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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.

Tracking Information
First Submitted Date  ICMJE March 14, 2022
First Posted Date  ICMJE March 22, 2022
Last Update Posted Date March 14, 2023
Actual Study Start Date  ICMJE July 6, 2022
Estimated Primary Completion Date December 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 21, 2022)
  • Number of adverse events in those treated with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants
  • Severity of adverse events in those treated with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants
  • Change from Baseline in serum Creatine Kinase [ Time Frame: 12 Months ]
    Adult participants
Original Primary Outcome Measures  ICMJE
 (submitted: March 14, 2022)
  • Number of AEs in those treated with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants
  • Severity of AEs in those treated with EDG-5506 or placebo [ Time Frame: 12 months ]
    All participants
  • Change from Baseline in serum Creatine Kinase (CK) [ Time Frame: 12 Months ]
    Adult participants
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 21, 2022)
  • Change from Baseline in fat fraction of upper leg muscles assessed by Magnetic Resonance Imaging [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in the North Star Ambulatory Assessment scale [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in the 10-meter walk/run test [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in 100-meter timed test [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in 4-stair climb [ Time Frame: 12 Months ]
    Adult participants
  • Pharmacokinetics as measured by steady state plasma concentration [ Time Frame: 12 Months ]
    All participants
Original Secondary Outcome Measures  ICMJE
 (submitted: March 14, 2022)
  • Change from Baseline in fat fraction of upper leg muscles assessed by Magnetic Resonance Imaging [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in North Star Ambulatory Assessment (NSAA) [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in the 10-meter walk/run test [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in 100-meter timed test [ Time Frame: 12 Months ]
    Adult participants
  • Change from Baseline in 4-stair climb [ Time Frame: 12 Months ]
    Adult participants
  • Pharmacokinetics as measured by steady state plasma concentration [ Time Frame: 12 Months ]
    All participants
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (CANYON)
Official Title  ICMJE 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
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Becker Muscular Dystrophy
Intervention  ICMJE
  • Drug: EDG-5506 Dose 1
    EDG-5506 is administered orally once per day
  • Drug: EDG-5506 Dose 2
    EDG-5506 is administered orally once per day
  • Drug: EDG-5506 Dose 3
    EDG-5506 is administered orally once per day
  • Drug: EDG-5506 Dose 4
    EDG-5506 is administered orally once per day
  • Drug: EDG-5506 Dose 5
    EDG-5506 is administered orally once per day
  • Drug: Placebo
    Placebo is administered orally once per day
Study Arms  ICMJE
  • Experimental: Adult Cohort 1
    Drug: EDG-5506 Drug: Placebo
    Interventions:
    • Drug: EDG-5506 Dose 1
    • Drug: Placebo
  • Experimental: Adult Cohort 2
    Drug: EDG-5506 Drug: Placebo
    Interventions:
    • Drug: EDG-5506 Dose 2
    • Drug: Placebo
  • Experimental: Adult Cohort 3
    Drug: EDG-5506 Drug: Placebo
    Interventions:
    • Drug: EDG-5506 Dose 3
    • Drug: Placebo
  • Experimental: Adolescent Cohort 4
    Drug: EDG-5506 Drug: Placebo
    Interventions:
    • Drug: EDG-5506 Dose 4
    • Drug: Placebo
  • Experimental: Adolescent Cohort 5
    Drug: EDG-5506 Drug: Placebo
    Interventions:
    • Drug: EDG-5506 Dose 5
    • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 11, 2022)		 66
Original Estimated Enrollment  ICMJE
 (submitted: March 14, 2022)		 54
Estimated Study Completion Date  ICMJE December 2023
Estimated Primary Completion Date December 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 12 Years to 50 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Edgewise Therapeutics 720-262-7002 studies@edgewisetx.com
Listed Location Countries  ICMJE Netherlands,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05291091
Other Study ID Numbers  ICMJE EDG-5506-201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Edgewise Therapeutics, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Edgewise Therapeutics, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Medpace, Inc.
Investigators  ICMJE
Study Chair: Sam Collins, MBBS, PhD Edgewise Therapeutics, Inc.
PRS Account Edgewise Therapeutics, Inc.
Verification Date March 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP