Health Trials

A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema

ConditionHereditary Angioedema (HAE)
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age12+
SponsorTakeda

About this trial

Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1-inhibitor (C1INH): HAE with normal C1INH levels and function (HAE-nC1INH) and HAE with deficiency in C1INH levels (HAE-C1INH-Type1) or dysfunction (HAE-C1INH-Type2). This study will focus on the practical use and accuracy of measuring the C1INH function alone to diagnose HAE-C1INH-Type1 and HAE-C1INH-Type2 compared to the tests used in normal clinical practice in Algeria.

The main goal of the study is to see how well a test focusing on the C1INH function alone works to diagnose HAE-C1INH as compared to the tests used in normal clinical practice (standard of care or SoC) in Algeria. Another aim is to determine a reference value (helps in determining the accuracy) of the C1INH function test. This study will also help to find out how many people who are thought to have HAE or who have family members with HAE actually get diagnosed and to gather participants' health background information, such as their age when they were diagnosed, what signs and symptoms they had, how long it took to get diagnosed, and how they were sent to the doctors or specialists who treated them.

During the study all participants will undergo two different methods of HAE testing: the test focusing on the C1INH function alone and the SoC tests. Test results will be confirmed via a second test run for newly index cases or for discordant results, but participants with a test result of "no HAE" and positive cases recruited through family screening will not undergo a second confirmatory test. In case of discordant test results in the second round, participants will undergo a third confirmatory test round. Participants can visit the clinic up to three times during the study. No further follow up is planned for participants, even for those who are diagnosed with HAE.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Adult and pediatric participants of both sexes (children over the age of 12 years).

Participants providing a signed informed consent form (ICF), or parental consent for minors.

Participants with a high suspicion of bradykinin-mediated HAE, referred to the center. This includes recurrent episodes of nonpitting angioedema without urticaria or itching, lasting between 1 and 5 days, and non-responsive to antihistamine and corticosteroid treatments.

Family members (from 1st to 4th degree relatives) of known HAE participants.

Disqualifiers

Confirmed diagnosis of HAE-C1INH-Type1 or HAE-C1INH-Type2.

Angioedema with urticaria or itching (suggesting histaminergic etiology).

Angioedema episodes lasting less than (<)1 day or greater than (>)5 days (not consistent with bradykinin-mediated HAE).

Any condition deemed unsuitable by the investigator that may interfere with study procedures or data integrity.

Trial design

Design model

Single group

Treatments tested in this trial

  • Technochrom C1-INH Kit

    Diagnostic test

    HAE biological diagnostic test that uses functional C1-INH technique by colorimetric method versus the reference test defined as the SoC (C4 and C1-INH antigenic level measurement and functional C1-INH assay).

Treatment groups

514 Participants
are divided into 1 treatment group
Group A: Suspected HAE ParticipantsExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Sensitivity and Specificity of Technochrom Compared to Standard of Care (SoC) for HAE Diagnosis

The diagnostic accuracy of isolated functional C1-inhibitor (C1-INH) measurement using Technochrom C1-INH kits, compared to the SoC (McNemar's test) as confirmatory testing for HAE diagnosis will be reported.

Time frame
Up to 12 months

Secondary outcomes

1

Laboratory-specific Cut-off Values for Functional C1-INH Measurement Using Technochrom

Laboratory-specific cut-off values for functional C1-INH measurement in the Algerian population using the Technochrom C1-INH kit and assess their impact on diagnostic accuracy will be reported.

Time frame
Up to 12 months
2

Sensitivity and Specificity of Technochrom Compared to Isolated Complement Component Test (C4 assay) for HAE Detection

Sensitivity and specificity of Technochrom compared to isolated C4 assay will be evaluated.

Time frame
Up to 12 months
3

Likelihood Ratios of Technochrom Compared to Isolated C4 Assay for HAE Detection

Positive and negative likelihood ratios of the Technochrom compared to isolated measurement of antigenic C4 assay will be evaluated.

Time frame
Up to 12 months
4

Sensitivity and Specificity of Technochrom Compared to C4 Assay and C1-INH Antigenic Testing

Sensitivity and specificity of Technochrom compared to C4 assay and C1-INH antigenic testing will be evaluated.

Time frame
Up to 12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Takeda

Lead sponsor

CRO Axelys Santé DZ

Collaborator

Source ClinicalTrials.gov
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