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

Trial ID: NCT05291091

Interventional - Phase 2 - Completed

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.

Conditions

• Becker Muscular Dystrophy

Interventions

• Sevasemten 10 mg
• Sevasemten 5 mg
• Sevasemten 12.5 mg
• Placebo

Eligibility

Inclusion criteria

• 1. 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.
• 2. Able to complete the 100-meter timed test in \< 200 seconds with or without use of mobility aid devices.
• 3. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.
• Key

Exclusion criteria

• 1. 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.
• 2. Cardiac echocardiogram ejection fraction \< 40%
• 3. Forced vital capacity predicted \<60% or using daytime ventilatory support
• 4. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
• 5. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.

Sponsor

Edgewise Therapeutics, Inc., Medpace, Inc., ImagingNMD, SYSNAV

Source: ClinicalTrials.gov

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