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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05291091
Recruitment Status : Recruiting
First Posted : March 22, 2022
Last Update Posted : March 14, 2023
Sponsor:
Collaborator:
Medpace, Inc.
Information provided by (Responsible Party):
Edgewise Therapeutics, Inc.

Study Description
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Brief Summary:
The CANYON study is a multicenter, randomized, Phase 2, double-blind, placebo-controlled study to evaluate the effect of EDG-5506 on the safety, pharmacokinetics, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy.

Condition or disease Intervention/treatment Phase
Becker Muscular Dystrophy Drug: EDG-5506 Dose 1 Drug: EDG-5506 Dose 2 Drug: EDG-5506 Dose 3 Drug: EDG-5506 Dose 4 Drug: EDG-5506 Dose 5 Drug: Placebo Phase 2

Detailed Description:

This study is a double-blind, randomized, placebo-controlled design to investigate the effect of EDG-5506 on the safety, pharmacokinetics, biomarkers and functional measures. Approximately 48 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 48 adult participants will randomize to Cohort 1 (Dose 1) or Cohort 2 (Dose 2) in a 1:1 ratio then each cohort will further randomize to EDG-5506 or placebo in a 3:1 ratio. Cohort 3 (Dose 3) will randomize participants in a 3:1 ratio to either EDG-5506 or placebo and will enroll after Cohort 2.

Approximately 9 adolescent participants will enroll in Cohort 4 (Dose 4) and randomize in a 2:1 ratio to EDG-5506 or placebo. Cohort 5 (Dose 5) will randomize an additional 9 participants in a 2:1 ratio to either EDG-5506 or placebo after Cohort 4.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 66 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : July 6, 2022
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Adult Cohort 1
Drug: EDG-5506 Drug: Placebo
 Drug: EDG-5506 Dose 1
EDG-5506 is administered orally once per day

Drug: Placebo
Placebo is administered orally once per day
Experimental: Adult Cohort 2
Drug: EDG-5506 Drug: Placebo
 Drug: EDG-5506 Dose 2
EDG-5506 is administered orally once per day

Drug: Placebo
Placebo is administered orally once per day
Experimental: Adult Cohort 3
Drug: EDG-5506 Drug: Placebo
 Drug: EDG-5506 Dose 3
EDG-5506 is administered orally once per day

Drug: Placebo
Placebo is administered orally once per day
Experimental: Adolescent Cohort 4
Drug: EDG-5506 Drug: Placebo
 Drug: EDG-5506 Dose 4
EDG-5506 is administered orally once per day

Drug: Placebo
Placebo is administered orally once per day
Experimental: Adolescent Cohort 5
Drug: EDG-5506 Drug: Placebo
 Drug: EDG-5506 Dose 5
EDG-5506 is administered orally once per day

Drug: Placebo
Placebo is administered orally once per day


Outcome Measures
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Primary Outcome Measures :
  1. Number of adverse events in those treated with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants

  2. Severity of adverse events in those treated with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants

  3. Change from Baseline in serum Creatine Kinase [ Time Frame: 12 Months ]
    Adult participants


Secondary Outcome Measures :
  1. Change from Baseline in fat fraction of upper leg muscles assessed by Magnetic Resonance Imaging [ Time Frame: 12 Months ]
    Adult participants

  2. Change from Baseline in the North Star Ambulatory Assessment scale [ Time Frame: 12 Months ]
    Adult participants

  3. Change from Baseline in the 10-meter walk/run test [ Time Frame: 12 Months ]
    Adult participants

  4. Change from Baseline in 100-meter timed test [ Time Frame: 12 Months ]
    Adult participants

  5. Change from Baseline in 4-stair climb [ Time Frame: 12 Months ]
    Adult participants

  6. Pharmacokinetics as measured by steady state plasma concentration [ Time Frame: 12 Months ]
    All participants


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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, OR Adolescents (12 to 17 years, inclusive) with genetic confirmation of an in-frame dystrophin mutation and a phenotype consistent with Becker muscular dystrophy as determined by the Investigator
  2. Able to complete the 100-meter timed test in < 150 seconds.
  3. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 10 to 32, inclusive for adults or a score of greater than 10 for adolescents.

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 >5 days in the previous 6 months at a dose of >5 mg equivalent per day. Lower oral doses or inhaled/intranasal steroids are permitted.
  5. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05291091


Contacts
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Contact: Edgewise Therapeutics 720-262-7002 studies@edgewisetx.com

Locations
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United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
United States, California
UC Davis Medical Center Recruiting
Sacramento, California, United States, 95817
United States, Colorado
UC Denver Recruiting
Aurora, Colorado, United States, 80045
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, Maryland
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
United States, Massachusetts
University of Massachusetts Memorial Medical Center Recruiting
Worcester, Massachusetts, United States, 01605
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
United States, Ohio
University of Cincinnati Gardner Neuroscience Institute Recruiting
Cincinnati, Ohio, United States, 45219
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, Virginia
Virginia Commonwealth University Health Recruiting
Richmond, Virginia, United States, 23298
Netherlands
Leids Universitair Medisch Centrum Recruiting
Leiden, Netherlands, 2333 ZA
United Kingdom
University College London Hospital Recruiting
London, United Kingdom, NW1 2PG
Newcastle Freeman Hospital Recruiting
Newcastle, United Kingdom, NE7 7DN
Sponsors and Collaborators
Edgewise Therapeutics, Inc.
Medpace, Inc.
Investigators
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Study Chair: Sam Collins, MBBS, PhD Edgewise Therapeutics, Inc.
More Information
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Additional Information:

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Responsible Party: Edgewise Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05291091    
Other Study ID Numbers: EDG-5506-201
First Posted: March 22, 2022    Key Record Dates
Last Update Posted: March 14, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Edgewise Therapeutics, Inc.:
Becker Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked