Long-term Study on Safety and Tolerability of Givinostat Treatment in Males with Duchenne Muscular Dystrophy Previously Treated with Givinostat

What this Trial Is About

Researchers are evaluating the long-term safety, tolerability, and effectiveness of Givinostat in patients with Duchenne Muscular Dystrophy DMD who have already participated in previous Givinostat studies. This open-label trial focuses on patients who have been previously treated with Givinostat, aiming to monitor ongoing effects and any adverse events over an extended period. Participants will take an oral suspension of Givinostat 10 mgmL twice daily while in a fed state. The starting dose will match what each patient received at the end of their prior Givinostat study and may be adjusted based on their weight and safety monitoring, especially platelet counts. Dose reductions or treatment interruptions may occur if certain safety concerns arise, such as severe diarrhea, serious adverse events, or specific blood count thresholds. The study includes weekly platelet monitoring during the first month and biweekly during the second month, with adjustments as needed. Throughout the study, participants will be closely monitored with regular evaluations of safety and tolerability, including blood tests to check platelet counts, white blood cells, hemoglobin, and heart rhythm QTcF intervals. Researchers will track the incidence of treatment-emergent adverse events over an average of one year. The investigator will manage any safety concerns and follow participants until any issues resolve or stabilize, after which participants will complete the study with an end-of-study visit. The total participation duration may vary based on individual progress and safety outcomes.

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study