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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
2025-10-30
Healthy Volunteers
No
Conditions
Locations (2)
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States