Summary

Eligibility
for males (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around

Description

Summary

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.

Official Title

A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study

Keywords

Duchenne Muscular Dystrophy, DMD, Pediatric, Long-term, Follow-up, Safety, Efficacy, Muscular Dystrophies, delandistrogene moxeparvovec

Eligibility

You can join if…

Open to males

  • Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study.
  • Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements.

You CAN'T join if...

  • Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers.

Other inclusion/exclusion criteria may apply.

Locations

  • UCLA Medical Center
    Los Angeles California 90095 United States
  • Rady Children's Hospital
    San Diego California 92123 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Sarepta Therapeutics, Inc.
ID
NCT05967351
Phase
Phase 3 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 400 study participants
Last Updated