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.
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).
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.
This study will be conducted in the US and plans to enroll 300 patients diagnosed with DMD and treated with givinostat as part of their routine clinical care. Patients will be recruited from expert centers treating DMD patients with specialists in neuromuscular conditions who are routinely involved in the care and treatment of patients with DMD, some of whom would have already gained experience with givinostat.
Cohort
Prospective
Not listed
Characterize the incidence of thrombocytopenia / decreased platelet counts in DMD patients treated with oral givinostat
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
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
Time to first thrombocytopenia event from index date
Characterize the incidence of serious events of bleeding in DMD patients treated with oral givinostat
Incidence rate of serious bleeding events (per 100-patient years of observation time) post index date
Characterize the incidence of other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat
Incidence rates of each AESI (per 100-patient years of observation time) post index date as well as the incidence proportion of AESIs resulting in dose modifications or givinostat discontinuation
Characterize the frequency other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat
Frequency and incidence proportion (overall and by maximum severity) of each AESI
Characterize the severity of other adverse events of special interest (AESIs) in DMD patients treated with oral givinostat
Time to first event of each AESI from index date
Characterize the incidence of all serious adverse events (SAEs) in DMD patients treated with oral givinostat
Incidence rates of SAEs (per 100-patient years of observation time) post index date by SOC and PT within SOC, and the number of SAEs per patient.
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