Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
- Registration Number
- NCT04004065
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 62
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A: Vesleteplirsen 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. Part B: Vesleteplirsen 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.
- Primary Outcome Measures
Name Time Method Part B: Change From Baseline in Dystrophin Protein Level at Week 28 Part B: Baseline, Week 28 Part A: Incidence of Adverse Events (AEs) Part A: Baseline up to 75 weeks
- Secondary Outcome Measures
Name Time Method 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
Related Research Topics
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Trial Locations
- Locations (25)
University of California Davis Health
🇺🇸Sacramento, California, United States
Connecticut Children's
🇺🇸Farmington, Connecticut, United States
Northwest Florida Clinical Research Group, LLC
🇺🇸Gulf Breeze, Florida, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
University of Kansas Medical Center Research Inst.
🇺🇸Kansas City, Kansas, United States
University of Massachusetts
🇺🇸Worcester, Massachusetts, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Austin Neuromuscular Center
🇺🇸Austin, Texas, United States
Children's Health Ambulatory Pavilion
🇺🇸Dallas, Texas, United States
Scroll for more (15 remaining)University of California Davis Health🇺🇸Sacramento, California, United States