This is a 2-part, multi-center, Phase 2 study to evaluate the effect of EDG-5506 on safety, pharmacokinetics and biomarkers of muscle damage in approximately 27 children with DMD treated with oral, once-daily EDG-5506 for 12 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), a 40-week open-label extension period (Part B), and a 2-week follow up period.~Twenty-seven (27) participants aged 4 to 9 years inclusive will be randomized to EDG-5506 or placebo in a 2:1 ratio. Three dose cohorts will be enrolled sequentially. Within each cohort, randomization will be stratified by age at screening (< 7.0 or ≥ 7.0 years old).
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.