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A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study

Status
Recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-505043-39-00
Acronym
SRP-9001-305
Enrollment
75
Registered
2024-03-14
Start date
2024-05-24
Completion date
Unknown
Last updated
2025-10-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

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

Sarepta Therapeutics Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
Male
Age
0 Years to 64 Years

Design outcomes

Primary

MeasureTime frame
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)

Secondary

MeasureTime 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

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026