Status:
ENROLLING_BY_INVITATION
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
Brief 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 participa...
Eligibility Criteria
Inclusion
- Key
- Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.
- Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.
- Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.
- Key
Exclusion
- Is currently participating in any DMD interventional study at the time of this study enrollment.
- Has declined to provide consent for collection of their genetic data.
- Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:
- The participant's ability to comply with the protocol-required procedures
- The participant's wellbeing or safety, and/or
- The clinical interpretability of the data collected from the participant.
- Other inclusion/exclusion criteria may apply.
Key Trial Info
Start Date :
January 7 2019
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2033
Estimated Enrollment :
300 Patients enrolled
Trial Details
Trial ID
NCT06606340
Start Date
January 7 2019
End Date
December 31 2033
Last Update
September 10 2025
Active Locations (20)
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1
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, United States, 72202
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
3
University of California Davis Medical Center
Sacramento, California, United States, 95817
4
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010