Hereditary Angioedema Kininogen Assay

Hereditary Angioedema Kininogen Assay: A Multicenter, Epidemiological, Observational Study.

Status

Terminated

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04091113

Acronym

HAEKA

Enrollment

Registered

2019-09-16

Start date

2019-09-01

Completion date

2021-12-31

Last updated

2022-03-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Angioedema

Keywords

Hereditary Angioedema, Cleaved high-molecular weight kininogen, Hereditary Angioedema Type I, Hereditary Angioedema Type II

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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).

Other

Measure	Time frame	Description
Studying other metabolites as potential biomakers 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	All samples will be analyzed for potential biomarker candidates via Liquid Chromatography Multiple Reaction-monitoring Mass Spectrometry (LC/MRM-MS).

Countries

Germany

Outcome results