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

Launched by EDGEWISE THERAPEUTICS, INC. · Mar 14, 2022

Keywords

ClinConnect Summary

The GRAND CANYON trial is a study testing a new drug called sevasemten (EDG-5506) for adults with Becker Muscular Dystrophy, a condition that affects muscle strength. This study aims to see how safe and effective the drug is in helping people with this condition. Currently, the trial is looking for adult participants aged 18 to 50 who have a specific type of genetic change related to Becker muscular dystrophy, and who have been able to walk independently for many years. Participants will need to complete a timed walking test and demonstrate their ability to perform certain activities related to movement.

Those who join the study will be randomly assigned to receive either the drug or a placebo (a treatment that looks the same but has no active ingredients) to see how well it works. Participants can expect regular check-ups and assessments throughout the study to monitor their health and progress. It's important to note that some people may not qualify if they have certain health issues or have taken specific medications recently. If you or someone you know has Becker muscular dystrophy and fits the criteria, this might be a good opportunity to contribute to research that could help others in the future.

Gender

MALE

Eligibility criteria

• • The CANYON Study including the adolescent cohorts are fully enrolled.
• • GRAND CANYON eligibility is listed below.
• Key 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.

Trial Officials