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

NCT07127978 · tracked via the Priya Life Science USA tracker

Sponsor

ITF Therapeutics LLC

Phase

Observational

Started

2025-10-23

Last updated

2026-07-02

Condition(s) studied

Duchene Muscular Dystrophy

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.

Eligibility

Sex

ALL

Min age

6 Years

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat — From follow up (after first date of givinostat treatment) through end of study (up to 5 years)
Incidence rate of thrombocytopenia (per 100 patient years of observation time) post index date
Characterize the frequency of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat — [Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)]
Frequency and incidence proportion (overall and by maximum severity) of thrombocytopenia
Characterize severity of serious events thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat — From follow up (after first date of givinostat treatment) through end of study (up to 5 years)
Time to first thrombocytopenia event from index date
Characterize the incidence of serious events of bleeding in DMD patients treated with oral givinostat — From follow up (after first date of givinostat treatment) through end of study (up to 5 years)
Incidence rate of serious bleeding events (per 100-patient years of observation time) post index date
Characterize the frequency of serious events of bleeding in DMD patients treated with oral givinostat — From follow up (after first date of givinostat treatment) through end of study (up to 5 years)
Frequency and incidence proportion (overall and by maximum severity) of serious bleeding events
Characterize the severity of serious events of bleeding in DMD patients treated with oral givinostat — From follow up (after first date of givinostat treatment) through end of study (up to 5 years)
Time to first serious bleeding event from index date

Trial sites (9)

Facility	City	Region	Status
Arkansas Children's Research Institute	Little Rock	Arkansas	Recruiting
Children's National Hospital	Washington D.C.	District of Columbia	Recruiting
Ann and Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	Recruiting
Boston Children's Hospital	Boston	Massachusetts	Recruiting
University of Massachusetts Chan Medical School	Worcester	Massachusetts	Recruiting
Columbia University Medical Center	New York	New York	Recruiting
Duke University / Lenox Baker Children's Hospital	Durham	North Carolina	Recruiting
Oregon Health and Science University	Portland	Oregon	Recruiting
Seattle Children's Hospital	Seattle	Washington	Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07127978 on ClinicalTrials.gov ↗ ← All trials in the USA