Study 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.
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Request More InfoInterventions
No interventions listed.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| 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 |