Open-Label Extension of EDG-5506 in Participants with Becker Muscular Dystrophy
Research type
Research Study
Full title
An Open-Label Extension Study to Assess the Long-term Effect of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy
IRAS ID
1008268
Contact name
Edgewise Clinical Studies N/A
Contact email
Sponsor organisation
Edgewise Therapeutics Inc
Research summary
EDG-5506 is an investigational drug for Becker muscular dystrophy (BMD), a genetic condition that causes progressive weakness in the muscles of the body due to partially working dystrophin, a protein important in muscle function. EDG-5506 is thought to work by protecting fast muscle fibers from muscle damage, thus modifying disease progression. This is an open-label (everyone receives EDG-5506) study for participants with BMD who participated in either the EDG-5506-002, EDG-5506-201, or EDG-5506-202 study.
The purpose of this study is to continue to learn about the safety and effect of EDG-5506 in adults and adolescents diagnosed with BMD.
This research study will assess how safe EDG-5506 is under long-term treatment. Research participants will be asked if they are experiencing potential side effects at each visit and at the phone calls in between visits.
This research study will assess the safety of EDG-5506 with tests such as blood tests, heart function tests and lung function tests to measure any changes in these parameters in individuals taking EDG-5506.
This trial is planned to be conducted at multiple sites in the USA, Europe, and Asia Pacific where adults and adolescents with BMD have been enrolled in one of the studies with EDG-5506 (EDG-5506-002, EDG-5506-201, or EDG-5506-202 study). The research study consists of an 18-month Treatment period and a Follow up period about 4 weeks later.
EDG-5506, at doses of 5, 10, or 12.5 mg, will be given orally in tablet form, once daily for 18 months.
This research study is designed to minimise risk by monitoring participants’ safety through blood tests and other safety assessments. BMD is a progressive disease and there is currently no treatment available to mitigate the natural progression of the disease. While the efficacy of EDG-5506 has not yet been demonstrated, the potential benefit may outweigh potential risks.REC name
West of Scotland REC 1
REC reference
23/WS/0151
Date of REC Opinion
6 Nov 2023
REC opinion
Further Information Favourable Opinion