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A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Status: Recruiting
Location: See all (7) locations...
Study Type: Observational
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• 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).
Locations
United States
Arkansas
Arkansas Children's Research Institute
RECRUITING
Little Rock
Washington, D.c.
Children's National Hospital
RECRUITING
Washington D.c.
Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
University of Massachusetts Chan Medical School
RECRUITING
Worcester
New York
Columbia University Medical Center
RECRUITING
New York
Washington
Seattle Children's Hospital
RECRUITING
Seattle
Time Frame
Start Date:2025-10-23
Estimated Completion Date:2030-07-30
Participants
Target number of participants:300
Treatments
Ambulatory
Ambulatory is defined as being able to complete the 10-meter Walk-Run (10mWR) test within 30 seconds without an assistive device.~This cohort will include up to 120 patients who are ambulatory prior to starting givinostat treatment.~Within this cohort , 95 patients must have had prior use or a referral for concomitant use of dystrophin-enhancing therapy (DET), such as exon skipping oligonucleotides or gene therapy.~Enrollment is capped at 25 patients without prior or referred DET use.
Non-Ambulatory
Non-ambulatory status is determined by inability to perform the 10mWR test as defined above.~This cohort will include approximately 180 patients who are non-ambulatory prior to starting givinostat.