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

Muscular Dystrophy, Duchenne Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03675126
• Other study ID #: 5051-102
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: December 19, 2018
• Est. completion date: August 25, 2021

Study information

• Verified date: February 2023
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: August 25, 2021
• Est. primary completion date: August 25, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• SRP-5051
SRP-5051 administered as an IV infusion.

Locations

Country	Name	City	State
Canada	London Health Sciences Centre	London	Ontario
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	Children's Medical Center Dallas	Dallas	Texas
United States	NW FL Clinical Research Group, LLC	Gulf Breeze	Florida
United States	Connecticut Children's Medical Center	Hartford	Connecticut
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants Experiencing Adverse Events		Up to 152 weeks
Secondary	Plasma Concentration of SRP-5051		Pre-dose and at multiple time periods after infusion