A two part study to assess safety and tolerability, pharmacokinetics, effects on histology and on different clinical parameters of Givinostat in ambulant children with Duchenne Muscular Dystrophy

Paolo Bettica, MD PhD
Italfarmaco SpA

3rd July 2012

Italfarmaco proposes to test the histone deacetylase inhibitor (HDAC), Givinostat, in Duchenne Muscular Dystrophy (DMD). This is an interesting proposal with a novel drug and approach to DMD. Givinostat has been evaluated in inflammatory and oncological diseases in humans including children dosed for up to 6 months with a good safety profile. Published pre-clinical data suggests a benefit of other HDAC inhibitors in the mdx mouse model of DMD. Investigators found that Givinostat increases the size of myofibers, decreases inflammation and prevents formation of fibrosis in the mdx mouse model of muscular dystrophy. Additional pre-clinical data on the mdx mouse model and potentially the canine model including biomarker identification and cardiac effects were encouraged. The committee recommended adding a placebo control group to both the safety and POC/efficacy studies of Givinostat, consideration of alternative or additional proof of concept outcome measures other than muscle histology, and consistency in the duration of dosing within treatment groups.

12 Apr 2017