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RECRUITING
NCT07127978

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

Sponsor: ITF Therapeutics LLC

View on ClinicalTrials.gov

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

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