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

A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Becker Muscular Dystrophy
• Intervention / Treatment: Drug: Placebo; Drug: Sevasemten 10 mg; Drug: Sevasemten 5 mg; Drug: Sevasemten 12.5 mg

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 Type: Interventional
• Enrollment (Actual): 244
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • Fitzroy, Australia, VIC, 3065
    • St Vincent's Hospital Melbourne
• Belgium
  • Leuven, Belgium
    • Universitaire Ziekenhuizen Leuven
  • Liège, Belgium, 4000
    • Centre Hospitalier Regional de la Citadelle
  • Belgium
    • Ghent, Belgium, Belgium, 9000
      • University Hospital Gent
• Denmark
  • Copenhagen, Denmark
    • Rigshospitalet
• France
  • Marseille, France, 13005
    • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
  • Nantes, France, 44093
    • Chu de Nantes
  • Nice, France, 06001
    • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires
  • Paris, France, 75013
    • AP-HP Hopital Pitie-Salpetriere
• Germany
  • Munich, Germany, 80336
    • Klinikum der Ludwig-Maximilians-Universitaet Muenchen
• Israel
  • Jerusalem, Israel, 91240
    • Hadassah University Hospital
  • Petah Tikva, Israel, 49202
    • Schneider Children's Hospital of Israel
• Italy
  • Milan, Italy, 20122
    • Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano
  • Padova, Italy, 35128
    • Azienda Ospedale - Università Padova
  • Rome, Italy, 00168
    • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
• Netherlands
  • Leiden, Netherlands, 2333 ZA
    • Leids Universitair Medisch Centrum
• New Zealand
  • Auckland, New Zealand, 1010
    • Optimal Clinical Trials
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitario Vall d'Hebron
  • Barcelona, Spain, 08907
    • Hospital Universitari de Bellvitge
  • Donostia / San Sebastian, Spain, 20014
    • Hospital Universitario Donostia
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe
• United Kingdom
  • London, United Kingdom, SW17 0QT
    • St. George's University Hospitals NHS Foundation Trust
  • London, United Kingdom, NW1 2PG
    • University College London Hospital
  • Newcastle, United Kingdom, NE7 7DN
    • Newcastle Freeman Hospital
  • Salford, United Kingdom, M68HD
    • Salford Royal Hospital
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • La Jolla, California, United States, 92037
      • UC San Diego
    • Los Angeles, California, United States, 90095
      • UCLA Medical Center
    • Orange, California, United States, 92868
      • UC Irvine Medical Center
    • Palo Alto, California, United States, 94304
      • Stanford Neuroscience Health Center
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center
  • Colorado
    • Aurora, Colorado, United States, 80045
      • UC Denver
  • Florida
    • Gainesville, Florida, United States, 32611
      • University of Florida
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Rare Disease Research
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University School of Medicine
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Kennedy Krieger Institute
  • Massachusetts
    • Worcester, Massachusetts, United States, 01605
      • University of Massachusetts Memorial Medical Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New York
    • Rochester, New York, United States, 14642
      • University of Rochester Medical Center
  • North Carolina
    • Hillsborough, North Carolina, United States, 27278
      • Rare Disease Research, LLC NC
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Gardner Neuroscience Institute
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • University of Pittsburgh
  • Texas
    • Austin, Texas, United States, 78759
      • National Neuromuscular Research Institute
    • Denton, Texas, United States, 76208
      • Neurology Rare Disease Center
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 50 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Adult Cohort 1; Drug: Sevasemten Drug: Placebo	Drug: Placebo; Placebo is administered orally once per day; Drug: Sevasemten 10 mg; Sevasemten is administered orally once per day
Experimental: Adult Cohort 2; Drug: Sevasemten Drug: Placebo	Drug: Placebo; Placebo is administered orally once per day; Drug: Sevasemten 10 mg; Sevasemten is administered orally once per day
Experimental: Adult Cohort 6; Drug: Sevasemten Drug: Placebo	Drug: Placebo; Placebo is administered orally once per day; Drug: Sevasemten 10 mg; Sevasemten is administered orally once per day
Experimental: Adolescent Cohort 4; Drug: Sevasemten Drug: Placebo	Drug: Placebo; Placebo is administered orally once per day; Drug: Sevasemten 5 mg; Sevasemten is administered orally once per day
Experimental: Adolescent Cohort 5; Drug: Sevasemten Drug: Placebo	Drug: Placebo; Placebo is administered orally once per day; Drug: Sevasemten 12.5 mg; Sevasemten is administered orally once per day

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Edgewise Therapeutics, Inc.
• Collaborators: Medpace, Inc.; SYSNAV; ImagingNMD
• Investigators: Study Chair: Joanne Donovan, MD, PhD, Edgewise Therapeutics, Inc.; Study Chair: Roxana D. Dreghici, Edgewise Therapeutics, Inc.

Publications and helpful links

Helpful Links

• Sponsor Website

Study record dates

Study Major Dates

• Study Start (Actual): November 10, 2022
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2026

Study Registration Dates

• First Submitted: March 14, 2022
• First Submitted That Met QC Criteria: March 14, 2022
• First Posted (Actual): March 22, 2022

Study Record Updates

• Last Update Posted (Actual): May 20, 2026
• Last Update Submitted That Met QC Criteria: May 18, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Becker Muscular Dystrophy
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne; Substandard Drugs; Pharmaceutical Preparations; Counterfeit Drugs
• Other Study ID Numbers: EDG-5506-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No