Real-world experience of oral givinostat for Duchenne muscular dystrophy in the U.S.
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
The study will track safety and functional outcomes in people aged 6 and older with Duchenne muscular dystrophy who start oral givinostat during routine U.S. care to see how the medicine is tolerated and how patients do over time.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 300 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | ITF Therapeutics LLC Industry-sponsored |
| Locations | 7 sites (Little Rock, Arkansas and 6 other locations) |
| Trial ID | NCT07127978 on ClinicalTrials.gov |
What this trial studies
This is a prospective, observational U.S. study that follows patients with genetically confirmed Duchenne muscular dystrophy who are newly starting oral givinostat or who began treatment within the prior six months. No treatments are provided by the study; data are collected from routine clinical visits and medical records over a planned minimum follow-up of two years and up to five years for early enrollees. The primary focus is on safety, including monitoring for thrombocytopenia and serious bleeding events, along with other adverse events, gastrointestinal effects, and functional outcomes. The FDA requires this post‑marketing study to better characterize the incidence, frequency, and severity of these safety events in real-world practice.
Who should consider this trial
Good fit: Ideal participants are people aged 6 years or older with genetically confirmed DMD who have been prescribed oral givinostat according to the U.S. label, can provide consent (and assent when applicable), and have the required medical record and laboratory data with treatment started no more than six months before enrollment.
Not a fit: Patients who previously discontinued givinostat, who started commercial givinostat more than six months before consent, or who lack necessary medical records or lab results are unlikely to gain meaningful information from joining this observational study.
Why it matters
Potential benefit: If successful, the study could clarify real-world safety risks such as thrombocytopenia and bleeding and help guide safer use of givinostat in people with DMD.
How similar studies have performed: Prior clinical trials of givinostat contributed to FDA approval, but prospective real-world safety monitoring studies like this are complementary and aim to capture events not always seen in trials.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Little Rock, Arkansas and 6 other locations
- Arkansas Children's Research Institute — Little Rock, Arkansas, United States (Recruiting)
- Children's National Hospital — Washington D.C., District of Columbia, United States (Recruiting)
- Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- University of Massachusetts Chan Medical School — Worcester, Massachusetts, United States (Recruiting)
- Columbia University Medical Center — New York, New York, United States (Recruiting)
- Seattle Children's Hospital — Seattle, Washington, United States (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.