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

Study Overview
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Brief Summary
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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.

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

Conditions
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Muscular Dystrophy, Duchenne

Intervention / Treatment
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* Drug: SRP-5051


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 4 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SRP-5051<br>Participants will receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 studies. | Drug: SRP-5051<br>* SRP-5051 administered as an IV infusion.<br>* Other names: vesleteplirsen;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Experiencing Adverse Events |  | Up to 152 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Plasma Concentration of SRP-5051 |  | Pre-dose and at multiple time periods after infusion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Nonambulatory, Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 51, Ambulatory, Pediatric, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), Duchenne