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 range6 Years

SexALL

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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.

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)

Characterize the severity 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