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

NCT05876221 | Completed

• 1 other identifier: 2020-11-17
• Study Type: observational
• Target: 223
• Locations: 1 country
• Sites: 1

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

Keywords

caplacizumab; platelet responses; aTTP; iTTP

Outcome Measures

Primary Outcomes (1)

• Reliable description and prediction of platelet responses to caplacizumab

  Reliable description and prediction of platelet responses to caplacizumab employing mathematic modelling algorithms

  Enrollment

Secondary Outcomes (3)

• Determination of different clusters of platelet responses to caplacizumab

  Enrollment

• Correlation of platelet responses to caplacizumab with patient outcome

  Enrollment

• Risk stratification of iTTP patients based on their platelet response to caplacizumab

  Enrollment

Study Arms (1)

aTTP-Patients

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

Drug: Cablivi

Interventions

Cablivi DRUG

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

aTTP-Patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

In this trial will be enclosed iTTP patients treated with caplacizumab from: * the German real-world cohort of iTTP patients treated with caplacizumab in the years 2018 to 2020 , and * the respective TITAN and HERCULES trial cohorts.

You may qualify if:

• 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

You may not qualify if:

• Hereditary thrombotic thrombocytopenic purpura

Sponsors & Collaborators

• University of Cologne: lead

Study Sites (1)

University Hospital of Cologne

Cologne, Germany

Location

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Interventions

caplacizumab

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Lucas Kühne, MD

  Department II of Internal Medicine, University Hospital of Cologne

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Consultant in Nephrology

Study Record Dates

• First Submitted: May 16, 2023
• First Posted: May 25, 2023
• Study Start: October 30, 2023
• Primary Completion: March 30, 2025
• Study Completion: March 30, 2025
• Last Updated: September 12, 2025

Record last verified: 2025-09

Locations

DE (1)