Hereditary Angioedema Kininogen Assay

Terminated

• Conditions: Hereditary Angioedema

• Registration Number: NCT04091113
• Lead Sponsor: CENTOGENE GmbH Rostock

Brief Summary

A multicenter epidemiological observational study aiming to explore the cleaved high-molecular weight kininogen (cHMWK) including identification and characterization of other metabolite/biomarkers in HAE type 1/2 patients

Detailed Description

Hereditary angioedema (HAE) is a rare autosomal dominant genetic disorder characterized by recurrent tissue angioedema episodes, mainly caused by mutations in the SERPING1 gene that encodes C1 inhibitor (C1-INH), a protease involved in limiting bradykinin production. Low levels of C1-INH (HAE type 1) or dysfunctional C1-INH (HAE type 2) lead to bradykinin accumulation, resulting in capillary leakage and tissue swelling.

High Molecular Weight Kininogen (HMWK) proteolysis, by active plasma kallikrein, results in bradykinin and cHMWK generation.

The goal of this study is to explore the cHMWK concentrations in HAE type 1/2 patients, as a biomarker for this disease.

The HAEKA study is performed in collaboration with Shire. Shire is a wholly owned subsidiary of Takeda Pharmaceutical Company Limited.

Study Timeline

• Study Start: 2019-09-01
• Study First Submitted: 2019-09-06
• Study First Submitted QC: 2019-09-13
• Study First Posted: 2019-09-16
• Primary Completion: 2021-09-12
• Study Completion: 2021-12-31
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-09
• Last Update Posted: 2022-03-10

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 59

Inclusion Criteria

• Informed consent is obtained from the participant
• The patient with diagnosis of Hereditary Angioedema (HAE) type 1/2 based on international guidelines
• The patient experienced ≥4 HAE attacks within last 12 month before enrolment in the study
• The participant is older than 18 years old

Exclusion Criteria

• Inability to provide informed consent
• The patient is not diagnosed with Hereditary Angioedema (HAE) type 1/2
• The patient experienced ˂ 4 HAE attacks within last 12 month before enrolment in the study
• The participant is younger than 18 years old

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Exploring the cleaved high-molecular weight kininogen (cHMWK) as a biomarker in HAE type 1/2 patients, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.	30 months	Samples from HAE patients without lanadelumab treatment versus patients on lanadelumab treatment will be analyzed for cHMWK via Liquid Chromatography Multiple Reaction-monitoring Mass Spectrometry (LC/MRM-MS).

Secondary Outcome Measures

Name	Time	Method
Studying cHMWK as a biomarker in HAE type 1/2 patients with edema attack, as well as to study the differences between HAE type 1/2 patients without lanadelumab treatment versus patients on lanadelumab treatment.	30 months	Samples from HAE patients without lanadelumab treatment versus patients on lanadelumab treatment collected before/during/after angioedema attacks will be analyzed for cHMWK via Liquid Chromatography Multiple Reaction-monitoring Mass Spectrometry (LC/MRM-MS).

Trial Locations

Locations (7)

Medizinische Hochschule Hannover; 🇩🇪 Hannover, Germany

Universitäts-Hautklinik Leipzig; 🇩🇪 Leipzig, Germany

Hämophilie-Zentrum Rhein Main GmbH; 🇩🇪 Mörfelden-Walldorf, Germany

Klinik für Hals-Nasen-Ohrenheilkunde, Universitätsklinikum Düsseldorf,; 🇩🇪 Düsseldorf, Germany

Klinikum der Johann-Wolfgang-Goethe-Universität; 🇩🇪 Frankfurt, Germany

Charité - Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany

Universitätsklinikum Ulm; 🇩🇪 Ulm, Germany