The study (NCT04585464) is now enrolling up to 152 healthy volunteers and adults with BMD, ages 19 to 55, at its single site in San Antonio, Texas. More information on this location and study contacts can be found here.
EDG-5506 is designed to halt the deterioration of muscle fibers, the hallmark of muscular dystrophy. By limiting the inflammation and scarring that accompanies fiber breakdown, EDG-5506 could improve patients’ physical function and possibly benefit broad populations of muscular dystrophy patients, the company reported.
“Initiation of our Phase 1 study of EDG-5506 is an important milestone for our company,” Kevin Koch, PhD, president and CEO of Edgewise, said in a press release. “I’m excited about the potential of EDG-5506 for patients affected by BMD as well as Duchenne muscular dystrophy (DMD), who are currently underserved with limited treatment options.”
To be eligible for the study, patients must have a confirmed genetic diagnosis of BMD and be able to walk.
Investigators will assess EDG-5506’s safety, tolerability and pharmacokinetics — the movement of a compound into, through, and out of the body — in healthy volunteers prior to recruiting Becker patients. Healthy adults will be randomized to test both single ascending and multiple ascending doses of the treatment, or to a placebo, given once a day. Follow-up exams include the taking of blood and urine samples.
The effects of food or its absence (fasting) on EDG-5506 will also be assessed in a volunteer group on single ascending doses.
The study’s primary goal will be to assess the safety and tolerability of EDG-5506, measured by monitoring for treatment-related adverse events for up to 30 days, as well as abnormalities in test results or examinations.
Secondary goals include assessments of EDG-5506 levels in the blood and urine.
“We look forward to the results of our Phase 1 trial, which will help us select the optimal dose of EDG-5506 for pivotal clinical studies in these patient populations,” Koch said.
This Phase 1 study is expected to conclude in October 2021.
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