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

NCT07293364 | Not Yet Recruiting

• 1 other identifier: TAK-743-4028
• Study Type: interventional
• Target: 514
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

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.

  Up to 12 months

Secondary Outcomes (8)

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

  Up to 12 months

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

  Up to 12 months

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

  Up to 12 months

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

  Up to 12 months

• Likelihood Ratios of Technochrom Compared to C4 Assay and C1-INH Antigenic Testing

  Up to 12 months

Study Arms (1)

Suspected HAE Participants

EXPERIMENTAL

Participants with suspected HAE having a high suspicion of bradykinin-mediated HAE (no urticaria or itching, lasting between 1 and 5 days, non-responsive to antihistamine and corticosteroid treatments) and/or family members of known HAE participants will be enrolled in the study.

Diagnostic Test: Technochrom C1-INH Kit

Interventions

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).

Suspected HAE Participants

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead
• CRO Axelys Santé DZ: collaborator

Study Sites (1)

EPH de Rouiba (Etablissement Public Hospitalier)

Algiers, 16017, Algeria

Location

Related Links

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MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 8, 2025
• First Posted: December 19, 2025
• Study Start (Estimated): May 15, 2026
• Primary Completion (Estimated): January 7, 2027
• Study Completion (Estimated): January 7, 2027
• Last Updated: April 1, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

AL (1)