Clinical Trial Alert: Phase 2 Pivotal Study of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy

Researchers at Edgewise Therapeutics are seeking adults living with Becker muscular dystrophy to participate in a global Phase 2 pivotal study, GRAND CANYON, to evaluate the safety and efficacy of sevasemten (EDG-5506) in adults with Becker. Sevasemten is an investigational oral pill designed to prevent contraction-induced muscle damage that occurs with daily activity in Becker.

This trial is a multi-center, randomized, double-blind, placebo-controlled study. This means that participants will be randomly assigned to either sevasemten or a placebo. The total trial duration for each participant will be approximately 19 months, which includes up to 7 on-site clinic visits. An open label study will be available after completion of the 18-month period, where all participants would receive sevasemten.

Study drug will be administered as oral tablets given once per day. The effects of sevasemten will be evaluated using blood tests, safety assessments, and functional assessments over the course of the study.

To be eligible, individuals must meet the following inclusion criteria:

• Age 18-50 years, with documented dystrophin mutation and phenotype consistent with BMD, currently ambulatory
• 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

Individuals may not be eligible to participate if they are affected by another illness or receiving another treatment that might interfere with study procedures.

Please visit this link for a listing of inclusion and exclusion criteria.

Travel and other resources will be coordinated and provided for eligible participants.

To learn more about the study or inquire about participation, please visit the trial website here or email the study coordinator at studies@edgewisetx.com.