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

• Status: Active, not recruiting
• Trial end date: 2024-07-31
• Target enrollment: 0
• Participant gender: Male

Summary

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sarepta Therapeutics; Sarepta Therapeutics, Inc.

Criteria

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.