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

Recruitingn/aAges 6 Years - anyAllITF Therapeutics LLC
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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.
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What it tests

  • Not listed
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Where

9 sites
Arkansas Children's Research Institute
Little Rock, United States
Children's National Hospital
Washington D.C., United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
Boston Children's Hospital
Boston, United States
University of Massachusetts Chan Medical School
Worcester, United States
Columbia University Medical Center
New York, United States
Duke University / Lenox Baker Children's Hospital
Durham, United States
Oregon Health and Science University
Portland, United States

โ€ฆand 1 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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