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

This is an interventional treatment trial for Becker Muscular Dystrophy focused on measuring Becker Muscular Dystrophy Read More

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, OR Adolescents (12 to 17 years, inclusive) with genetic confirmation of an in-frame dystrophin mutation and a phenotype consistent with Becker muscular dystrophy as determined by the Investigator
2. Able to complete the 100-meter timed test in < 150 seconds.
3. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 10 to 32, inclusive for adults or a score of greater than 10 for adolescents.
4. Vaccinated against SARS-CoV-2 per local requirements

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 >5 days in the previous 6 months at a dose of >5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted.
5. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.