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

Korábbi kezelés

• 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.

Laborértékek

• 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%

Egyéb

• Forced vital capacity predicted <60% or using daytime ventilatory support

• 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.

A regiszterben nem jelölt feltételek

• GRAND CANYON eligibility is listed below.