Actively Recruiting
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Led by ITF Therapeutics LLC · Updated on 2026-04-15
300
Participants Needed
7
Research Sites
248 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 will not qualify if you...
- Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment.
- Patient started commercial givinostat for > 6 months before signing of informed consent.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 7 locations
1
Arkansas Children's Research Institute
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
3
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States, 01655
Actively Recruiting
6
Columbia University Medical Center
New York, New York, United States, 10032
Actively Recruiting
7
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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