Enrolling by invitation NCT06606340
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Eteplirsen, Golodirsen, Casimersen.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: No limits · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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
- Drug 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. - Drug 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. - Drug 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.
Primary outcome measures
- Loss of Ambulation (LOA) [Time frame: Up to 5 years]
- Time to Rise From the Floor (Supine to Stand) [Time frame: Up to 5 years]
- Time to Walk/Run 10 Meters [Time frame: Up to 5 years]
- Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A [Time frame: Up to 5 years]
- Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted) [Time frame: Up to 5 years]
- Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO) [Time frame: Up to 5 years]
Eligibility criteria
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.
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:
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 20 centers
- Arkansas Children's Hospital Research Institute — Little Rock
- Children's Hospital Los Angeles — Los Angeles
- University of California Davis Medical Center — Sacramento
- Children's National Hospital — Washington D.C.
- University of Florida — Gainesville
- University Of Iowa Hospitals And Clinics — Iowa City
- Kennedy Krieger Institute — Baltimore
- Washington University — St Louis
- … and 12 more centers
Publications
- Tian C, Grabich S, Veerapandiyan A, Scharf RJ, Santra S, Hornibrook S, Drummond K, Sehinovych I, Waldrop M. Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD. J Comp Eff Res. 2026 May;15(5):e250108. doi: 10.57264/cer-2025-0108. Epub 2026 Apr 10. PMID 41961051
Identifiers
NCT: NCT06606340 · 4658-403