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

JRCT ID: jRCT2053240056

Registered date:10/06/2024

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Duchenne muscular dystrophy (DMD)
• Date of first enrollment: 10/06/2024
• Target sample size: 10
• Countries of recruitment: US,Japan,UK,Japan,Belgium,Japan,Spain,Japan,Germany,Japan,Italy,Japan,Hong Kong,Japan,Taiwan,Japan,France,Japan,Sweden,Japan,Israel,Japan,Australia,Japan,Canada,Japan,South Korea,Japan
• Study type: Observational

Outcome(s)

• Primary Outcome: 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

- Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
- Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Male

Include 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.

• Exclude criteria: Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers.

Related Information

• Primary Sponsor: O Malley Patrick
• Secondary Sponsor: Hoffmann-La Roche
• Secondary ID(s): NCT05967351