Follows boys with Duchenne muscular dystrophy taking givinostat in routine care
Observes safety and daily function in patients starting oral givinostat for Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Key facts
- Where
- Boston, Chicago, Durham, Little Rock, New York, Portland, Seattle, Washington D.C., and Worcester, United StatesSee the 9 sites
Where in United States
- Boston Children's HospitalBoston, United StatesTaking people
- Ann and Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesTaking people
- Duke University / Lenox Baker Children's HospitalDurham, United StatesTaking people
- Arkansas Children's Research InstituteLittle Rock, United StatesTaking people
- Columbia University Medical CenterNew York, United StatesTaking people
- Oregon Health and Science UniversityPortland, United StatesTaking people
- Seattle Children's HospitalSeattle, United StatesTaking people
- Children's National HospitalWashington D.C., United StatesTaking people
- University of Massachusetts Chan Medical SchoolWorcester, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- ITF Therapeutics LLC
- Trial code
- NCT07127978
- Record updated
- 2 July 2026
- Original description
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.Read full original descriptionShow less
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
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.
Inclusion: 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: 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.