Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy

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)

Status Completed

Clinical Trial Summary

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.

Clinical Trial Description

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 ;

Related Conditions & MeSH terms

• Becker Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

• NCT number: NCT03238235
• Study type: Interventional
• Source: Italfarmaco
• Status: Completed
• Phase: Phase 2
• Start date: December 12, 2017
• Completion date: March 19, 2021