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A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Sponsor: Sarepta Therapeutics, Inc.
Summary
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.
Official title: A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
300
Start Date
2019-01-07
Completion Date
2033-12-31
Last Updated
2025-09-10
Healthy Volunteers
No
Conditions
Interventions
Eteplirsen
No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.
Golodirsen
No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.
Casimersen
No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.
Locations (20)
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