An open-label, single-arm, multicenter study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy without first-line therapeutic plasma exchange in adults with immunemediated thrombotic thrombocytopenic purpura

Conditions

Thrombotic Thrombocytopenic Purpura

Brief summary

Proportion of participants achieving Remission without requiring therapeutic plasma exchange (TPE).

Detailed description

Proportion of participants achieving Remission, Proportion of participants who require TPE, The occurrence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs), Proportion of participants achieving Clinical Response (On-treatment period from day 1 to day 84), Proportion of participants achieving Clinical Response (Overall study period from day 1 to day 168), Time to platelet count response, Proportion of participants refractory to therapy, Proportion of participants with TTP-related death (On-treatment period from day 1 to day 84), Proportion of participants with TTP-related death (Overall study period from day 1 to day 168), Proportion of participants with a clinical exacerbation of iTTP (On-treatment period from day 1 to day 84), Proportion of participants with a clinical exacerbation of iTTP (Overall study period from day 1 to day 168), Proportion of participants with a clinical relapse of iTTP (On-treatment period from day 1 to day 84), Proportion of participants with a clinical relapse of iTTP (Overall study period from day 1 to day 168)

Related papers

No related papers found yet.

Interventions

DRUGCablivi 10 mg powder and solvent for solution for injection

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Proportion of participants achieving Remission without requiring therapeutic plasma exchange (TPE).	—

Secondary

Measure	Time frame
Proportion of participants achieving Remission, Proportion of participants who require TPE, The occurrence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs), Proportion of participants achieving Clinical Response (On-treatment period from day 1 to day 84), Proportion of participants achieving Clinical Response (Overall study period from day 1 to day 168), Time to platelet count response, Proportion of participants refractory to therapy, Proportion of participants with TTP-related death (On-treatment period from day 1 to day 84), Proportion of participants with TTP-related death (Overall study period from day 1 to day 168), Proportion of participants with a clinical exacerbation of iTTP (On-treatment period from day 1 to day 84), Proportion of participants with a clinical exacerbation of iTTP (Overall study period from day 1 to day 168), Proportion of participants with a clinical relapse of iTTP (On-treatment period from day 1 to da	—

Measure

Time frame

Proportion of participants achieving Remission, Proportion of participants who require TPE, The occurrence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs), Proportion of participants achieving Clinical Response (On-treatment period from day 1 to day 84), Proportion of participants achieving Clinical Response (Overall study period from day 1 to day 168), Time to platelet count response, Proportion of participants refractory to therapy, Proportion of participants with TTP-related death (On-treatment period from day 1 to day 84), Proportion of participants with TTP-related death (Overall study period from day 1 to day 168), Proportion of participants with a clinical exacerbation of iTTP (On-treatment period from day 1 to day 84), Proportion of participants with a clinical exacerbation of iTTP (Overall study period from day 1 to day 168), Proportion of participants with a clinical relapse of iTTP (On-treatment period from day 1 to da

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Countries

Austria, Belgium, Czechia, France, Germany, Greece, Italy, Netherlands, Spain

Outcome results

None listed