Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
Purpose
A study of EDG-5506 in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON. GRAND CANYON is designed similar to a Phase 3 study and seeks to demonstrate whether the safety and efficacy of EDG-5506 are sufficient to support a marketing application. It is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker muscular dystrophy. CANYON is fully enrolled; GRAND CANYON is currently enrolling.
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. 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.
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 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.
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 6 |
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
The EDG-5506-201 protocol was amended to include an additional cohort thus consists of two parts. Part 1: CANYON is a double-blind, randomized, placebo-controlled design to investigate the effect of EDG-5506 on the safety, pharmacokinetics, biomarkers, and functional measures. Approximately 32 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 32 adult participants will randomize to Cohort 1 or Cohort 2 in a 1:1 ratio then each cohort will further randomize to EDG-5506 or placebo in a 3:1 ratio. Approximately 9 adolescent participants will enroll in Cohort 4 and randomize in a 2:1 ratio to EDG-5506 or placebo. Cohort 5 will randomize an additional 9 participants in a 2:1 ratio to either EDG-5506 or placebo after Cohort 4. CANYON is now fully enrolled. Part 2: GRAND CANYON or Cohort 6 is a double-blind, randomized, placebo-controlled design to investigate the safety and efficacy of EDG-5506 in adults with Becker muscular dystrophy after 18 months of treatment. Approximately 120 adults with Becker muscular dystrophy are planned to enroll in this study. This study will have up to a 4-week Screening period, an 18-month Treatment period, followed by a 4-week follow-up period. Approximately 120 adult participants will be randomized in Cohort 6 in a 2:1 ratio either to EDG-5506 or placebo.