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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
62
Vesleteplirsen injection, for IV use
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
...and 15 more locations
Part A: Incidence of Adverse Events (AEs)
Time frame: Part A: Baseline up to 75 weeks
Part B: Change From Baseline in Dystrophin Protein Level at Week 28
Time frame: Part B: Baseline, Week 28
Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen
Time frame: Pre-dose and at multiple time points (up to 32 hours) after end of infusion
Part A: PK: Urine Concentration of Vesleteplirsen
Time frame: 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
Time frame: Part B: Baseline, Week 28
Part B: Incidence of Adverse Events (AEs)
Time frame: Part B: Baseline up to Week 304
Part B: PK: Plasma Concentration of Vesleteplirsen
Time frame: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion
Part B: PK: Urine Concentration of Vesleteplirsen
Time frame: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion
Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28
Time frame: Part B: Baseline, Week 28
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