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

Becker Muscular Dystrophy Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05291091
• Other study ID #: EDG-5506-201
• Status: Recruiting
• Phase: Phase 2
• Start date: July 6, 2022
• Est. completion date: June 2026

Study information

• Verified date: June 2024
• Source: Edgewise Therapeutics, Inc.
• Contact: Edgewise Therapeutics
• Phone: 720-262-7002
• Email: studies[@]edgewisetx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 170
• Est. completion date: June 2026
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 50 Years

Eligibility

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.

Related Conditions & MeSH terms

• Becker Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• 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

Locations

Country	Name	City	State
Netherlands	Leids Universitair Medisch Centrum	Leiden
Spain	Hospital Universitari i Politecnic La Fe	Valencia
United Kingdom	University College London Hospital	London
United Kingdom	Newcastle Freeman Hospital	Newcastle
United Kingdom	Salford Royal Hospital	Salford
United States	Rare Disease Research	Atlanta	Georgia
United States	UC Denver	Aurora	Colorado
United States	National Neuromuscular Research Institute	Austin	Texas
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	Northwestern University	Chicago	Illinois
United States	University of Cincinnati Gardner Neuroscience Institute	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Neurology Rare Disease Center	Denton	Texas
United States	University of Florida	Gainesville	Florida
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	UC San Diego	La Jolla	California
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	UC Irvine Medical Center	Orange	California
United States	Stanford Neuroscience Health Center	Palo Alto	California
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Virginia Commonwealth University Health	Richmond	Virginia
United States	University of Rochester Medical Center	Rochester	New York
United States	UC Davis Medical Center	Sacramento	California
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Massachusetts Memorial Medical Center	Worcester	Massachusetts

Sponsors (4)

Lead Sponsor	Collaborator
Edgewise Therapeutics, Inc.	ImagingNMD, Medpace, Inc., SYSNAV

Countries where clinical trial is conducted

United States,  Netherlands,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	Change from Baseline in serum Creatine Kinase	Adult participants	12 Months (CANYON Cohorts 1, 2)
Primary	Change from Baseline in the North Star Ambulatory Assessment scale	Adult participants	18 months (GRAND CANYON Cohort 6)
Secondary	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)
Secondary	Change from Baseline in the North Star Ambulatory Assessment scale	Adult participants	12 Months (CANYON Cohorts 1, 2)
Secondary	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)
Secondary	Change from Baseline in the 10-meter walk/run test	Adult participants	12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Secondary	Change from Baseline in 100-meter timed test	Adult participants	12 Months (CANYON Cohorts 1, 2), 18 Months (GRAND CANYON Cohort 6)
Secondary	Change from Baseline in stride velocity (95th percentile)	Adult participants	18 Months (GRAND CANYON Cohort 6)
Secondary	Pharmacokinetics as measured by steady state plasma concentration	All participants	12 Months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)
Secondary	Change from Baseline in growth as assessed by height centile on World Health Organization growth charts	Adolescent participants	12 months (CANYON Cohorts 4, 5)
Secondary	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)

External Links

•   Sponsor Website