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Observational Study of Givinostat in Duchenne Muscular Dystrophy

This is an observational study looking at the real-world experience of givinostat in patients with Duchenne Muscular Dystrophy (DMD). Givinostat is an oral medication approved for DMD in patients aged 6 and older. This study aims to understand the safety of givinostat, specifically focusing on how often and how severely patients experience thrombocytopenia (low platelet counts, which help blood clot). You may be able to join if you are at least 6 years old, have DMD confirmed by a genetic test, and have a prescription for oral givinostat. The study is currently unclear on its recruitment status and plans to enroll about 300 participants. This study will not change your treatment plan; it will gather information from your routine medical care.

Study design
This is an observational study, meaning it will collect information about your care without providing any treatment. It is designed to follow patients for up to 5 years.
What's involved
Your participation will involve sharing information from your routine medical records. All assessments will be done during your regular doctor visits.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed from the start of givinostat treatment through the end of the study, for up to 5 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07127978

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

Recruiting
Not specifiedAges 6+Observational
ITF Therapeutics LLC
~300 participants
Updated 2026-07-02 on ClinicalTrials.gov

At a glance

Recruiting sites
9 of 9 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat
Measured over From follow up (after first date of givinostat treatment) through end of study (up to 5 years)
+5 more outcomes measured

Conditions

Duchene Muscular Dystrophy

Explore related trials

Duchenne Muscular Dystrophy

NCT07127978

Where you'd take part

This study runs at 9 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinoisstudy coordinator listed

    Recruiting

  • Arkansas Children's Research Institute

    Little Rock, Arkansasstudy coordinator listed

    Recruiting

  • Boston Children's Hospital

    Boston, Massachusettsstudy coordinator listed

    Recruiting

  • Children's National Hospital

    Washington D.C., District of Columbiastudy coordinator listed

    Recruiting

  • Columbia University Medical Center

    New York, New Yorkstudy coordinator listed

    Recruiting

  • Duke University / Lenox Baker Children's Hospital

    Durham, North Carolinastudy coordinator listed

    Recruiting

  • Oregon Health and Science University

    Portland, Oregonstudy coordinator listed

    Recruiting

  • Seattle Children's Hospital

    Seattle, Washingtonstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Domencia Hunter
[email protected]
973-932-9211
Email the study team

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Eligibility criteria

Inclusion

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

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.

What this trial measures

  • Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostatFrom 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 givinostatFrom 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 givinostatFrom 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 givinostatFrom 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 givinostatFrom 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