Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy

• Status: Not yet recruiting
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: Male

Summary

The CANYON study is a multicenter, randomized, Phase 2, double-blind, placebo-controlled study to evaluate the effect of EDG-5506 on the safety, pharmacokinetics (PK), biomarkers, and functional measures in adults and adolescents with Becker Muscular Dystrophy (BMD).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Edgewise Therapeutics, Inc.
• Collaborator: Medpace, Inc.

Criteria

Inclusion Criteria:

1. Adults (aged 18 to 50 years, inclusive) with a documented in-frame dystrophin mutation and phenotype consistent with BMD, 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 BMD as determined by the Investigator

2. Able to complete the 100-meter timed test in < 180 seconds.

3. Able to perform the North Star Ambulatory Assessment (NSAA) and achieve a score of 10 to 32, inclusive.

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 (MRI) such as non MR compatible implanted medical devices or severe claustrophobia.

2. Cardiac echocardiogram ejection fraction < 40%

3. Forced vital capacity (FVC) 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.