Status:
ACTIVE_NOT_RECRUITING
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
Lead Sponsor:
Edgewise Therapeutics, Inc.
Collaborating Sponsors:
Medpace, Inc.
ImagingNMD
Conditions:
Becker Muscular Dystrophy
Eligibility:
MALE
12-50 years
Phase:
PHASE2
Brief 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 adul...
Detailed 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 ...
Eligibility Criteria
Inclusion
- The CANYON Study including the adolescent cohorts are fully enrolled.
- GRAND CANYON eligibility is listed below.
- Key
- 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.
- Key
Exclusion
- 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.
Key Trial Info
Start Date :
November 10 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2026
Estimated Enrollment :
175 Patients enrolled
Trial Details
Trial ID
NCT05291091
Start Date
November 10 2022
End Date
September 1 2026
Last Update
December 1 2025
Active Locations (51)
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1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
2
UC San Diego
La Jolla, California, United States, 92037
3
UCLA Medical Center
Los Angeles, California, United States, 90095
4
UC Irvine Medical Center
Orange, California, United States, 92868