Status:

COMPLETED

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

Lead Sponsor:

Sanofi

Conditions:

Thrombotic Thrombocytopenic Purpura

Eligibility:

All Genders

18+ years

Phase:

PHASE2

PHASE3

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 Object...

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.

Eligibility Criteria

Inclusion

  • 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.

Exclusion

    Key Trial Info

    Start Date :

    October 21 2019

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    May 19 2021

    Estimated Enrollment :

    21 Patients enrolled

    Trial Details

    Trial ID

    NCT04074187

    Start Date

    October 21 2019

    End Date

    May 19 2021

    Last Update

    September 22 2025

    Active Locations (12)

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    Page 1 of 3 (12 locations)

    1

    Investigational Site Number 3920009

    Iruma-Gun, Japan

    2

    Investigational Site Number 3920014

    Kanazawa, Japan

    3

    Investigational Site Number 3920007

    Kashihara-shi, Japan

    4

    Investigational Site Number 3920013

    Kawasaki-Shi, Japan