A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Registration Number
- NCT06606340
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- 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 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 300
- 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
-
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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Eteplirsen Eteplirsen - Golodirsen Golodirsen - Casimersen Casimersen -
- Primary Outcome Measures
Name Time Method Loss of Ambulation (LOA) Up to 5 years Time to Rise From the Floor (Supine to Stand) Up to 5 years Time to Walk/Run 10 Meters Up to 5 years Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A Up to 5 years Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted) Up to 5 years Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO) Up to 5 years
- Secondary Outcome Measures
Name Time Method
Related Research Topics
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Trial Locations
- 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, 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
🇺🇸Saint 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