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Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

key information

study id #: NCT02439216

condition: Muscular Dystrophy, Duchenne

status: completed


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.

intervention: Edasalonexent, Placebo

mechanism of action: NF-KB inhibitor to prevent muscle damage and promote muscle regeneration

results: https://clinicaltrials.gov/ct2/show/results/NCT02439216

last updated: November 27, 2019