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

Phase 2

Active, not recruiting

• Conditions: Becker Muscular Dystrophy
• Interventions: Drug: Sevasemten 10 mg; Drug: Sevasemten 5 mg; Drug: Sevasemten 12.5 mg; Drug: Placebo

• Registration Number: NCT05291091
• Lead Sponsor: Edgewise Therapeutics, Inc.

Brief Summary

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 120 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.

CANYON is fully enrolled; GRAND CANYON is currently enrolling.

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 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.

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 sevasemten 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 sevasemten or placebo.

Study Timeline

• Study First Submitted: 2022-03-14
• Study First Submitted QC: 2022-03-14
• Study First Posted: 2022-03-22
• Study Start: 2022-07-06
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-02
• Last Update Posted: 2025-07-04
• Primary Completion: 2026-08
• Study Completion: 2026-08-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 175

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Adult Cohort 2	Placebo	Drug: Sevasemten Drug: Placebo
Adult Cohort 6	Sevasemten 10 mg	Drug: Sevasemten Drug: Placebo
Adult Cohort 6	Placebo	Drug: Sevasemten Drug: Placebo
Adolescent Cohort 4	Sevasemten 5 mg	Drug: Sevasemten Drug: Placebo
Adolescent Cohort 4	Placebo	Drug: Sevasemten Drug: Placebo
Adolescent Cohort 5	Sevasemten 12.5 mg	Drug: Sevasemten Drug: Placebo
Adolescent Cohort 5	Placebo	Drug: Sevasemten Drug: Placebo
Adult Cohort 1	Placebo	Drug: Sevasemten Drug: Placebo
Adult Cohort 2	Sevasemten 10 mg	Drug: Sevasemten Drug: Placebo
Adult Cohort 1	Sevasemten 10 mg	Drug: Sevasemten Drug: Placebo

Primary Outcome Measures

Name	Time	Method
Number of adverse events in those treated with sevasemten or placebo	12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)	All participants
Severity of adverse events in those treated with sevasemten or placebo	12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)	All participants
Change from Baseline in serum Creatine Kinase	12 Months (CANYON Cohorts 1, 2)	Adult participants
Change from Baseline in the North Star Ambulatory Assessment scale	18 months (GRAND CANYON Cohort 6)	Adult participants

Secondary Outcome Measures

Name	Time	Method
Change from Baseline in the protein fast skeletal muscle Troponin I	12 months (CANYON Cohorts 1, 2), 18 months (GRAND CANYON Cohort 6)	Adult participants
Change from Baseline in the North Star Ambulatory Assessment scale	12 Months (CANYON Cohorts 1, 2)	Adult participants
Change from Baseline in the North Star Assessment for Limb-Girdle Type Muscular Dystrophies scale	12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)	Adult participants
Change from Baseline in the 10-meter walk/run test	12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)	Adult participants
Change from Baseline in 100-meter timed test	12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)	Adult participants
Change from Baseline in stride velocity (95th percentile)	18 Months (GRAND CANYON Cohort 6)	Adult participants
Pharmacokinetics as measured by steady state plasma concentration	12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)	All participants
Change from Baseline in growth as assessed by height centile on World Health Organization growth charts	12 months (CANYON Cohorts 4, 5)	Adolescent participants
Month 18 change from Baseline in fat fraction of upper leg muscles as assessed by Magnetic Resonance Imaging	18 months (GRAND CANYON Cohort 6)	Adult participants

Trial Locations

Locations (51)

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

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