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Hereditary Angioedema Kininogen Assay

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ClinicalTrials.gov Identifier: NCT04091113
Recruitment Status : Recruiting
First Posted : September 16, 2019
Last Update Posted : September 16, 2019
Sponsor:
Information provided by (Responsible Party):
Centogene AG Rostock

Brief Summary:

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.


Condition or disease Intervention/treatment
Hereditary Angioedema Diagnostic Test: Withdraw of blood

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Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Hereditary Angioedema Kininogen Assay. A Multicenter Epidemiological Observational Study.
Actual Study Start Date : September 1, 2019
Estimated Primary Completion Date : September 1, 2020
Estimated Study Completion Date : September 1, 2021

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Participants without lanadelumab treatment Diagnostic Test: Withdraw of blood

A blood sample will be taken on seven subsequent visits with 3 month intervals in the first 12 months (visit 1-5) and 6 month intervals thereafter (visit 6-7) and the cHMWK biomarker and other metabolite levels will be biochemically assessed.

DBS based biochemical measurements of C4 complement and C1-INH levels will be tested for HAE type 1 and type 2 via liquid chromatography multiple reaction-monitoring mass spectrometry, and the cHMWK biomarker and other metabolite levels will be biochemically assessed.

Additionally, every patient will be asked to pay attention for occurring HAE attacks: at the beginning, 3 hours, 6 hours, 12 hours and 24 hours after the beginning of the attack.


Participants on lanadelumab treatment Diagnostic Test: Withdraw of blood

A blood sample will be taken on seven subsequent visits with 3 month intervals in the first 12 months (visit 1-5) and 6 month intervals thereafter (visit 6-7) and the cHMWK biomarker and other metabolite levels will be biochemically assessed.

DBS based biochemical measurements of C4 complement and C1-INH levels will be tested for HAE type 1 and type 2 via liquid chromatography multiple reaction-monitoring mass spectrometry, and the cHMWK biomarker and other metabolite levels will be biochemically assessed.

Additionally, every patient will be asked to pay attention for occurring HAE attacks: at the beginning, 3 hours, 6 hours, 12 hours and 24 hours after the beginning of the attack.





Primary Outcome Measures :
  1. To explore 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. [ Time Frame: 30 months ]

Secondary Outcome Measures :
  1. To study 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. [ Time Frame: 30 months ]

Other Outcome Measures:
  1. To study other metabolites as a 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. [ Time Frame: 30 months ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Hereditary Angioedema type 1/2 patients
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04091113


Locations
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Germany
Centogene AG Recruiting
Rostock, Germany, 18055
Contact: Volha Skrahina, Dr    +49-381-80113-594    volha.skrahina@centogene.com   
Sponsors and Collaborators
Centogene AG Rostock

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Responsible Party: Centogene AG Rostock
ClinicalTrials.gov Identifier: NCT04091113     History of Changes
Other Study ID Numbers: HAEKA 01-2019
First Posted: September 16, 2019    Key Record Dates
Last Update Posted: September 16, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Centogene AG Rostock:
Hereditary Angioedema
Cleaved high-molecular weight kininogen
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Bradykinin
Kininogens
Cysteine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Vasodilator Agents