Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (CANYON)
Study Details
Study Description
Brief 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, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Adult Cohort 1 Drug: EDG-5506 Drug: Placebo |
Drug: EDG-5506 Dose 1
EDG-5506 is administered orally once per day
Drug: Placebo
Placebo is administered orally once per day
|
| Experimental: Adult Cohort 2 Drug: EDG-5506 Drug: Placebo |
Drug: EDG-5506 Dose 2
EDG-5506 is administered orally once per day
Drug: Placebo
Placebo is administered orally once per day
|
| Experimental: Adult Cohort 3 Drug: EDG-5506 Drug: Placebo |
Drug: EDG-5506 Dose 3
EDG-5506 is administered orally once per day
Drug: Placebo
Placebo is administered orally once per day
|
| Experimental: Adolescent Cohort 4 Drug: EDG-5506 Drug: Placebo |
Drug: EDG-5506 Dose 4
EDG-5506 is administered orally once per day
Drug: Placebo
Placebo is administered orally once per day
|
| Experimental: Adolescent Cohort 5 Drug: EDG-5506 Drug: Placebo |
Drug: EDG-5506 Dose 5
EDG-5506 is administered orally once per day
Drug: Placebo
Placebo is administered orally once per day
|
Outcome Measures
Primary Outcome Measures
- Number of adverse events in those treated with EDG-5506 or placebo [12 months]
All participants
- Severity of adverse events in those treated with EDG-5506 or placebo [12 months]
All participants
- Change from Baseline in serum Creatine Kinase [12 Months]
Adult participants
Secondary Outcome Measures
- Change from Baseline in fat fraction of upper leg muscles assessed by Magnetic Resonance Imaging [12 Months]
Adult participants
- Change from Baseline in the North Star Ambulatory Assessment scale [12 Months]
Adult participants
- Change from Baseline in the 10-meter walk/run test [12 Months]
Adult participants
- Change from Baseline in 100-meter timed test [12 Months]
Adult participants
- Change from Baseline in 4-stair climb [12 Months]
Adult participants
- Pharmacokinetics as measured by steady state plasma concentration [12 Months]
All participants
Eligibility Criteria
Criteria
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
-
Able to complete the 100-meter timed test in < 150 seconds.
-
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.
-
Vaccinated against SARS-CoV-2 per local requirements
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.
-
Cardiac echocardiogram ejection fraction < 40%
-
Forced vital capacity predicted <60% or using daytime ventilatory support
-
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.
-
Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Massachusetts | Worcester | Massachusetts | United States | 01605 |
Sponsors and Collaborators
- Edgewise Therapeutics, Inc.
- Medpace, Inc.
Investigators
- Study Chair: Sam Collins, MBBS, PhD, Edgewise Therapeutics, Inc.
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- EDG-5506-201