A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

• delandistrogene moxeparvovec (Genetic), also known as SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS: No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.

Primary outcomes

• Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) [Up to 10 years]

Secondary outcomes

• Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]
• Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]
• Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]
• Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]
• Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]
• Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]
• Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to the End of the Study Participation of Delandistrogene Moxeparvovec [Baseline, up to 10 years]
• Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]
• Change in Musculoskeletal MRI Findings From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation [Baseline, up to 10 years]

Sex: Male

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:

* Not applicable

NIH grants: 2023