Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
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.
• Are an ambulant male aged ≥6 years at randomisation with DMD characteristic clinical symptoms or signs (e.g., proximal muscle weakness, Gowers' maneuver, elevated serum creatinine kinase level) already present at screening;
• Have DMD diagnosis confirmed by genetic testing;
• Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to local regulations);
• Are able to complete 2 Four Stairs Climb test (4SC) screening assessments; the results of these tests must be within ±1 second of each other;
• Have the mean of 2 screening 4SC assessments ≤8 seconds;
• Have time to rise from floor between ≥3 and \<10 seconds at screening
• Have manual muscle testing (MMT) of quadriceps at screening Grade ≥- 3;
• Have used systemic corticosteroids for a minimum of 6 months immediately prior to the start of study treatment, with no significant change in corticosteroids type or dosage or dosing regimen (excluding changes related to body weight change) for a minimum of 6 months immediately prior to start of study treatment and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
• Subjects must be willing to use adequate contraception.