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

Status: Completed
Location: See location...
Intervention Type: Drug
Study Type: Observational
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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Germany
University Hospital of Cologne
Cologne
Time Frame
Start Date: 2023-10-30
Completion Date: 2025-03-30
Participants
Target number of participants: 223
Treatments
aTTP-Patients
Patients with immune Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®)
Related Therapeutic Areas
Sponsors
Leads: University of Cologne

This content was sourced from clinicaltrials.gov