Home › Duchenne Muscular Dystrophy › NCT07127978
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Who can join
Ages 6 and older · All sexes
Full eligibility criteria
Inclusion Criteria: * Patients of any gender at least 6 years of age, diagnosed with DMD (according to genetic test) and have received a prescription for oral givinostat in accordance with the USPI. * Patient has provided informed consent (and assent when applicable) for participation in the study. * Patient's index date (first date of givinostat treatment) is no more than 6 months prior to signing of informed consent. * Patient has the required data available (DMD diagnosis, givinostat administration \[dose and schedule\], laboratory results \[hematology and triglycerides\]) for addressing the study objectives for the period between index date and study entry (for patients enrolled after the index date). Exclusion Criteria: * Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment or patient started commercial givinostat for \> 6 months before signing of informed consent.
About the study
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.
Sponsor: ITF Therapeutics LLC · Participants: 300 · Started: Oct 23, 2025
Contact the study team
Official record on ClinicalTrials.gov — NCT07127978
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.