A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
- Conditions
- Duchenne Muscular Dystrophy
- Registration Number
- NCT05967351
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 400
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) Up to 5 years after dosing
- Secondary Outcome Measures
Name Time Method Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec Baseline, 5 years
Trial Locations
- Locations (37)
Oxford University Hospitals NHS Foundation Trust
🇬🇧Oxford, Oxfordshire, United Kingdom
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
UCLA Medical Center
🇺🇸Los Angeles, California, United States
Lucile Packard Children's Hospital Stanford (LPCH)
🇺🇸Palo Alto, California, United States
University of California, Davis
🇺🇸Sacramento, California, United States
Rady Children's Hospital
🇺🇸San Diego, California, United States
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
University of Florida Clinical Research Center
🇺🇸Gainesville, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Iowa ICTS Clinical Research Unit
🇺🇸Iowa City, Iowa, United States
Scroll for more (27 remaining)Oxford University Hospitals NHS Foundation Trust🇬🇧Oxford, Oxfordshire, United Kingdom