Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura

Acquired Thrombotic Thrombocytopenic Purpura Clinical Trial

— PREDICT2020  

Official title:

Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05876221
• Other study ID #: 2020-11-17
• Status: Recruiting
• Start date: October 30, 2023
• Est. completion date: June 30, 2024

Study information

• Verified date: November 2023
• Source: University of Cologne
• Contact: Lucas Kühne, MD
• Phone: +49 221 478 36990
• Email: lucas.kuehne[@]uk-koeln.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The interpretation of platelet counts has to be revaluated in the light of caplacizumab. By effectively blocking platelet binding sites on VWF-multimers, the nanobody leads to a rapid normalization of the platelet count within 3 to 4 days. Most importantly, caplacizumab uncouples platelet counts from ADAMTS13 activity and thereby launches unprecedented thrombocyte dynamics, with potential pitfalls for over- and undertreatment.

A relevant number of patients responds to caplacizumab with a brisk increase in platelet count, followed by a marked dip of platelets (patient on the left). This may mislead treating physicians into re-intensifying therapy, with a respective risk for adverse side-effects and complications. Taken together, these observations call for reliable descriptions and the identification of predictive parameters to predict the platelet response upon administration of caplacizumab in a large patient cohort. Here, PREDICT-2020 is designed as a retrospective study to specifically address the following aspects:

• Identifying and describing clusters of platelet responses to caplacizumab

• Identifying potential pitfalls for treating physicians

• Predicting the individual thrombocyte response

• Correlating platelet responses with individual patient outcome

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 160
• Est. completion date: June 30, 2024
• Est. primary completion date: June 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Confirmed diagnosis of autoimmune thrombotic thrombocytopenic purpura

• Treatment with at least a single dose of caplacizumab, either i.v. or s.c.

• Male or female patients older than 18 years of age

Exclusion Criteria:

• Hereditary thrombotic thrombocytopenic purpura

Related Conditions & MeSH terms

• Acquired Thrombotic Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombotic Thrombocytopenic

Intervention

Drug:
• Cablivi
Patients with immune Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®)

Locations

Country	Name	City	State
Germany	University Hospital of Cologne	Cologne

Sponsors (1)

Lead Sponsor	Collaborator
University of Cologne

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reliable description and prediction of platelet responses to caplacizumab	Reliable description and prediction of platelet responses to caplacizumab employing mathematic modelling algorithms	Enrollment
Secondary	Determination of different clusters of platelet responses to caplacizumab	It will be hypothesized the existence of different clusters of caplacizumab responders during the first weeks of therapy, when ADAMTS13 activity typically is still <10%. A detailed cluster analysis and description of thrombocyte responses to caplacizumab in a large cohort will reliably identify these different responders	Enrollment
Secondary	Correlation of platelet responses to caplacizumab with patient outcome	It will be hypothesized the existence of different clusters of caplacizumab responders during the first weeks of therapy, when ADAMTS13 activity typically is still <10%. A detailed cluster analysis and description of thrombocyte responses to caplacizumab in a large cohort will reliably identify these different responders	Enrollment
Secondary	Risk stratification of iTTP patients based on their platelet response to caplacizumab	Description: Predicting the individual thrombocyte response to caplacizumab improves risk stratification of iTTP patients after initiation of caplacizumab therapy. An early risk stratification allows an optimal timing of monitoring intervals during the first weeks after diagnosis, which are often critical	Enrollment