A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt

NCT07218393 | Not Yet Recruiting

• 1 other identifier: TAK-743-4031
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 7

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. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency); HAE with C1-INH deficiency can be divided into Type1, with low levels of C1-INH, and Type 2, in which the protein is there, but does not work properly. This study will look at people with HAE Type1 or Type2. The main aim of this study is to describe the diagnosis and management of people with HAE Type1 or HAE Type2 in Egypt. Other aims are to learn more about people with HAE Type1 or Type2, including, but not limited to, other conditions they may have, family history, impact of HAE on daily life, if treatment is stopped and the reasons. During the study, information from already existing data in a participant's medical record will be reviewed and new data will be collected during routine visits of a participant to the study clinic.

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (17)

• Age of Participants at Diagnosis (in years) of HAE-C1INH Type1 or Type2

  At Day 1

• Percentage of Participants With HAE-C1INH Type1 or Type2

  At Day 1

• Number and Type of First Symptom at Initial Manifestation

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants With First Symptom at Initial Manifestation

  Up to 5 months retrospectively, up to 12 months prospectively

• Duration of First Symptom at Initial Manifestation

  Up to 5 months retrospectively, up to 12 months prospectively

• Number and Type of Confirmed Diagnosis of HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants With Confirmed Diagnosis of HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Duration of Confirmed Diagnosis of HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants with Recorded Diagnostic Test Results for C1-INH Antigenic Level, Serum Complement Component 4 (C4) Level, and C1-INH Functional Level

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants Receiving On-demand (OD), Short-term prophylaxis (STP), and Multiple Long-Term Prophylaxis (LTP) Therapies

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants who Received Prescriptions for Therapeutic Management With HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Treatment Options Used for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Routes of Administration Used for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Dose Regimens Used for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants Switching Treatment for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants With Frequency of Treatment Changes for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants With Reason for Switching Treatment for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2

  Up to 5 months retrospectively, up to 12 months prospectively

Secondary Outcomes (33)

• Number of Participants With Clinical Symptoms of HAE-C1INH Type1 or Type2 at Last Clinical Appointment

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants With Clinical Symptom Types of HAE-C1INH Type1 or Type2 at Last Clinical Appointment

  Up to 5 months retrospectively, up to 12 months prospectively

• Duration of Clinical Symptoms of HAE-C1INH Type1 or Type2 at Last Clinical Appointment

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants With Known Triggers at Diagnosis

  Up to 5 months retrospectively, up to 12 months prospectively

• Number of Participants With History of Upper Airway Edema

  Up to 5 months retrospectively, up to 12 months prospectively

Study Arms (2)

Retrospective Phase

All participants who were diagnosed with HAE-C1INH type1 or type2 from January 2015 to August 2025; retrospective clinical data will be extracted for 5 months exclusively from patients' medical charts, and no direct patient interaction will occur.

Other: No intervention

Prospective Phase

All participants with any newly diagnosed or admitted or follow-up HAE-C1INH type1 or type2; prospective clinical data will be actively collected over 12 months through routine visits and electronic data capture (EDC) entries.

Other: No intervention

Interventions

No intervention OTHER

This is a non-interventional study.

Prospective Phase; Retrospective Phase

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult and pediatric participants with HAE-C1INH type1 or type2, treated within a routine clinical setting in Egypt.

You may qualify if:

• The participant has a confirmed HAE-C1INH type1 or type2 diagnosis in medical records (based on clinical history and/or laboratory diagnosis in medical records).
• The participant was diagnosed and/or treated from January 2015 to August 2025.
• The participant has a physician-confirmed HAE-C1INH type1 or type2 diagnosis (based on clinical history and/or laboratory diagnosis).
• The participant signed an informed consent or assent.
• The participant should have had at least one visit to the treating physician/investigator during enrollment and the follow-up period.
• The participant was not enrolled in the study's retrospective phase.

You may not qualify if:

• Participant with AAE-C1INH (acquired angioedema), drug-induced angioedema (AE-DI) (example angiotensin-converting enzyme inhibitors-I \[ACE-I\] angioedema), allergic mediated angioedema, inflammatory angioedema, or idiopathic angioedema.
• HAE participants with normal C1-INH (HAE-nC1INH).
• Participant deemed unsuitable for participation for any reason, based on the investigator's clinical judgment.

Sponsors & Collaborators

• Takeda: lead

Study Sites (7)

Mansoura university hospital

Al Mansurah, 35511, Egypt

Location

Alexandria university_Clinical Research Center

Alexandria, 21526, Egypt

Location

Ain shams university hospital_Pediatrics

Cairo, 11535, Egypt

Location

Ain shams university hospital

Cairo, 11535, Egypt

Location

Cairo university hospital_Pediatrics

Giza, 11632, Egypt

Location

Cairo university hospital

Giza, 11632, Egypt

Location

Zagazig university hospital

Zagazig, 44511, Egypt

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: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 16, 2025
• First Posted: October 20, 2025
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: May 1, 2026

Record last verified: 2026-04

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

EG (7)