The purpose of this prospective observational study (NCT07127978) is to assess the safety, tolerability, and functional results of DMD patients who are starting oral Duvyzat (Givinostat) for the first time or who have already begun therapy within six months as part of standard clinical care in the United States. With a 24-month enrollment period and a minimum of two years of follow-up, the trial is expected to last up to five years for the initial patients recruited.
Learn More: What is Duvyzat (Givinostat)?
The study will enroll 300 participants (180 non-ambulatory and up to 120 ambulatory patients). Participants will be followed for at least two years, with data collected during routine clinical visits. Assessments include lab parameters (e.g., platelet counts, triglycerides), cardiac and pulmonary function, patient-reported outcomes, and motor function scales such as the North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL) scale.
Duvyzat (Givinostat) Study – NCT07127978 –
This study evaluating the Real-World Experience of Givinostat in patients with Duchenne Muscular Dystrophy.
Study Start (Estimated): 2025-09-15
Primary Completion (Estimated): 2030-06-30
Study Completion (Estimated): 2030-07-30
Number of Participants (Estimated): 300
Study Type: Observational
Locations: No location data yet.
Ages Eligible for Study: 6 Years and older (Child, Adult, Older Adult)
Principal inclusion and exclusion criteria. – NCT07127978 –
