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

Sponsor
Sanofi
Study ID
NCT04074187
Phase
PHASE2/PHASE3
Status
Completed

Conditions

  • Thrombotic Thrombocytopenic Purpura

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • 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)

Study Details

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

Key Dates

Start date
Oct 21, 2019
Status verified
Sep 2025
Primary completion
May 19, 2021
Completion
May 19, 2021

Study Design

Enrollment
21 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Caplacizumab
    Eligible study participants will receive caplacizumab in addition to standard of care such as daily plasma exchange (PE) and corticosteroid treatment (mandatory), immunosuppressive treatment (if needed)

Primary Outcome Measure

Proportion of participants with a recurrence of acquired thrombotic thrombocytopenic purpura (aTTP) [ Time Frame: Approximately 2 months up to approximately 6 months ]

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