Study to Test the Safety and Effectiveness of Givinostat in Non-Walking Boys Aged 9 to 17 with Duchenne Muscular Dystrophy

What this Trial Is About

Researchers are evaluating the efficacy, safety, and tolerability of givinostat in non-ambulant male children and adolescents aged 9 to under 18 years with Duchenne Muscular Dystrophy DMD. This phase 3, randomized, double-blind, placebo-controlled, multicenter study aims to show whether givinostat can reduce muscle decline in this population, building on previous studies in ambulant patients. The study involves 138 participants randomized 21 to receive either givinostat or placebo alongside standard corticosteroid care. Participants will receive givinostat or placebo twice daily with food, following a flexible dose regimen based on their weight, for 18 months. The study includes a screening period of about 4 weeks to confirm eligibility, a baseline visit for randomization, an 18-month treatment phase, and an end-of-study visit at week 72. After this, participants can join a long-term safety extension study where they will receive givinostat, or attend a follow-up visit 4 weeks later if they opt out. During the trial, participants will be closely monitored through various assessments including the Performance of the Upper Limb PUL 2.0 test, lung function tests like Peak Expiratory Flow and Forced Vital Capacity, heart evaluations with electrocardiogram and echocardiogram, and safety labs. Researchers will track adverse events, muscle function, respiratory health, and cardiac status over the 18 months of treatment. Total time in the study can be up to 83 weeks, including screening, treatment, and follow-up.

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy