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Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
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.
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.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
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
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 10 mg
|
EXPERIMENTAL: Adult Cohort 2
| DRUG: Sevasemten 10 mg
|
EXPERIMENTAL: Adult Cohort 6
| DRUG: Sevasemten 10 mg
|
EXPERIMENTAL: Adolescent Cohort 4
| DRUG: Sevasemten 5 mg
|
EXPERIMENTAL: Adolescent Cohort 5
| DRUG: Sevasemten 12.5 mg
|
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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