ClinicalTrials.Veeva

Menu
The trial is taking place at:
A

Austin Neuromuscular Center | Austin, TX

Veeva-enabled site

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

Edgewise Therapeutics logo

Edgewise Therapeutics

Status and phase

Enrolling
Phase 2

Conditions

Becker Muscular Dystrophy

Treatments

Drug: Placebo
Drug: Sevasemten 10 mg
Drug: Sevasemten 5 mg
Drug: Sevasemten 12.5 mg

Study type

Interventional

Funder types

Industry
Other

Identifiers

NCT05291091
EDG-5506-201

Details and patient eligibility

About

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.

Full 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.

Enrollment

170 estimated patients

Sex

Male

Ages

12 to 50 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Quadruple Blind

170 participants in 5 patient groups

Adult Cohort 1
Experimental group
Description:
Drug: Sevasemten Drug: Placebo
Treatment:
Drug: Sevasemten 10 mg
Drug: Placebo
Adult Cohort 2
Experimental group
Description:
Drug: Sevasemten Drug: Placebo
Treatment:
Drug: Sevasemten 10 mg
Drug: Placebo
Adult Cohort 6
Experimental group
Description:
Drug: Sevasemten Drug: Placebo
Treatment:
Drug: Sevasemten 10 mg
Drug: Placebo
Adolescent Cohort 4
Experimental group
Description:
Drug: Sevasemten Drug: Placebo
Treatment:
Drug: Sevasemten 5 mg
Drug: Placebo
Adolescent Cohort 5
Experimental group
Description:
Drug: Sevasemten Drug: Placebo
Treatment:
Drug: Sevasemten 12.5 mg
Drug: Placebo

Trial contacts and locations

29

Loading...

Central trial contact

Edgewise Therapeutics

Data sourced from clinicaltrials.gov

Clinical trials

Find clinical trialsTrials by location
© Copyright 2024 Veeva Systems