A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Enrolling by invitation
Conditions studied: Duchenne Muscular Dystrophy
In brief
This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.
Key facts
- Study ID
- NCT06606340
- Run by
- Sarepta Therapeutics, Inc.
- People needed
- 300
- Starts
- 2019-01-07
- Expected to finish
- 2033-12-31
- Last updated by the study team
- 2025-09-10
Who can join
Age: any. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Arkansas Children's Hospital Research Institute — Little Rock, Arkansas, United States
- Children's Hospital Los Angeles — Los Angeles, California, United States
- University of California Davis Medical Center — Sacramento, California, United States
- Children's National Hospital — Washington D.C., District of Columbia, United States
- University of Florida — Gainesville, Florida, United States
- University Of Iowa Hospitals And Clinics — Iowa City, Iowa, United States
- Kennedy Krieger Institute — Baltimore, Maryland, United States
- Washington University — St Louis, Missouri, United States
- Columbia University Medical Center - PIN — New York, New York, United States
- Duke Lenox Baker Children's Hospital — Durham, North Carolina, United States
- Atrium Health Wake Forest University Health Sciences — Winston-Salem, North Carolina, United States
- Children's Hospital Medical Center — Cincinnati, Ohio, United States
- Nationwide Children's Hospital — Columbus, Ohio, United States
- Penn State Health Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Cook Children's Hospital — Fort Worth, Texas, United States
- Texas Children's Hospital — Houston, Texas, United States
- UVA Children's Hospital — Charlottesville, Virginia, United States
- Seattle Children's Hospital — Seattle, Washington, United States
Full record on ClinicalTrials.gov
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