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

NCT04074187 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2025-09-22

No results posted yet for this study

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

Conditions

  • Thrombotic Thrombocytopenic Purpura

Interventions

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)

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-10-21
Primary Completion
2021-05-19
Completion
2021-05-19

Countries

  • Japan

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04074187 on ClinicalTrials.gov