Duchenne Muscular Dystrophy
Conditions
Brief summary
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Detailed description
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 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 (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
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 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 (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 Reso | — |
Countries
Belgium, Germany, Italy, Spain