A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Sponsor: ITF Therapeutics LLC
No open prediction endpoints
Endpoints are classified and published by ProgramSignal analysts.
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Primary Endpoints (CT.gov)
Characterize the incidence 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)
Characterize the frequency of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat
Time frame: [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
Time frame: 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
Time frame: From follow up (after first date of givinostat treatment) through end of study (up to 5 years)
Secondary Endpoints
Characterize the incidence of other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat
Characterize the frequency other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat
Characterize the severity of other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat
Eligibility Criteria
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.
✦ Analyst Commentary
Expert commentary on why this trial matters and what to watch for.
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