NCT05540860

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

A 2-part 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

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 76 (actual)
• Sponsor: Edgewise Therapeutics, Inc. · Industry
• Sex: Male
• Age: 4 Years – 9 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This is a 2-part, multi-center, Phase 2 study to evaluate the effect of sevasemten (EDG-5506) on safety, pharmacokinetics and biomarkers of muscle damage in approximately 72 children with DMD treated with oral, once-daily sevasemten for up to 48 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), up to a 196-week open-label extension period (Part B), and a 2-week follow up period. Approximately 72 participants aged 4 to 9 years inclusive will be randomized to sevasemten or placebo in a 2:1 ratio. Five dose cohorts (C1, C2, C3, C4 and C5) of approximately 9 participants each will be enrolled sequentially. Approximately 18 total additional participants may be added across Cohorts 2, 3, or 4. An additional cohort, Cohort 2NS, to include participants (aged 4 to 7 years inclusive) not currently treated with corticosteroids, will enroll approximately 9 participants after Cohort 2 safety review and in parallel with the additional cohorts. After review of emerging data, the protocol was amended so all dose cohorts receive the same dose in Part B.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Sevasemten Dose 1	Sevasemten is administered orally once per day
DRUG	Sevasemten Dose 2	Sevasemten is administered orally once per day
DRUG	Sevasemten Dose 3	Sevasemten is administered orally once per day
DRUG	Sevasemten Dose 4	Sevasemten is administered orally once per day
DRUG	Sevasemten Dose 5	Sevasemten is administered orally once per day
DRUG	Placebo	Placebo is administered orally once per day

Timeline

• Start date: 2022-10-24
• Primary completion: 2027-01-01
• Completion: 2027-01-01
• First posted: 2022-09-15
• Last updated: 2025-11-12

Locations

14 sites across 1 country: United States

Regulatory

• FDA-regulated drug study