Long-term Follow-up for Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy
(EXPEDITION Trial)

Recruiting in Palo Alto (17 mi)

+37 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Sarepta Therapeutics, Inc.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

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.

Research Team

Eligibility Criteria

This study is for individuals with Duchenne muscular dystrophy who previously received delandistrogene moxeparvovec. Participants must either have a parent or caregiver, or be adults who understand and can follow the study's schedule and requirements.

Inclusion Criteria

I have received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a past study.

I am over 18 or have a caregiver and can follow the study plan.

Exclusion Criteria

I prefer not to share my trial participation with my doctor or other healthcare providers.

Treatment Details

Interventions

delandistrogene moxeparvovec (Gene Therapy)

Trial OverviewThe trial monitors long-term safety and effectiveness of delandistrogene moxeparvovec in participants from an earlier study. No new drug will be given; it tracks health for at least 5 years after the initial treatment.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Delandistrogene MoxeparvovecExperimental Treatment1 Intervention

Participant received delandistrogene moxeparvovec in a previous clinical study.