A phase II, double-blind, placebo-controlled clinical trial evaluated the efficacy and safety of givinostat in 51 adults with Becker muscular dystrophy (BMD). According to the results after one year of treatment, published in January 2023 :
- givinostat had no effect on total muscle fibrosis, the primary endpoint of this study.
- magnetic resonance imaging (MRI) showed stabilisation of the fat fraction in the whole thigh and quadriceps under givinostat.
- adverse events, mainly mild to moderate, were reported by 52.9% of placebo patients compared to 88.2% of givinostat participants, 11 of whom had to discontinue the trial (vs. one person in the placebo group).
