NCT05876221

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 223 (actual)
• Sponsor: University of Cologne · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Interventions

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

Timeline

• Start date: 2023-10-30
• Primary completion: 2025-03-30
• Completion: 2025-03-30
• First posted: 2023-05-25
• Last updated: 2025-09-12

Locations

1 site across 1 country: Germany