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

NCT03675126 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: 5051-102
• Study Type: interventional
• Target: 15
• Locations: 2 countries
• Sites: 8

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.

Conditions

Muscular Dystrophy, Duchenne

Keywords

Duchenne muscular dystrophy; Exon Skipping; DMD; Exon 51; Ambulatory; Pediatric; Nonambulatory; Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO); Duchenne

Outcome Measures

Primary Outcomes (1)

• Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)

  A TEAE was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.

  Up to approximately 135 weeks

Secondary Outcomes (1)

• Plasma Concentration of SRP-5051

  Day 1, Day 84, every 84 days after Day 84 (up to a maximum of approximately 135 weeks) (pre-dose, immediately prior to end of infusion, up to 4-6 hours post-dose)

Study Arms (1)

SRP-5051

EXPERIMENTAL

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

Interventions

SRP-5051 DRUG

SRP-5051 administered as an IV infusion.

Also known as: vesleteplirsen

SRP-5051

Eligibility Criteria

• Age: 4 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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.
• \* The dose of steroids must remain constant except for modifications to accommodate changes in weight.

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (8)

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

Location

NW FL Clinical Research Group, LLC

Gulf Breeze, Florida, 32561, United States

Location

Center for Integrative Rare Disease Research (CIRDR)

Atlanta, Georgia, 30318, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Children's Medical Center Dallas

Dallas, Texas, 75207, United States

Location

London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Medical Director
• Organization: Sarepta Therapeutics, Inc.

SareptAlly@sarepta.com

1-888-727-3782

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2018
• First Posted: September 18, 2018
• Study Start: December 19, 2018
• Primary Completion: August 25, 2021
• Study Completion: August 25, 2021
• Last Updated: September 19, 2024
• Results First Posted: September 19, 2024

Record last verified: 2024-08

Locations

US (7); CA (1)