A Study for Participants With Duchenne Muscular Dystrophy (DMD) Who Participated in Studies of SRP-5051
- Conditions
- Duchenne Muscular DystrophyMedDRA version: 20.1Level: PTClassification code 10052655Term: Duchenne muscular dystrophy gene carrierSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2020-004803-15-Outside-EU/EEA
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Male
- Target Recruitment
- Not specified
- Has completed a study of SRP-5051 and continues to meet the Eligibility Criteria of Study 5051-102.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0
- 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), betablockers, 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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this study is to evaluate the adverse event (AE) profile of repeat every-4-week (Q4W) administration of SRP-5051.;Secondary Objective: The secondary objectives of this study are to:<br>- To determine the pharmacokinetics (PK) of SRP-5051 following repeat Q4W administration<br>- To evaluate additional safety and tolerability of repeat Q4W administration of SRP-5051;Primary end point(s): Number of Participants Experiencing Adverse Events;Timepoint(s) of evaluation of this end point: Up to 152 weeks
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Plasma Concentration of SRP-5051;Timepoint(s) of evaluation of this end point: Pre-dose and at multiple time periods after infusion