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NCT05876221 | RECRUITING | Acquired Thrombotic Thrombocytopenic Purpura


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

University of Cologne

Information provided by (Responsible Party):

Prof. Dr. Paul Brinkkoetter

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

Condition or disease

Acquired Thrombotic Thrombocytopenic Purpura

Intervention/treatment

Cablivi

Study Type : OBSERVATIONAL
Estimated Enrollment : 160 participants
Official Title : Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura
Actual Study Start Date : 2023-10-30
Estimated Primary Completion Date : 2025-03-30
Estimated Study Completion Date : 2025-03-30

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
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

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

Location Details

NCT05876221


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Locations


RECRUITING

Germany,

University Hospital of Cologne

Cologne, Germany,

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