Active — Not RecruitingPhase 2

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

United States244 participantsStarted 2022-11-10

Plain-language 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.

Who can participate

Age range12 Years – 50 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids.
✓. Able to complete the 100-meter timed test in \< 200 seconds with or without use of mobility aid devices.
✓. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.

Exclusion criteria

✕. Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia.
✕. Cardiac echocardiogram ejection fraction \< 40%
✕. Forced vital capacity predicted \<60% or using daytime ventilatory support
✕. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
✕. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.

What they're measuring

Number of adverse events in those treated with sevasemten or placebo

Timeframe: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

Severity of adverse events in those treated with sevasemten or placebo

Timeframe: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

Change from Baseline in serum Creatine Kinase

Timeframe: 12 Months (CANYON Cohorts 1, 2)

Change from Baseline in the North Star Ambulatory Assessment scale

Timeframe: 18 months (GRAND CANYON Cohort 6)

Trial details

NCT IDNCT05291091

SponsorEdgewise Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-09

View on ClinicalTrials.gov More Becker Muscular Dystrophy trials

Plain-language summary

Who can participate

Age range12 Years – 50 Years

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids.
✓. Able to complete the 100-meter timed test in \< 200 seconds with or without use of mobility aid devices.
✓. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.

Exclusion criteria

✕. Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia.
✕. Cardiac echocardiogram ejection fraction \< 40%
✕. Forced vital capacity predicted \<60% or using daytime ventilatory support
✕. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
✕. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.

What they're measuring

Number of adverse events in those treated with sevasemten or placebo

Timeframe: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

Severity of adverse events in those treated with sevasemten or placebo

Timeframe: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

Change from Baseline in serum Creatine Kinase

Timeframe: 12 Months (CANYON Cohorts 1, 2)

Change from Baseline in the North Star Ambulatory Assessment scale

Timeframe: 18 months (GRAND CANYON Cohort 6)