A Phase 2 interventional study of Sevasemten 10 mg and Sevasemten 5 mg in Becker Muscular Dystrophy, sponsored by Edgewise Therapeutics, Inc.. Completed at 51 sites in 12 countries. Open to male participants aged 12 Years to 50 Years. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by Edgewise Therapeutics, Inc. · Phase 2, Interventional, and Treatment
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 175 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.
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.
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 175 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 175 adult participants will be randomized in Cohort 6 in a 2:1 ratio either to sevasemten or placebo.
Enrollment for CANYON and GRAND CANYON has been completed.
GRAND CANYON eligibility is listed below.
Key Inclusion Criteria:
Key Exclusion Criteria:
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 10 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 10 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 10 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 5 mg · Drug: Placebo
Drug: Sevasemten Drug: Placebo
Drug: Sevasemten 12.5 mg · Drug: Placebo
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Sevasemten is administered orally once per day
Placebo is administered orally once per day
Number of treatment emergent adverse events in those treated with sevasemten or placebo
All participants
Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Severity of treatment emergent adverse events in those treated with sevasemten or placebo
All participants
Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Adult participants
Time frame: Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in total North Star Ambulatory Assessment score
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in total North Star Ambulatory Assessment score
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2)
Pharmacokinetics as measured by steady state plasma concentration
All participants
Time frame: 12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Change from Baseline in growth as assessed by height centile on World Health Organization growth charts
Adolescent participants
Time frame: 12 months (CANYON Cohorts 4, 5)
Incidence of laboratory test-related treatment emergent adverse events
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum Creatine Kinase
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in plasma fast skeletal muscle Troponin I
Adult participants
Time frame: Averaged across Months 6 and 12 (CANYON Cohorts 1, 2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in serum myoglobin
Adult participants
Time frame: Averaged across Months 6, 9, and 12 (CANYON Cohorts 1,2); 18 months (GRAND CANYON Cohort 6)
Change from Baseline in total NSAD score (CANYON Cohorts 1,2)
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2)
Change from Baseline in 4-stair climb test
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Change from Baseline in the 10-meter walk/run test
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in 100-meter timed test
Adult participants
Time frame: 12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Change from Baseline in stride velocity (95th percentile)
Adult participants
Time frame: 18 Months (GRAND CANYON Cohort 6)
Month 18 change from Baseline in fat fraction of upper leg muscles as assessed by Magnetic Resonance Imaging
Adult participants
Time frame: 18 months (GRAND CANYON Cohort 6)
Plan to share: No
This study is completed, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Edgewise Therapeutics, Inc.