NCT07127978Recruiting
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
Source: ClinicalTrials.gov · Last updated 2 Jul 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 300 (estimated)
- First submitted
- 10 Jun 2025
- Start date
- 23 Oct 2025
- Primary completion date
- 30 Jun 2030
- Lead sponsor
- ITF Therapeutics LLC
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT07127978
What you commit to
- Study type: Observational
- Planned participants: 300
- From 23 Oct 2025 to the primary completion date, 30 Jun 2030
- 9 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Who the study is looking for
- 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).
Age in the registry: 6 years to - · Sex: All · Does not accept healthy volunteers
Sites (9)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
| Ann and Robert H. Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Recruiting |
| Duke University / Lenox Baker Children's Hospital Coordinator of this site? Confirm its status | Durham | United States | Recruiting |
| Arkansas Children's Research Institute Coordinator of this site? Confirm its status | Little Rock | United States | Recruiting |
| Columbia University Medical Center Coordinator of this site? Confirm its status | New York | United States | Recruiting |
| Oregon Health and Science University Coordinator of this site? Confirm its status | Portland | United States | Recruiting |
| Seattle Children's Hospital Coordinator of this site? Confirm its status | Seattle | United States | Recruiting |
| Children's National Hospital Coordinator of this site? Confirm its status | Washington D.C. | United States | Recruiting |
| University of Massachusetts Chan Medical School Coordinator of this site? Confirm its status | Worcester | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.