A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Key facts
- Where
- Baltimore, Charlottesville, Cincinnati, Columbus, Durham, Fort Worth, Gainesville, Hershey, Houston, Iowa City, Kansas City, Little Rock, Los Angeles, New York, Orange, Philadelphia, Pittsburgh, Sacramento, Seattle, St Louis, The Bronx, Washington D.C., and Winston-Salem, United StatesSee the 23 sites
Where in United States
- Kennedy Krieger InstituteBaltimore, United StatesNo longer taking part
- UVA Children's HospitalCharlottesville, United StatesNo longer taking part
- Children's Hospital Medical CenterCincinnati, United StatesNo longer taking part
- Nationwide Children's HospitalColumbus, United StatesNo longer taking part
- Duke Lenox Baker Children's HospitalDurham, United StatesNo longer taking part
- Cook Children's HospitalFort Worth, United StatesNo longer taking part
- University of FloridaGainesville, United StatesNo longer taking part
- Penn State Health Milton S. Hershey Medical CenterHershey, United StatesNo longer taking part
- Texas Children's HospitalHouston, United StatesNo longer taking part
- University of Iowa Hospitals and ClinicsIowa City, United StatesNo longer taking part
- University of Kansas Medical CenterKansas City, United StatesNo longer taking part
- Arkansas Children's Hospital Research InstituteLittle Rock, United StatesNo longer taking part
- Children's Hospital Los AngelesLos Angeles, United StatesNo longer taking part
- Columbia University Medical Center - PINNew York, United StatesNo longer taking part
- Children's Hospital of Orange County - Main Campus - PINOrange, United StatesNo longer taking part
- Children's Hospital of PhiladelphiaPhiladelphia, United StatesNo longer taking part
- UPMC Children's Hospital of PittsburghPittsburgh, United StatesNo longer taking part
- University of California, Davis Medical CenterSacramento, United StatesNo longer taking part
- Seattle Children's HospitalSeattle, United StatesNo longer taking part
- Washington UniversitySt Louis, United StatesNo longer taking part
- Montefiore Medical CenterThe Bronx, United StatesNo longer taking part
- Children's National HospitalWashington D.C., United StatesNo longer taking part
- Atrium Health Wake Forest University Health SciencesWinston-Salem, United StatesNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Sarepta Therapeutics, Inc.
- Trial code
- NCT06606340
- Record updated
- 2 November 2026
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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.
Exclusion: 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; 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.