RecruitingNot Applicable

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

United States300 participantsStarted 2025-10-23

Plain-language 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.

Who can participate

Age range

6 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

Characterize the frequency of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat

Timeframe: [Time Frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)]

Characterize severity of serious events thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat

Timeframe: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

Characterize the incidence of serious events of bleeding in DMD patients treated with oral givinostat

Timeframe: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

Characterize the frequency of serious events of bleeding in DMD patients treated with oral givinostat

Timeframe: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)

Trial details

NCT IDNCT07127978

SponsorITF Therapeutics LLC

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2030-06-30

View on ClinicalTrials.gov More Duchene Muscular Dystrophy trials