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

NCT05291091 · tracked via the Priya Life Science UK tracker

Sponsor

Edgewise Therapeutics, Inc.

Phase

Phase 2

Started

2022-11-10

Last updated

2026-07-31

Condition(s) studied

Becker Muscular Dystrophy

Investigational drug(s) / intervention(s)

Sevasemten 10 mgSevasemten 5 mgSevasemten 12.5 mgPlacebo

Sevasemten 10 mg: Sevasemten is administered orally once per day

Sevasemten 5 mg: Sevasemten is administered orally once per day

Sevasemten 12.5 mg: Sevasemten is administered orally once per day

Placebo: Placebo is administered orally once per day

Study 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 and GRAND CANYON are fully enrolled.

Eligibility

Sex

MALE

Min age

12 Years

Max age

50 Years

Healthy volunteers

The CANYON Study including the adolescent cohorts are fully enrolled. 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.

Primary outcome measure(s)

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

Trial sites (51)

Facility	City	Region
Arkansas Children's Hospital	Little Rock	Arkansas
UC San Diego	La Jolla	California
UCLA Medical Center	Los Angeles	California
UC Irvine Medical Center	Orange	California
Stanford Neuroscience Health Center	Palo Alto	California
UC Davis Medical Center	Sacramento	California
UC Denver	Aurora	Colorado
University of Florida	Gainesville	Florida
Rare Disease Research	Atlanta	Georgia
Northwestern University	Chicago	Illinois
Indiana University School of Medicine	Indianapolis	Indiana
University of Iowa	Iowa City	Iowa
University of Kansas Medical Center	Kansas City	Kansas
Kennedy Krieger Institute	Baltimore	Maryland
University of Massachusetts Memorial Medical Center	Worcester	Massachusetts
University of Minnesota	Minneapolis	Minnesota
Washington University School of Medicine	St Louis	Missouri
University of Rochester Medical Center	Rochester	New York
Rare Disease Research, LLC NC	Hillsborough	North Carolina
University of Cincinnati Gardner Neuroscience Institute	Cincinnati	Ohio
Nationwide Children's Hospital	Columbus	Ohio
University of Pennsylvania	Philadelphia	Pennsylvania
University of Pittsburgh	Pittsburgh	Pennsylvania
National Neuromuscular Research Institute	Austin	Texas
Neurology Rare Disease Center	Denton	Texas
Virginia Commonwealth University Health	Richmond	Virginia
St Vincent's Hospital Melbourne	Fitzroy	Australia
University Hospital Gent	Ghent	Belgium
Universitaire Ziekenhuizen Leuven	Leuven	Belgium
Centre Hospitalier Régional de la Citadelle	Liège	Belgium
Rigshospitalet	Copenhagen	Denmark
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone	Marseille	France
CHU de Nantes	Nantes	France
CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires	Nice	France
AP-HP Hopital Pitie-Salpetriere	Paris	France
Klinikum der Ludwig-Maximilians-Universitaet Muenchen	Munich	Germany
Hadassah University Hospital	Jerusalem	Israel
Schneider Children's Hospital of Israel	Petah Tikva	Israel
Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano	Milan	Italy
Azienda Ospedale - Università Padova	Padova	Italy

+ 11 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05291091 on ClinicalTrials.gov ↗ ← All trials in the UK