Observational Study of Oral Givinostat Treatment in Patients Aged 6 and Older With Duchenne Muscular Dystrophy

What this Trial Is About

Researchers are conducting a prospective observational study to evaluate the safety, tolerability, and functional outcomes of patients with Duchenne Muscular Dystrophy DMD who are newly starting oral givinostat or have started it within the past 6 months as part of routine clinical care in the United States. Givinostat is an oral histone deacetylase inhibitor approved by the FDA in March 2024 for treating DMD in patients aged 6 years and older. The study aims to gather real-world data on adverse events, treatment patterns, and effectiveness in this population over a long period. Participants include up to 300 patients divided into two cohorts ambulatory patients able to complete a 10-meter walk-run test within 30 seconds and non-ambulatory patients unable to perform this test. The study does not provide any treatment but observes patients receiving oral givinostat as part of their regular care. The study will last up to 5 years, including a 24-month enrollment period and at least 2 years of follow-up, during which data from medical records and routine clinical visits will be collected. Throughout the study, participants will undergo assessments such as laboratory tests including platelet counts and triglycerides, cardiac and lung function evaluations, patient-reported outcomes, and motor function tests like the North Star Ambulatory Assessment NSAA and Performance of Upper Limb PUL scale. The study will monitor serious events related to thrombocytopenia and bleeding, as well as other adverse events and treatment patterns. Data collection aligns with routine care visits, and no additional treatments or interventions are administered as part of the study.

A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy