An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

5051-102

The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

2018-12-19

2021-08-25

2021-08-25

Terminated

Early phase I

15

Inclusion Criteria: • Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102. Exclusion Criteria: * Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) since completing a study administering SRP-5051 and while participating in this study for any of the following: angiotensin converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), beta-blockers, potassium and steroids\*. * Requires antiarrhythmic and/or diuretic therapy for heart failure. * Use of any herbal medication/supplement containing aristolochic acid. * Treatment with any experimental therapy since entering original study or any experimental gene therapy for the treatment of DMD at any time. * Participation in an interventional clinical trial since completing original study. Other inclusion/exclusion criteria apply. \* The dose of steroids must remain constant except for modifications to accommodate changes in weight.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 15, 'type': 'ACTUAL'}}

2023-09-28