RECRUITINGOBSERVATIONAL
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
- 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 willing to sign a consent form (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 willing to sign a consent form.
- 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).
Who Should NOT Join This Trial:
- Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment or patient started commercial givinostat for \> 6 months before signing of willing to sign a consent form.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
* 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).
Exclusion Criteria:
* Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment or patient started commercial givinostat for \> 6 months before signing of informed consent.
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