Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2019-08-01
Target enrollment:
Participant gender:
Summary
The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy,
pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in
pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from ≥4 to <8
years of age will be enrolled.
Edasalonexent is an orally administered small molecule targeted to inhibit activated NF-κB, a
molecule that is activated from infancy in DMD and which is central to causing muscle damage
and preventing muscle regeneration. Data on magnetic resonance imaging of the lower and upper
leg muscles, physical function (including timed function tests) and muscle strength will be
studied.