Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (CANYON)
Purpose
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, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy.
Condition
- Becker Muscular Dystrophy
Eligibility
- Eligible Ages
- Between 12 Years and 50 Years
- Eligible Genders
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Adult Cohort 1 |
Drug: EDG-5506 Drug: Placebo |
|
|
Experimental Adult Cohort 2 |
Drug: EDG-5506 Drug: Placebo |
|
|
Experimental Adult Cohort 3 |
Drug: EDG-5506 Drug: Placebo |
|
|
Experimental Adolescent Cohort 4 |
Drug: EDG-5506 Drug: Placebo |
|
|
Experimental Adolescent Cohort 5 |
Drug: EDG-5506 Drug: Placebo |
|
Recruiting Locations
Kansas City, Kansas 66160
More Details
- Status
- Recruiting
- Sponsor
- Edgewise Therapeutics, Inc.
Detailed Description
This study is a double-blind, randomized, placebo-controlled design to investigate the effect of EDG-5506 on the safety, pharmacokinetics, biomarkers and functional measures. Approximately 48 adults and 18 adolescents with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, a 12 month Treatment period, followed by a 4-week follow-up period. Approximately 48 adult participants will randomize to Cohort 1 (Dose 1) or Cohort 2 (Dose 2) in a 1:1 ratio then each cohort will further randomize to EDG-5506 or placebo in a 3:1 ratio. Cohort 3 (Dose 3) will randomize participants in a 3:1 ratio to either EDG-5506 or placebo and will enroll after Cohort 2. Approximately 9 adolescent participants will enroll in Cohort 4 (Dose 4) and randomize in a 2:1 ratio to EDG-5506 or placebo. Cohort 5 (Dose 5) will randomize an additional 9 participants in a 2:1 ratio to either EDG-5506 or placebo after Cohort 4.