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Completed

NCT Number: NCT04074187

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

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

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Key information

Conditions

Thrombotic Thrombocytopenic Purpura Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombophilia Thrombotic Microangiopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Investigational Site Number 3920009, Iruma-Gun, Japan

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Japanese participant must be 18 years or older at the time of signing the informed consent.
  • Participants who have a clinical diagnosis of aTTP (initial or recurrent), which includes thrombocytopenia (defined as platelet count <100,000/µL), microangiopathic hemolytic anemia as evidenced by red blood cell fragmentation (eg, presence of schistocytes), and increased levels of LDH
  • Participants who require initiation of daily PE treatment and have received a maximum of 1 PE treatment prior to enrollment in the study
  • Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
  • Capable of giving signed informed consent

Exclusion criteria

  • Platelet count ≥100,000/µL,
  • Serum creatinine level > 2.3mg/dL in case platelet count is > 30,000µL
  • Known other causes of thrombocytopenia
  • Congenital TTP
  • Clinically significant active bleeding or high risk of bleeding
  • Malignant arterial hypertension
  • Known chronic treatment with anticoagulant treatment that cannot be stopped
  • Participants who were previously enrolled in a clinical study with caplacizumab and received caplacizumab or for whom the assigned treatment arm is unknown
  • Participants currently or less than 28 days prior to enrollment in this study, enrolled in a clinical study with another investigational drug or device
  • Clinical condition other than that associated with TTP, with life expectancy < 6 months, such as end-stage malignancy
  • Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
  • Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Treatment and study plan

Caplacizumab (ALX-0081)

Drug

Pharmaceutical form:Lyophilized powder for solution for injection Route of administration: IV (first dose), SC (all subsequent doses)

Plasma exchange (PE)

Drug

Pharmaceutical form:Plasma (e.g. fresh frozen plasma) Route of administration: Plasma exchange

Corticosteroid treatment (Methylprednisolone or prednisolone)

Drug

Pharmaceutical form:Solution for injection or Tablet Route of administration: IV or Oral

Immunosuppressive treatment (eg, rituximab)

Drug

Pharmaceutical form:Solution for injection (depending on product) Route of administration: IV (depending on product)

Primary outcomes

  1. Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP)

    Time frame: Approximately 2 months up to approximately 6 months

    Proportion of participants with a recurrence of aTTP during the overall study period. The success criterion for this study is proportion of evaluable participants (per-protocol population) with a recurrence of aTTP during the overall study period to be 20% or less.

Secondary outcomes

  1. Number of recurrences of TTP

    Time frame: Approximately 2 months up to approximately 6 months

    Number of recurrences of TTP during study drug treatment (including extensions) and follow-up, as well as during overall study period.

  2. Proportion of participants with composite endpoint consisting of aTTP-related mortality, recurrence of aTTP and major thromboembolic events

    Time frame: Approximately 2 months up to approximately 6 months

    Proportion of participants with TTP-related death, a recurrence of TTP, or at least one treatmentemergent major thromboembolic event (eg, myocardial infarction, cerebrovascular accident, pulmonary embolism or deep venous thrombosis [DVT]) during the overall treatment period (including extensions).

  3. Time to platelet count response

    Time frame: Approximately 2 months up to approximately 6 months

    Time to platelet count response, defined as initial platelet count ≥150,000/μL with subsequent stop of daily plasma exchange (PE) within 5 days.

  4. Proportion of participant who have a platelet count ≥150,000/μL

    Time frame: Approximately 2 months up to approximately 6 months

    Proportion of participant who have a platelet count ≥150,000/μL on Day 1, 2, 3, 4, 5 and Day 10 and end of study drug treatment (ie, last weekly visit during the overall treatment period).

  5. Proportion of participants with refractory TTP

    Time frame: Approximately 2 months up to approximately 6 months

    Proportion of participant with refractory TTP, defined as persistent thrombocytopenia, lack of sustained platelet count increment or platelet counts <50,000/μL and persistently elevated LDH (>1.5 x upper limit of normal [ULN]) despite 5 PEs and steroid treatment.

  6. Time to normalization of 3 organ damage marker levels

    Time frame: Approximately 2 months up to approximately 6 months

    Time to normalization of all 3 of the following organ damage marker levels: Time to LDH ≤ 1 x ULN, and cTnI ≤ 1 x ULN, and serum creatinine ≤ 1 x ULN and time to individual organ damage marker level.

  7. Time to stop of daily plasma exchnage (PE)

    Time frame: Approximately 2 months up to approximately 6 months

    Time to stop of daily PE

  8. Number of days to plasma exchange

    Time frame: Approximately 2 months up to approximately 6 months

    The endpoint will be assessed in 4 time periods: daily PE period (the first daily PE period only), overall treatment period, follow-up period (of 4 weeks after stop of study drug treatment), and overall study period

  9. Total volume of plasma

    Time frame: Approximately 2 months up to approximately 6 months

    The endpoint will be assessed in 4 time periods: daily PE period (the first daily PE period only), overall treatment period, follow-up period (of 4 weeks after stop of study drug treatment), and overall study period

  10. Number of days in ICU and in hospital

    Time frame: Approximately 2 months up to approximately 6 months

    Number of days in ICU and in hospital in 4 time periods: daily PE period (the first daily PE period only), overall treatment period, in the Follow-up period (of 4 weeks after stop of study drug treatment) and overall study period.

  11. Change from baseline in the standardized mini mental state exam (SMMSE) total score

    Time frame: Approximately 2 months up to approximately 6 months

    Change from baseline in SMMSE total score on Day 1, (Day 2, 3, 4 as optional) and Day 5 of daily Plasma Exchange (PE) period, and Weeks 1 and 5 of the 30-day postdaily PE period, and the first (7 days after last dosing) and final follow-up (28 days after last dosing) visit.

  12. Proportion of participants with at least one treatment emergent thromboembolic event

    Time frame: Approximately 2 months up to approximately 6 months

    The treatment-emergent major thromboembolic event (eg, myocardial infarction, cerebrovascular accident, pulmonary embolism or deep venous thrombosis [DVT]) during the overall treatment period (including extensions) will be evaluated.

  13. Number of patients with treatment emergent adverse events

    Time frame: Approximately 2 months up to approximately 6 months

    Number of Patients with treatment emergent Adverse events (AEs) and serious adverse events (SAEs) and bleeding events

  14. Pharmacodynamic (PD) markers

    Time frame: Approximately 2 months up to approximately 6 months

    PD parameters: von Willebrand factor antigen(vWF:Ag), coagulation factor VIII clotting activity (FVIII:C), von Willebrand factor ristocetin cofactor activity (vWF:RICO)

  15. Pharmacokineticks: plasma concentration

    Time frame: Approximately 2 months up to approximately 6 months

    Total caplacizumab plasma concentrations

  16. Immunogenicity of caplacizumab

    Time frame: Approximately 2 months up to approximately 6 months

    Anti-drug antibodies

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2021
Study completion
2021
First posted
Aug 29, 2019
Registry last updated
Sep 22, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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