Summary

Eligibility
for males (full criteria)
Location
at UC Davis
Dates
study started
completion around

Description

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.

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

Keywords

Duchenne Muscular Dystrophy, DMD, Post-marketing, Observational, Phase 4, Eteplirsen, Golodirsen, Casimersen, Muscular Dystrophies

Eligibility

You can join if…

Open to males

  • 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.

You CAN'T join if...

  • 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/exclusion criteria may apply.

Locations

  • University of California Davis Medical Center
    Sacramento California 95817 United States
  • Children's Hospital Los Angeles
    Los Angeles California 90027 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Sarepta Therapeutics, Inc.
ID
NCT06606340
Study Type
Observational
Participants
Expecting 300 study participants
Last Updated