A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
brief summary
Primary Objective: To evaluate the effect of caplacizumab on prevention of recurrence of aTTP (proportion of participants with a recurrence of aTTP) during the overall study period. Secondary Objectives: * To evaluate effect of caplacizumab on * prevention of recurrence of TTP (the number of recurrences of TTP) during overall study period. * a composite endpoint consisting of aTTP-related mortality, recurrence of aTTP and major thromboembolic events during study drug treatment * restoring platelet counts as a measure of prevention of further microvascular thrombosis * refractory disease * biomarkers of organ damage: LDH, cardiac troponin I, serum creatinine * plasma exchange (PE) parameters (days of PE and volume of plasma used), days in intensive care unit, days in hospital * cognitive status of Japanese patients * To evaluate safety profile of caplacizumab in Japanese patients * To evaluate effect of caplacizumab on pharmacodynamic (PD) markers in Japanese patients * To evaluate pharmacokinetic (PK) profile of caplacizumab in Japanese patients * To evaluate immunogenicity of caplacizumab in Japanese patients
detailed description
Study duration per participant is approximately 2 months up to approximately 6 months in case of treatment extension and recurrence during the study drug treatment period.
official title
An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura