Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy
Research type
Research Study
Full title
A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy.
IRAS ID
1005137
Contact name
Edgewise Clinical Studies N/A
Contact email
Sponsor organisation
Edgewise Therapeutics
Eudract number
2021-006819-28
Clinicaltrials.gov Identifier
Research summary
EDG-5506-201 is a Phase 2 clinical trial study funded by Edgewise Therapeutics. It is a multicentre, randomised, double-blind, placebo-controlled study evaluating the safety and efficacy of investigational study drug, EDG-5506, in adults and adolescents with Becker Muscular Dystrophy (BMD). EDG-5506 binds to the muscle protein myosin and by doing so may limit muscle breakdown and disease progression in BMD.
This study will enrol approximately 48 male adults and 18 male adolescents. A 2-step randomisation process is used to assign participants to active or placebo. Adult participants may receive 10 mg, 15 mg or 20 mg. Adolescent participates may receive 5 mg or 12.5 mg. Depending on the dose assignment, participants may start a lower dose and increase after 4 weeks. Study drug will be taken by all participants, orally, daily for 52 weeks. Neither the participants nor the study doctors will be informed which group the participants are assigned to.
For each participant, the study will run for approximately 14 months, composed of a 4-week screening period, a 52-week treatment period and a follow-up period taking place approximately 4 weeks after the end of the treatment period. During the study, each participant will attend the study site at least 10 times for safety and efficacy study assessments.
The primary study objectives are to assess the safety and tolerability of EDG-5506, determined by the frequency and severity of study drug/placebo side effects. Moreover, creatine kinase (CK) levels in the blood will be monitored as an additional primary study objective to assess the effects of EDG-5506. Creatine kinase is a protein that is released from the muscles to the blood stream when muscle fibres are damaged.REC name
South Central - Oxford A Research Ethics Committee
REC reference
22/SC/0182
Date of REC Opinion
7 Oct 2022
REC opinion
Further Information Favourable Opinion