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

NCT ID: NCT05876221

Last Updated: 2025-09-12

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 223 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-10-30
• Study Completion Date: 2025-03-30

Brief 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

Conditions

Acquired Thrombotic Thrombocytopenic Purpura

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

aTTP-Patients

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

Cablivi

Intervention Type: DRUG

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

Interventions

Cablivi

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

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Cologne

OTHER

Sponsor Role: lead

Responsible Party

Prof. Dr. Paul Brinkkoetter

Consultant in Nephrology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Lucas Kühne, MD

Role: PRINCIPAL_INVESTIGATOR

Department II of Internal Medicine, University Hospital of Cologne

Locations

University Hospital of Cologne

Cologne, , Germany

Countries

Germany

Other Identifiers

2020-11-17

Identifier Type: -

Identifier Source: org_study_id