A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
Overview
- Phase
- Phase 2
- Intervention
- Vesleteplirsen
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- Sarepta Therapeutics, Inc.
- Enrollment
- 62
- Locations
- 25
- Primary Endpoint
- Part B: Change From Baseline in Dystrophin Protein Level at Week 28
- Status
- Terminated
- Last Updated
- last year
Overview
Brief Summary
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Part A: Vesleteplirsen
Participants received escalating dose levels of vesleteplirsen, every 4 weeks, via intravenous (IV) infusion for up to 75 weeks during Part A. Once the doses have been selected for Part B, all participants who have completed Part A will transition to Part B.
Intervention: Vesleteplirsen
Part B: Vesleteplirsen
Participants received vesleteplirsen at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 5 years. This included the participants who rolled over from Part A, as well as the additional participants who enrolled at the beginning of Part B.
Intervention: Vesleteplirsen
Outcomes
Primary Outcomes
Part B: Change From Baseline in Dystrophin Protein Level at Week 28
Time Frame: Part B: Baseline, Week 28
Part A: Incidence of Adverse Events (AEs)
Time Frame: Part A: Baseline up to 75 weeks
Secondary Outcomes
- Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28(Part B: Baseline, Week 28)
- Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen(Pre-dose and at multiple time points (up to 32 hours) after end of infusion)
- Part A: PK: Urine Concentration of Vesleteplirsen(Pre-dose and at multiple time periods (up to 48 hours) after end of infusion)
- Part B: Change From Baseline in Exon-Skipping Levels at Week 28(Part B: Baseline, Week 28)
- Part B: Incidence of Adverse Events (AEs)(Part B: Baseline up to Week 304)
- Part B: PK: Plasma Concentration of Vesleteplirsen(Part B predose and at multiple timepoints (up to 48 hours) after end of infusion)
- Part B: PK: Urine Concentration of Vesleteplirsen(Part B predose and at multiple timepoints (up to 48 hours) after end of infusion)