Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne Muscular Dystrophy, DMD, Post-marketing, Observational, Phase 4, Eteplirsen, Golodirsen, Casimersen
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.
Interventions
DRUGEteplirsen
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.
DRUGGolodirsen
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.
DRUGCasimersen
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.
Sponsors
Sarepta Therapeutics, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * 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 criteria
* 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: 1. The participant's ability to comply with the protocol-required procedures 2. The participant's wellbeing or safety, and/or 3. The clinical interpretability of the data collected from the participant. Other inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 |
Countries
United States
Outcome results
None listed