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

Edgewise Therapeutics, Inc.244 enrolled

Overview

A study of sevasemten (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 175 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker.

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 sevasemten 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 sevasemten or placebo in a 3:1 ratio. Approximately 9 adolescent participants will enroll in Cohort 4 and randomize in a 2:1 ratio to sevasemten or placebo. Cohort 5 will randomize an additional 9 participants in a 2:1 ratio to either sevasemten or placebo after Cohort 4. Part 2: GRAND CANYON or Cohort 6 is a double-blind, randomized, placebo-controlled design to investigate the safety and efficacy of sevasemten in adults with Becker muscular dystrophy after 18 months of treatment. Approximately 175 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 175 adult participants will be randomized in Cohort 6 in a 2:1 ratio either to sevasemten or placebo. Enrollment for CANYON and GRAND CANYON has been completed.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

244

Conditions

Becker Muscular Dystrophy

Interventions

Sevasemten 10 mgDRUG

Sevasemten is administered orally once per day

Sevasemten 5 mgDRUG

Sevasemten is administered orally once per day

Sevasemten 12.5 mgDRUG

Sevasemten is administered orally once per day

Eligibility

Sex: MALEMin age: 12 YearsMax age: 50 Years

Medical Language ↔ Plain English

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.

Locations (51)

Outcomes

Primary Outcomes

Number of treatment emergent adverse events in those treated with sevasemten or placebo

All participants

Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

Severity of treatment emergent adverse events in those treated with sevasemten or placebo

All participants

Time frame: 12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

Change from Baseline in serum Creatine Kinase

Adult participants

Time frame: Averaged across Months 6, 9, 12 Months (CANYON Cohorts 1, 2)

Change from Baseline in total North Star Ambulatory Assessment score

Adult participants

Time frame: 18 months (GRAND CANYON Cohort 6)

Secondary Outcomes

Change from Baseline in total North Star Ambulatory Assessment score

Adult participants

Time frame: 12 Months (CANYON Cohorts 1, 2)

Pharmacokinetics as measured by steady state plasma concentration

All participants

Time frame: 12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

Change from Baseline in growth as assessed by height centile on World Health Organization growth charts

Adolescent participants

Time frame: 12 months (CANYON Cohorts 4, 5)

Incidence of laboratory test-related treatment emergent adverse events

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Arkansas Children's Hospital

Little Rock, Arkansas, United States

UC San Diego

La Jolla, California, United States

UCLA Medical Center

Los Angeles, California, United States

UC Irvine Medical Center

Orange, California, United States

Stanford Neuroscience Health Center

Palo Alto, California, United States

UC Davis Medical Center

Sacramento, California, United States

UC Denver

Aurora, Colorado, United States

University of Florida

Gainesville, Florida, United States

Rare Disease Research

Atlanta, Georgia, United States

Northwestern University

Chicago, Illinois, United States

...and 41 more locations