Becker Muscular Dystrophy Clinical Trial
Official title:
A Randomised, Double Blind, Placebo Controlled Study to Evaluate the Micro-macroscopic Effects on Muscles, the Safety and Tolerability, and the Efficacy of Givinostat in Patients With Becker Muscular Dystrophy (BMD)
This is a phase 2, randomised, double-blind, placebo controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy. Approximately 48 eligible patients will be randomized in a 2:1 ratio to be treated with givinostat or placebo for a period of 12 months.
Givinostat or placebo oral suspension (10 mg/mL) will be administered orally as 2 oral doses daily while the subject is in fed state, according to the subject's weight. Study drug should be permanently stopped if any of the following occur: - severe drug-related diarrhoea; - any drug-related Serious Adverse Event (SAE); - QTcorrected by Fridericia's formulas (QTcF) >500 msec; - platelets (PLT) count ≤50 x 1.000.000.000/L (10E9/L); - White blood cell (WBC) ≤ 2.0 x 10E9/L; - Hemoglobin (Hb) ≤ 8.0 g/dL. Study drug should be temporarily stopped if any of the following occur: - PLT count <75 x 10E9/L but >50 x 10E9/L; - WBC < 3.0 x 10E9/L but > 2.0 x 10E9/L; - Hb < 10.0 g/dL but > 8.0 g/dL; - moderate or severe diarrhoea. - tryglicerides >300 mg/dL In case the study drug was temporarily stopped, the study drug can be resumed at a level 20% smaller than the dose at which the Adverse Event leading to temporary stop occurred, once platelets and/or WBC and/or Hb and/or tryglicerides are normalized or when diarrhoea is mild ;
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