A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
About
Brief 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.
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Join this Trial
Enrolling by Invitation
These studies are not open to everyone who meets the eligibility criteria, but
only to people in that particular population, who are specifically invited to participate.
Study Stats
Protocol No.
23-000873
Category
Genetic and Rare Diseases
Musculoskeletal Disorders
Pediatric and Prenatal Disorders
Principal Investigator
Contact
Location
- UCLA Westwood