NCT04074187

A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Thrombotic Thrombocytopenic Purpura

Interventions

Type	Name	Description
DRUG	Caplacizumab (ALX-0081)	Pharmaceutical form:Lyophilized powder for solution for injection Route of administration: IV (first dose), SC (all subsequent doses)
DRUG	Plasma exchange (PE)	Pharmaceutical form:Plasma (e.g. fresh frozen plasma) Route of administration: Plasma exchange
DRUG	Corticosteroid treatment (Methylprednisolone or prednisolone)	Pharmaceutical form:Solution for injection or Tablet Route of administration: IV or Oral
DRUG	Immunosuppressive treatment (eg, rituximab)	Pharmaceutical form:Solution for injection (depending on product) Route of administration: IV (depending on product)

Timeline

• Start date: 2019-10-21
• Primary completion: 2021-05-19
• Completion: 2021-05-19
• First posted: 2019-08-29
• Last updated: 2025-09-22

Locations

12 sites across 1 country: Japan