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

Italfarmaco · 2018
ClinicalTrials.gov · Datasets · License: Public Domain · 2018
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Objectives: Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months. Secondary Objectives: * To establish the macroscopic muscle effects of Givinostat versus placebo administered over 12 months assessed by Magnetic Resonance Imaging (MRI)/Magnetic Resonance Spectroscopy (MRS). * To determine the other histological effects of Givinostat versus placebo administered over 12 months. * To establish the efficacy of Givinostat versus placebo administered chronically over 12 months in slowing disease progression. * To assess the safety and tolerability of Givinostat versus placebo administered chronically. * To evaluate the pharmacokinetic (PK) profile of Givinostat administered chronically in the target population. * To evaluate the impact of Givinostat versus placebo administered chronically on quality of life and activities of daily living.
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