A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Start Date
5/6/2024
Completion Date
5/6/2031
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 Criteria
Age Range: No minimum to 17 years
Interventions
SGT-003
Conditions
Locations
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United States
University of California, Los Angeles Medical Center
Los Angeles, California 90095
United States
University of California, Davis
Sacramento, California 95817
United States
University of California
San Diego, California 92037
United States
Rare Disease Research
Atlanta, Georgia 30329
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611-2605
United States
Washington University in St. Louis
St Louis, Missouri 63110
United States
Nationwide Children's Hospital
Columbus, Ohio 43215
United States
Oregon Health and Sciences University
Portland, Oregon 97239
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Children's Hospital of the King's Daughters
Norfolk, Virginia 23510
United States
Seattle Children's Hospital
Seattle, Washington 98105
United States
The Hospital for Sick Children
Toronto, Ontario M5G 0A4
Canada
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, 00168
Italy
Great Ormond Street Hospital
London, WC1N 3JH
United Kingdom