A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
ITF Therapeutics LLC
Summary
This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.
Description
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder that leads to progressive muscle degeneration and weakness. Givinostat, an oral histone deacetylase inhibitor, was approved by the FDA in March 2024 for treatment of DMD in patients aged 6 years and older, regardless of mutation type. Study participation will not influence the course of treatment in any way (including other treatments for DMD), and no treatment will be provided as part of this study. All assessments are intended to be performed at the time of a routine clinical care visit according to clinical practice, and data wil…