Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP

Recruiting

• Conditions: Acquired Thrombotic Thrombocytopenic Purpura
• Interventions: Drug: Cablivi®

• Registration Number: NCT04985318
• Lead Sponsor: University of Cologne

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-03-25
• Study First Submitted: 2021-07-21
• Study First Submitted QC: 2021-07-21
• Study First Posted: 2021-08-02
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-04
• Last Update Posted: 2024-12-09
• Primary Completion: 2034-12-12
• Study Completion: 2034-12-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 350

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Registergroup	Cablivi®	Patients with acquired Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®)

Primary Outcome Measures

Name	Time	Method
Treatment of aTTP with caplacizumab	Enrollment	Description of the prescription rationale and practice of caplacizumab in the treatment of aTTP

Secondary Outcome Measures

Name	Time	Method
Stabilization of thrombocyte	Enrollment	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
Normalization of haptoglobin	Enrollment	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
Effect of caplacizumab on adjunct treatments with PEX	Enrollment	Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of therapeutic plasma exchange therapy (PEX)
Normalization of LDH	Enrollment	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
Risk factors for adverse outcomes	Enrollment	Identification of risk factors for adverse outcomes
Risk factors for complications	Enrollment	Identification of risk factors for complications
Risk factors for persistent autoimmune activity	Enrollment	Identification of risk factors for persistent autoimmune activity
Parameters for therapy guidance	Enrollment	Identification of parameters that guide duration and regimen of caplacizumab treatment
Effect of caplacizumab on adjunct treatments	Enrollment	Descriptions of the effect of caplacizumab on dose, duration and number and severity of adverse events of glucocorticoids

Trial Locations

Locations (1)

University Hospital of Cologne; 🇩🇪 Cologne, Germany