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

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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.

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Study details:

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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

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.

Able to complete the 100-meter timed test in < 200 seconds with or without use of mobility aid devices.

Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive.

Exclusion criteria

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.

Cardiac echocardiogram ejection fraction < 40%

Forced vital capacity predicted <60% or using daytime ventilatory support

Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.

Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.

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Eligibility

Age eligible for study : 12 and older

Healthy volunteers accepted : No

Gender eligible for study: Male

Things to know

Study dates

Study start: 2022-07-06

Primary completion: 2026-08-01

Study completion finish: 2026-08-01

Study type

TREATMENT

Phase

PHASE2

Trial ID

NCT05291091

Intervention or treatment

DRUG: Sevasemten 10 mg

DRUG: Sevasemten 5 mg

DRUG: Sevasemten 12.5 mg

DRUG: Placebo

Conditions

• Becker Muscular Dystrophy

Image related to Becker Muscular Dystrophy

Condition: Becker Muscular Dystrophy
DRUG: Sevasemten 10 mg and other drugs
Fitzroy, Not Specified, Australia
Sponsor: Edgewise Therapeutics, Inc.

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Find a site

Closest Location:

St Vincent's Hospital Melbourne

Research sites nearby

Select from list below to view details:

St Vincent's Hospital Melbourne
Fitzroy, Not Specified, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Adult Cohort 1 Drug: Sevasemten Drug: Placebo	DRUG: Sevasemten 10 mg Sevasemten is administered orally once per day
EXPERIMENTAL: Adult Cohort 2 Drug: Sevasemten Drug: Placebo	DRUG: Sevasemten 10 mg Sevasemten is administered orally once per day
EXPERIMENTAL: Adult Cohort 6 Drug: Sevasemten Drug: Placebo	DRUG: Sevasemten 10 mg Sevasemten is administered orally once per day
EXPERIMENTAL: Adolescent Cohort 4 Drug: Sevasemten Drug: Placebo	DRUG: Sevasemten 5 mg Sevasemten is administered orally once per day
EXPERIMENTAL: Adolescent Cohort 5 Drug: Sevasemten Drug: Placebo	DRUG: Sevasemten 12.5 mg Sevasemten is administered orally once per day

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
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)
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)
Change from Baseline in serum Creatine Kinase	Adult participants	12 Months (CANYON Cohorts 1, 2)
Change from Baseline in the North Star Ambulatory Assessment scale	Adult participants	18 months (GRAND CANYON Cohort 6)

Secondary outcome

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

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References

Clinical Trials Gov: Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)