An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Status:
Terminated
Trial end date:
2020-10-26
Target enrollment:
Participant gender:
Summary
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate
the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in
pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed
CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th
birthday) will be enrolled.
Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a key
link between loss of dystrophin and disease pathology and plays a fundamental role in the
initiation and progression of skeletal and cardiac muscle disease in DMD.