P129 Givinostat in DMD: results of the Epidys study with particular attention to MR measures of muscle fat fraction

A randomized, double blinded, placebo controlled, multicenter Phase 3 clinical trial (Epidys; NCT02851797) examined the safety and efficacy of givinostat, an orally active histone deacetylase inhibitor in development, in ambulant boys with Duchenne muscular dystrophy (DMD). A total of 179 DMD boys aged ≥6 years at baseline were enrolled and treated for 18 months. The primary efficacy assessment was the time to climb 4 standard stairs (4SC). The primary endpoint was complemented by six key secondary efficacy endpoints: NSAA, time to rise from floor, 6MWT, knee extension and elbow flexion muscle s trength, and a biomarker consisting of fat fraction of the vastus lateralis (VL) measured by magnetic resonance spectroscopy (MRS) which has proven to be sensitive to disease progression in DMD and predictive of loss of ambulation.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research