Duchenne Muscular Dystrophy MedDRA version: 20.1 Level: PT Classification code 10052655 Term: Duchenne muscular dystrophy gene carrier System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Vesleteplirsen
Product Code: SRP-5051
Pharmaceutical Form: Lyophilisate for solution for infusion
INN or Proposed INN: Vesleteplirsen
CAS Number: 2101570-09-4
Current Sponsor code: SRP-5
Sponsors
Sarepta Therapeutics, Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - 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
Exclusion criteria
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), 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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: - To determine the pharmacokinetics (PK) of SRP-5051 following repeat Q4W administration - 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
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
Canada, United States
Contacts
Public ContactPatient Recruitment
Sarepta Therapeutics, Inc.
Outcome results
None listed