An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Drug: SRP-5051
- Registration Number
- NCT03675126
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 15
• Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description SRP-5051 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.
- Primary Outcome Measures
Name Time Method Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) Up to approximately 135 weeks 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.
- Secondary Outcome Measures
Name Time Method 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) 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.
Trial Locations
- Locations (8)
Connecticut Children's Medical Center
🇺🇸Hartford, Connecticut, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
NW FL Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, United States
Center for Integrative Rare Disease Research (CIRDR)
🇺🇸Atlanta, Georgia, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
London Health Sciences Centre
🇨🇦London, Ontario, Canada
Children's Medical Center Dallas
🇺🇸Dallas, Texas, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States