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enrolling by invitation

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)

key information

study id #: NCT03917719

condition: Duchenne Muscular Dystrophy

status: enrolling by invitation

purpose:

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 4-10 years of age (up to 11th 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.

intervention: Edasalonexent

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

results: https://clinicaltrials.gov/ct2/show/results/NCT03917719

last updated: December 11, 2019