A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Start Date
11/23/2020
Completion Date
2/29/2028
Summary
Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed. This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.
Eligibility Criteria
Age Range: 2 years to No maximum
Interventions
delandistrogene moxeparvovec
Conditions
Locations
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United States
University of California, San Diego
La Jolla, California 92037
United States
University of California, Los Angeles
Los Angeles, California 90095
United States
Stanford University
Palo Alto, California 94304
United States
University of California, Davis
Sacramento, California 95616
United States
University of Florida
Gainesville, Florida 32610
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
Washington University in St. Louis
St Louis, Missouri 63110
United States
Duke University Medical Center
Durham, North Carolina 27705
United States
Nationwide Children's Hospital
Columbus, Ohio 43205
United States
Neurology Rare Disease Center
Flower Mound, Texas 75028
United States
Children's Hospital of The King's Daughters
Norfolk, Virginia 23507
United States