Clinical Trial Alert: Phase 2 Study of EDG-5506 in Adults or Adolescents with BMD

Researchers at Edgewise Therapeutics are seeking adults and adolescents living with Becker muscular dystrophy (BMD) to participate in a Phase 2 clinical trial (CANYON) to evaluate the safety and efficacy of EDG-5506 in the treatment of BMD. EDG-5506 was designed to protect susceptible muscle fibers from the damage that occurs as a result of everyday activities in people living with BMD.

This trial is a randomized, double-blind, placebo-controlled study. This means that participants will be randomly assigned to either EDG-5506 or a placebo. The total trial duration for each participant will be approximately 14 months, which includes up to 10 on-site clinic visits and 5 telephone visits. An open label study may be available after completion of the first 12-month period. In an open label study, all participants would receive EDG-5506.

Study drug will be administered as oral tablets given once per day. The effects of EDG-5506 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
• Adolescents (12-17 years), genetic confirmation of in-frame dystrophin mutation and phenotype consistent with BMD
• Able to complete the 100-meter timed test in <150 seconds.
• Able to perform the North Star Ambulatory Assessment scale with a score of 10-32 for adults or >10 for adolescents

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.