Bio Clinical Trials · 617-337-4680 · clinicaltrials@solidbio.com
Always discuss trial participation with your own doctor first.
| Arkansas Children's Hospital | Little Rock, Arkansas, United States | Recruiting |
| University of California, Los Angeles Medical Center | Los Angeles, California, United States | Recruiting |
| University of California, Davis | Sacramento, California, United States | Recruiting |
| University of California | San Diego, California, United States | Recruiting |
| Rare Disease Research | Atlanta, Georgia, United States | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago, Illinois, United States | Recruiting |
| Washington University in St. Louis | St Louis, Missouri, United States | Recruiting |
| Nationwide Children's Hospital | Columbus, Ohio, United States | Recruiting |
| Oregon Health and Sciences University | Portland, Oregon, United States | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | Recruiting |
| Children's Hospital of the King's Daughters | Norfolk, Virginia, United States | Recruiting |
| Seattle Children's Hospital | Seattle, Washington, United States | Recruiting |
+ 3 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT06138639