Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP (REACT-2020)

Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura (aTTP)- REACT-2020

The objective of this national, prospective, multi-centre observational study is to describe the prescription rational and practice in Germany, confirm the efficacy of caplacizumab in a real-world setting, and identify predicting factors in iTTP-patients with regard to persistent autoimmune activity, therapy guidance and risk of complications. The rational is to develop new treatment algorithms that optimize overall patient outcome and reduce treatment cost.

Study Overview

• Status: Recruiting
• Conditions: Acquired Thrombotic Thrombocytopenic Purpura
• Intervention / Treatment: Drug: Cablivi®

• Study Type: Observational
• Enrollment (Anticipated): 350

Contacts and Locations

• Study Contact: Name: Linus Völker, MD; Phone Number: +49 221 478 86285; Email: linus.voelker@uk-koeln.de
• Study Contact Backup: Name: Lucas Kühne, MD; Email: lucas.kuehne@uk-koeln.de

Study Locations

• Germany
  • Cologne, Germany, 50937
    • Recruiting
    • University Hospital of Cologne
    • Principal Investigator: Linus Völker, MD
    • Contact: Linus Völker, MD; Phone Number: +49 221 478 86285; Email: linus.voelker@uk-koeln.de
    • Contact: Lucas Kühne, MD; Email: lucas.kuehne@uk-koeln.de
    • Sub-Investigator: Paul Brinkkötter, Prof.
    • Sub-Investigator: Lucas Kühne, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Male or female subjects with confirmed diagnosis of an acute episode of aquired thrombotic thrombocytopenic purpura, who have been treated with one dose of caplacizumab.

Description

Inclusion Criteria:

• Confirmed diagnosis of an acute episode of acquired thrombotic thrombocytopenic purpura
• Treatment with at least one single dose of caplacizumab (10 mg i.v. or s.c.)
• Male or female patients ≥ 18 years of age
• signed written informed consent

Exclusion Criteria:

• Hereditary thrombotic thrombocytopenic purpura
• disability to give informed consent

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Registergroup; Patients with acquired Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®)	Drug: Cablivi®; Intervention with Cablivi® take place outside of the study

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment of aTTP with caplacizumab	Description of the prescription rationale and practice of caplacizumab in the treatment of aTTP	Enrollment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Stabilization of thrombocyte	Documentation of the efficacy of caplacizumab in the treatment of aTTP outside of clinical trials (real-world data) using by means of stabilization of thrombocyte. Thrombocyte stabilization is defined as thrombocyte > 150 x 10E9/L	Enrollment
Normalization of LDH	Documentation of the efficacy of caplacizumab in the treatment of aTTP outside of clinical trials (real-world data) using by means of normalization of Lactate dehydrogenase (LDH). Normalization of LDH is defined as LDH below upper limit of normal	Enrollment
Normalization of haptoglobin	Documentation of the efficacy of caplacizumab in the treatment of aTTP outside of clinical trials (real-world data) using by means of normalization of haptoglobin. Normalization of haptoglobin is defined as haptoglobin above lower limit of normal	Enrollment
Risk factors for complications	Identification of risk factors for complications	Enrollment
Risk factors for adverse outcomes	Identification of risk factors for adverse outcomes	Enrollment
Risk factors for persistent autoimmune activity	Identification of risk factors for persistent autoimmune activity	Enrollment
Parameters for therapy guidance	Identification of parameters that guide duration and regimen of caplacizumab treatment	Enrollment
Effect of caplacizumab on adjunct treatments with PEX	Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of therapeutic plasma exchange therapy (PEX)	Enrollment
Effect of caplacizumab on adjunct treatments	Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of glucocorticoids	Enrollment

Collaborators and Investigators

• Sponsor: University of Cologne
• Investigators: Principal Investigator: Linus Völker, MD, Department II of Internal Medicine, University of Cologne

Study record dates

Study Major Dates

• Study Start (Actual): March 25, 2021
• Primary Completion (Anticipated): October 1, 2024
• Study Completion (Anticipated): March 1, 2025

Study Registration Dates

• First Submitted: July 21, 2021
• First Submitted That Met QC Criteria: July 21, 2021
• First Posted (Actual): August 2, 2021

Study Record Updates

• Last Update Posted (Actual): April 25, 2023
• Last Update Submitted That Met QC Criteria: April 24, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Keywords: aTTP; caplacizumab
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Hematologic Diseases; Hemorrhage; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombophilia; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombotic Thrombocytopenic
• Other Study ID Numbers: V01-19052020

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No