A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Status:
Completed
Trial end date:
2021-05-19
Target enrollment:
Participant gender:
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