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

Purpose

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.

Condition

  • Muscular Dystrophy, Duchenne

Eligibility

Eligible Ages
Over 4 Years
Eligible Genders
Male
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
SRP-5051 		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
    SRP-5051 administered as an IV infusion.
    Other names:
    • vesleteplirsen

More Details

Status
Terminated
Sponsor
Sarepta Therapeutics, Inc.

Study Contact