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

Stopped early · Phase 1/Phase 2

Conditions studied: Muscular Dystrophy, Duchenne

In brief

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.

Key facts

Study ID

NCT03675126

Run by

Sarepta Therapeutics, Inc.

People needed

15

Starts

2018-12-19

Expected to finish

2021-08-25

Last updated by the study team

2024-09-19

Who can join

Age: 4 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.

You may not qualify if…

• 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.

Where it is running

• Connecticut Children's Medical Center — Hartford, Connecticut, United States
• NW FL Clinical Research Group, LLC — Gulf Breeze, Florida, United States
• Center for Integrative Rare Disease Research (CIRDR) — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Children's Medical Center Dallas — Dallas, Texas, United States
• London Health Sciences Centre — London, Ontario, Canada

Full record on ClinicalTrials.gov

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