A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy

Recruiting now

Conditions studied: Duchene Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT07127978

Run by

ITF Therapeutics LLC

People needed

300

Starts

2025-10-23

Expected to finish

2030-07-30

Last updated by the study team

2026-07-02

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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).

You may not qualify if…

• 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 it is running

• Arkansas Children's Research Institute — Little Rock, Arkansas, United States (enrolling)
• Children's National Hospital — Washington D.C., District of Columbia, United States (enrolling)
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• University of Massachusetts Chan Medical School — Worcester, Massachusetts, United States (enrolling)
• Columbia University Medical Center — New York, New York, United States (enrolling)
• Duke University / Lenox Baker Children's Hospital — Durham, North Carolina, United States (enrolling)
• Oregon Health and Science University — Portland, Oregon, United States (enrolling)
• Seattle Children's Hospital — Seattle, Washington, United States (enrolling)

Full record on ClinicalTrials.gov

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