Summary

Eligibility
for males (full criteria)
Location
at UCLA
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
  • Lucile Packard Children's Hospital Stanford (LPCH)
    Palo Alto California 94304 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