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

Last updated: April 25, 2025
Sponsor: Edgewise Therapeutics, Inc.
Overall Status: Active - Not Recruiting

Phase

2

Condition

Muscular Dystrophy

Treatment

EDG-5506 Dose 2

EDG-5506 5 mg

EDG-5506 12.5 mg

Clinical Study ID

NCT05291091
EDG-5506-201
  • Ages 12-50
  • Male

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 is fully enrolled; GRAND CANYON is currently enrolling.

Eligibility Criteria

Inclusion

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 andphenotype consistent with Becker muscular dystrophy, and history of being ambulatorybeyond 16 years of age without steroids; history of being ambulatory beyond 18 yearsof age with steroids.

  2. Able to complete the 100-meter timed test in < 200 seconds with or without use ofmobility aid devices.

  3. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5to 32, inclusive.

Exclusion

Key Exclusion Criteria:

  1. Medical history or clinically significant physical examination/laboratory resultthat, in the opinion of the investigator, would render the participant unsuitablefor the study. This includes contraindications to magnetic resonance imaging such asnon-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 islonger) of the screening visit in the present study.

Study Design

Total Participants: 175
Treatment Group(s): 12
Primary Treatment: EDG-5506 Dose 2
Phase: 2
Study Start date:
July 06, 2022
Estimated Completion Date:
August 31, 2026

Study 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.

Connect with a study center

  • St Vincent's Hospital Melbourne

    Fitzroy, VIC, 3065
    Australia

    Site Not Available

  • University Hospital Gent

    Gent, 9000
    Belgium

    Site Not Available

  • Universitaire Ziekenhuizen Leuven

    Leuven,
    Belgium

    Site Not Available

  • Centre Hospitalier Régional de la Citadelle

    Liège, 4000
    Belgium

    Site Not Available

  • Rigshospitalet

    Copenhagen,
    Denmark

    Site Not Available

  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

    Marseille, 13005
    France

    Site Not Available

  • CHU de Nantes

    Nantes Cedex, 44093
    France

    Site Not Available

  • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires

    Nice Cedex 1, 06001
    France

    Site Not Available

  • AP-HP Hopital Pitie-Salpetriere

    Paris, 75013
    France

    Site Not Available

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    Munich, 80336
    Germany

    Site Not Available

  • Hadassah University Hospital

    Jerusalem, 91240
    Israel

    Site Not Available

  • Schneider Children's Hospital of Israel

    Petah Tiqwa, 49202
    Israel

    Site Not Available

  • Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano

    Milan, 20122
    Italy

    Site Not Available

  • Azienda Ospedale - Università Padova

    Padova, 35128
    Italy

    Site Not Available

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    Rome, 00168
    Italy

    Site Not Available

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZA
    Netherlands

    Site Not Available

  • Optimal Clinical Trials

    Auckland, 1010
    New Zealand

    Site Not Available

  • Hospital Universitari de Bellvitge

    Barcelona, 08907
    Spain

    Site Not Available

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035
    Spain

    Site Not Available

  • Hospital Universitario Donostia

    San Sebastian, 20014
    Spain

    Site Not Available

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026
    Spain

    Site Not Available

  • University Hospital Gent

    Gent, Belgium 9000
    United Kingdom

    Active - Recruiting

  • St. George's University Hospitals NHS Foundation Trust

    London, SW17 0QT
    United Kingdom

    Site Not Available

  • University College London Hospital

    London, NW1 2PG
    United Kingdom

    Site Not Available

  • Newcastle Freeman Hospital

    Newcastle, NE7 7DN
    United Kingdom

    Site Not Available

  • Salford Royal Hospital

    Salford, M68HD
    United Kingdom

    Site Not Available

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202
    United States

    Site Not Available

  • UC Davis Medical Center

    Davis, California 95817
    United States

    Site Not Available

  • UC San Diego

    La Jolla, California 92037
    United States

    Site Not Available

  • UCLA Medical Center

    Los Angeles, California 90095
    United States

    Site Not Available

  • UC Irvine Medical Center

    Orange, California 92868
    United States

    Site Not Available

  • Stanford Neuroscience Health Center

    Palo Alto, California 94304
    United States

    Site Not Available

  • UC Davis Medical Center

    Sacramento, California 95817
    United States

    Site Not Available

  • UC Denver

    Aurora, Colorado 80045
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32611
    United States

    Site Not Available

  • Rare Disease Research

    Atlanta, Georgia 30329
    United States

    Site Not Available

  • Northwestern University

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Indiana University School of Medicine

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • University of Iowa

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • University of Kansas Medical Center

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • Kennedy Krieger Institute

    Baltimore, Maryland 21205
    United States

    Site Not Available

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts 01605
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • Washington University School of Medicine

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • University of Rochester Medical Center

    Rochester, New York 14642
    United States

    Site Not Available

  • Rare Disease Research, LLC NC

    Hillsborough, North Carolina 27278
    United States

    Site Not Available

  • University of Cincinnati Gardner Neuroscience Institute

    Cincinnati, Ohio 45219
    United States

    Site Not Available

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • University of Pittsburgh

    Pittsburgh, Pennsylvania 15224
    United States

    Site Not Available

  • Austin Neuromuscular Center

    Austin, Texas 78759
    United States

    Active - Recruiting

  • National Neuromuscular Research Institute

    Austin, Texas 78759
    United States

    Site Not Available

  • Neurology Rare Disease Center

    Denton, Texas 76208
    United States

    Site Not Available

  • Virginia Commonwealth University Health

    Richmond, Virginia 23298
    United States

    Site Not Available

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