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

Phase 2TerminatedNCT03675126

Sarepta Therapeutics, Inc.15 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Muscular Dystrophy, Duchenne

Interventions

SRP-5051DRUG

SRP-5051 administered as an IV infusion.

Eligibility

Sex: MALEMin age: 4 Years

Medical Language ↔ Plain English

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.

Locations (8)

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

Outcomes

Primary Outcomes

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.

Time frame: Up to approximately 135 weeks

Secondary Outcomes

Plasma Concentration of SRP-5051

For pharmacokinetic (PK) analysis, plasma samples were collected pre-dose (approximately 30 minutes prior to the start of infusion), immediately prior to the end of infusion (prior to flush), and approximately 4 to 6 hours after the end of dosing. Results are reported in micrograms/liter (ug/L) and are presented as an average across the days specified in the time frame.

Time frame: 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)

Data from ClinicalTrials.gov

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