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A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Sponsor: ITF Therapeutics LLC
Summary
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.
Official title: A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Key Details
Gender
All
Age Range
6 Years - Any
Study Type
OBSERVATIONAL
Enrollment
300
Start Date
2025-10-23
Completion Date
2030-07-30
Last Updated
2026-04-15
Healthy Volunteers
No
Conditions
Locations (7)
Arkansas Children's Research Institute
Little Rock, Arkansas, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States
Columbia University Medical Center
New York, New York, United States
Seattle Children's Hospital
Seattle, Washington, United States