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

Primary Purpose

Muscular Dystrophy, Duchenne

Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
SRP-5051
Sponsored by
Sarepta Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 51, Ambulatory, Pediatric, Nonambulatory, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), Duchenne

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Connecticut Children's Medical Center
  • NW FL Clinical Research Group, LLC
  • Center for Integrative Rare Disease Research (CIRDR)
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • University of Kansas Medical Center
  • UPMC Children's Hospital of Pittsburgh
  • Children's Medical Center Dallas
  • London Health Sciences Centre

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

SRP-5051

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of Participants Experiencing Adverse Events

Secondary Outcome Measures

Plasma Concentration of SRP-5051

Full Information

First Posted
August 6, 2018
Last Updated
September 20, 2023
Sponsor
Sarepta Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03675126
Brief Title
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
Official Title
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Terminated
Why Stopped
The sponsor has decided to integrate 5051-102 into 5051-201. Participants from 5051-102 will be eligible to enroll in 5051-201 Part B.
Study Start Date
December 19, 2018 (Actual)
Primary Completion Date
August 25, 2021 (Actual)
Study Completion Date
August 25, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sarepta Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Duchenne
Keywords
Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 51, Ambulatory, Pediatric, Nonambulatory, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), Duchenne

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
SRP-5051
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
SRP-5051
Other Intervention Name(s)
vesleteplirsen
Intervention Description
SRP-5051 administered as an IV infusion.
Primary Outcome Measure Information:
Title
Number of Participants Experiencing Adverse Events
Time Frame
Up to 152 weeks
Secondary Outcome Measure Information:
Title
Plasma Concentration of SRP-5051
Time Frame
Pre-dose and at multiple time periods after infusion

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Sarepta Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Connecticut Children's Medical Center
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06106
Country
United States
Facility Name
NW FL Clinical Research Group, LLC
City
Gulf Breeze
State/Province
Florida
ZIP/Postal Code
32561
Country
United States
Facility Name
Center for Integrative Rare Disease Research (CIRDR)
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Children's Medical Center Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
Facility Name
London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada

12. IPD Sharing Statement

Learn more about this trial

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

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