At a glance
ClinicalIndex Comparison RecordStandardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
In Brief
A Phase 3 clinical trial evaluating givinostat and placebo for Duchenne Muscular Dystrophy. Completed, enrolled 179 participants across 42 sites in 11 countries.
Detailed Summary
Primary Objective The primary objective of the study was to establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects. Secondary Objectives The secondary objectives of this study were: * To assess the safety and tolerability of givinostat versus placebo administered chronically in DMD subjects * To evaluate the PK profile of givinostat administered chronically in DMD subjects * To evaluate the impact on quality of life (QoL) and activities of daily living of givinostat versus placebo administered chronically.
Study Details
Timeline
Interventions
The oral suspension of givinostat (10 mg/mL) was to be dosed in fed condition as described below: Givinostat or placebo starting dose * \> or =10 and \< 12.5 kg of weight: 13.3 mg bid = 1.3 ml oral suspension bid * \> or =12.5 and \< 20 kg: 16.7 mg bid =1.7 ml oral suspension bid * \> or = 20 and \< 25 kg: 20 mg bid = 2.0 ml oral suspension bid * \> or = 25 and \< 30 kg: 23.3 mg bid = 2.3 ml oral suspension bid * \> or = 30 and \< 40 kg: 26.7 mg bid = 2.7 ml oral suspension bid * \> or = 40 and \< 50 kg: 33.3 mg bid = 3.3 ml oral suspension bid * \> or = 50 and \< 60 kg: 36.7 mg bid = 3.7 ml oral suspension bid * \> or = 60 and \< 70 kg: 40 mg bid = 4 ml oral suspension bid * \> or = 70 kg: 46.7 mg bid = 4.7 ml oral suspension bid
The oral suspension of placebo, manufactured to mimic givinostat, was to be dosed in fed condition as described for givinostat.