Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
NCT ID: NCT05291091
Last Updated: 2026-02-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
244 participants
INTERVENTIONAL
2022-11-10
2026-09-30
Brief Summary
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CANYON and GRAND CANYON are fully enrolled.
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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Adult Cohort 1
Drug: Sevasemten Drug: Placebo
Sevasemten 10 mg
Sevasemten is administered orally once per day
Placebo
Placebo is administered orally once per day
Adult Cohort 2
Drug: Sevasemten Drug: Placebo
Sevasemten 10 mg
Sevasemten is administered orally once per day
Placebo
Placebo is administered orally once per day
Adult Cohort 6
Drug: Sevasemten Drug: Placebo
Sevasemten 10 mg
Sevasemten is administered orally once per day
Placebo
Placebo is administered orally once per day
Adolescent Cohort 4
Drug: Sevasemten Drug: Placebo
Sevasemten 5 mg
Sevasemten is administered orally once per day
Placebo
Placebo is administered orally once per day
Adolescent Cohort 5
Drug: Sevasemten Drug: Placebo
Sevasemten 12.5 mg
Sevasemten is administered orally once per day
Placebo
Placebo is administered orally once per day
Interventions
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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
Eligibility Criteria
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Inclusion Criteria
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.
Exclusion Criteria
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.
12 Years
50 Years
MALE
No
Sponsors
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Medpace, Inc.
INDUSTRY
ImagingNMD
UNKNOWN
SYSNAV
INDUSTRY
Edgewise Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Joanne Donovan, MD, PhD
Role: STUDY_CHAIR
Edgewise Therapeutics, Inc.
Roxana D. Dreghici
Role: STUDY_CHAIR
Edgewise Therapeutics, Inc.
Locations
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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
Indiana University School of Medicine
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
University of Rochester Medical Center
Rochester, New York, United States
Rare Disease Research, LLC NC
Hillsborough, North Carolina, United States
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
National Neuromuscular Research Institute
Austin, Texas, United States
Neurology Rare Disease Center
Denton, Texas, United States
Virginia Commonwealth University Health
Richmond, Virginia, United States
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
Padua, , Italy
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, , Italy
Leids Universitair Medisch Centrum
Leiden, , Netherlands
Optimal Clinical Trials
Auckland, , New Zealand
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitari de Bellvitge
Barcelona, , Spain
Hospital Universitario Donostia
Donostia / San Sebastian, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
University College London Hospital
London, , United Kingdom
St. George's University Hospitals NHS Foundation Trust
London, , United Kingdom
Newcastle Freeman Hospital
Newcastle, , United Kingdom
Salford Royal Hospital
Salford, , United Kingdom
Countries
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Related Links
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Sponsor Website
Other Identifiers
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EDG-5506-201
Identifier Type: -
Identifier Source: org_study_id
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