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

September 20, 2023 updated by: Sarepta Therapeutics, Inc.

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • London, Ontario, Canada, N6A 5W9
        • London Health Sciences Centre
  • United States
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Connecticut Children's Medical Center
    • Florida
      • Gulf Breeze, Florida, United States, 32561
        • NW FL Clinical Research Group, LLC
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Center for Integrative Rare Disease Research (CIRDR)
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • UPMC Children's Hospital of Pittsburgh
    • Texas
      • Dallas, Texas, United States, 75207
        • Children's Medical Center Dallas

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants Experiencing Adverse Events
Time Frame: Up to 152 weeks
Up to 152 weeks

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Investigators

  • Study Director: Medical Director, Sarepta Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 19, 2018

Primary Completion (Actual)

August 25, 2021

Study Completion (Actual)

August 25, 2021

Study Registration Dates

First Submitted

August 6, 2018

First Submitted That Met QC Criteria

September 14, 2018

First Posted (Actual)

September 18, 2018

Study Record Updates

Last Update Posted (Actual)

September 28, 2023

Last Update Submitted That Met QC Criteria

September 20, 2023

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 5051-102

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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