NCT05291091

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 244 (actual)
• Sponsor: Edgewise Therapeutics, Inc. · Industry
• Sex: Male
• Age: 12 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker. CANYON and GRAND CANYON are fully enrolled.

Detailed description

The EDG-5506-201 protocol was amended to include an additional cohort thus consists of two parts. Part 1: CANYON is a double-blind, randomized, placebo-controlled design to investigate the effect of sevasemten on the safety, pharmacokinetics, biomarkers, and functional measures. Approximately 32 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 32 adult participants will randomize to Cohort 1 or Cohort 2 in a 1:1 ratio then each cohort will further randomize to sevasemten or placebo in a 3:1 ratio. Approximately 9 adolescent participants will enroll in Cohort 4 and randomize in a 2:1 ratio to sevasemten or placebo. Cohort 5 will randomize an additional 9 participants in a 2:1 ratio to either sevasemten or placebo after Cohort 4. CANYON is now fully enrolled. Part 2: GRAND CANYON or Cohort 6 is a double-blind, randomized, placebo-controlled design to investigate the safety and efficacy of sevasemten in adults with Becker muscular dystrophy after 18 months of treatment. Approximately 120 adults with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, an 18-month Treatment period, followed by a 4-week follow-up period. Approximately 120 adult participants will be randomized in Cohort 6 in a 2:1 ratio either to sevasemten or placebo.

Conditions

• Becker Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Sevasemten 10 mg	Sevasemten is administered orally once per day
DRUG	Sevasemten 5 mg	Sevasemten is administered orally once per day
DRUG	Sevasemten 12.5 mg	Sevasemten is administered orally once per day
DRUG	Placebo	Placebo is administered orally once per day

Timeline

• Start date: 2022-11-10
• Primary completion: 2026-09-01
• Completion: 2026-09-01
• First posted: 2022-03-22
• Last updated: 2026-03-24

Locations

51 sites across 12 countries: United States, Australia, Belgium, Denmark, France, Germany, Israel, Italy, Netherlands, New Zealand, Spain, United Kingdom

Regulatory

• FDA-regulated drug study