This study is a double-blind, randomized, placebo-controlled design to investigate the effect of EDG-5506 on the safety, pharmacokinetics, biomarkers and functional measures. Approximately 48 adults and 18 adolescents with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, a 12 month Treatment period, followed by a 4-week follow-up period.~Approximately 48 adult participants will randomize to Cohort 1 (Dose 1) or Cohort 2 (Dose 2) in a 1:1 ratio then each cohort will further randomize to EDG-5506 or placebo in a 3:1 ratio. Cohort 3 (Dose 3) will randomize participants in a 3:1 ratio to either EDG-5506 or placebo and will enroll after Cohort 2.~Approximately 9 adolescent participants will enroll in Cohort 4 (Dose 4) and randomize in a 2:1 ratio to EDG-5506 or placebo. Cohort 5 (Dose 5) will randomize an additional 9 participants in a 2:1 ratio to either EDG-5506 or placebo after Cohort 4.
The CANYON study is a multicenter, randomized, Phase 2, double-blind, placebo-controlled study to evaluate the effect of EDG-5506 on the safety, pharmacokinetics, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy.