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An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
Sponsor: Sarepta Therapeutics, Inc.
Summary
This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.
Official title: A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Key Details
Gender
MALE
Age Range
4 Years - Any
Study Type
OBSERVATIONAL
Enrollment
500
Start Date
2024-02-07
Completion Date
2038-12-31
Last Updated
2025-12-02
Healthy Volunteers
No
Conditions
Interventions
Delandistrogene Moxeparvovec
No study medication will be provided by the sponsor during this study.
Standard of Care
No study medication will be provided by the sponsor during this study.
Locations (25)
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles - PIN
Los Angeles, California, United States
University of Colorado - PPDS
Aurora, Colorado, United States
Connecticut Children's Medical Center - Hartford
Hartford, Connecticut, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Nemours Children's Hospital - Orlando
Orlando, Florida, United States
All Children's Research Institute, Inc
St. Petersburg, Florida, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States
Indiana Clinical and Translational Science Institute
Indianapolis, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Michigan
Ann Arbor, Michigan, United States
Duke Lenox Baker Children's Hospital
Durham, North Carolina, United States
Childrens Hospital Medical Center of Akron
Akron, Ohio, United States
OHSU Healthcare (Oregon Health and Science University)
Portland, Oregon, United States
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Cook Children's Hospital
Fort Worth, Texas, United States
University of Virginia
Charlottesville, Virginia, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
The Board of Regents of the University of Wisconsin
Madison, Wisconsin, United States
The Medical College of Wisconsin
Milwaukee, Wisconsin, United States