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

N/ANot Yet RecruitingNCT07218393

Takeda100 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

100

Conditions

Hereditary Angioedema (HAE)

Interventions

No intervention

This is a non-interventional study.

Eligibility

Sex: ALLMin age: 1 Year

Medical Language ↔ Plain English

Inclusion Criteria: Retrospective Phase: 1. 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). 2. The participant was diagnosed and/or treated from January 2015 to August 2025. Inclusion Criteria: Prospective Phase: 1. The participant has a physician-confirmed HAE-C1INH type1 or type2 diagnosis (based on clinical history and/or laboratory diagnosis). 2. The participant signed an informed consent or assent. 3. The participant should have had at least one visit to the treating physician/investigator during enrollment and the follow-up period. 4. The participant was not enrolled in the study's retrospective phase. Exclusion Criteria: (For both retrospective and prospective phases): 1. 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. 2. HAE participants with normal C1-INH (HAE-nC1INH). 3. Participant deemed unsuitable for participation for any reason, based on the investigator's clinical judgment.

Locations (7)

Mansoura university hospital

Al Mansurah, Egypt

Alexandria university_Clinical Research Center

Alexandria, Egypt

Ain shams university hospital_Pediatrics

Cairo, Egypt

Ain shams university hospital

Cairo, Egypt

Cairo university hospital_Pediatrics

Giza, Egypt

Cairo university hospital

Giza, Egypt

Zagazig university hospital

Zagazig, Egypt

Outcomes

Primary Outcomes

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

Time frame: At Day 1

Percentage of Participants With HAE-C1INH Type1 or Type2

Time frame: At Day 1

Number and Type of First Symptom at Initial Manifestation

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With First Symptom at Initial Manifestation

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Duration of First Symptom at Initial Manifestation

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

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

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

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

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Duration of Confirmed Diagnosis of HAE-C1INH Type1 or Type2

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Secondary Outcomes

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

Time frame: 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

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

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

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Known Triggers at Diagnosis

Triggers include stress, trauma, infection, contraception, menstruation, medication, food, burns, surgical procedures, and others.

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With History of Upper Airway Edema

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With History of Abdominal Surgery due to HAE Misdiagnosis

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With HAE-related Death

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Response to Standard Allergy Medications

Standard allergy medications (e.g. antihistamines, glucocorticoids, and epinephrine) that will be reported based on the symptom improvement, time to relief, reduction in symptom severity, and need for additional interventions.

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Presence of Comorbidities

Central Contacts

Takeda Contact

CONTACT

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

Data from ClinicalTrials.gov

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Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Average Number of HAE Attacks per Month

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Average Number of Attacks Requiring Acute Treatment per Month

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Average Number of Attacks Requiring Emergency Care or Hospitalization per Month

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Severity of HAE Attack per Each Attack

Number of participants with severity of HAE attack (mild, moderate, severe as reported or documented) per each attack.

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Time of HAE Attack(s) at Each Onset During Follow-Up Visits or Date of Last Recorded HAE Attack

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Duration of Symptoms for Each HAE Attack Onset During Subsequent Follow-Up Visits

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of HAE Attacks per Month

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants Categorized by HAE Attack Characteristics

HAE attack characteristics will include most common attack site(s) (e.g., abdominal, cutaneous, laryngeal), number of recurrent abdominal attacks, site of attack per each attack onset for each subsequent follow-up visit, and symptoms experienced per attack (e.g., pain, swelling, nausea).

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Prodromal Symptom

Prodromal symptoms before attacks such as general discomfort, tiredness, irritability, skin symptoms, rash, and paresthesia per each attack onset for each subsequent follow-up visit will be reported.

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With any Known Triggers Identified by the Participant as Related to the Onset of the Attack per Each Attack Onset for Each Subsequent Follow-up Visits

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With On-demand Treatment Medication for Each HAE Attack

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number and Type of Hospitalizations due to HAE Attacks

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Life-threatening Attacks

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants (Family Members) Diagnosed With HAE-C1INH Type1 or Type2

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Degree of Relation (Parent, Sibling, Child) to Diagnosed Family Members

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Presence of Laryngeal or Life-threatening Attacks in Relatives

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Rate of Testing and of Diagnosis Among Tested Family Members

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With History of HAE-related Deaths in the Family

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participant's Relatives who Underwent Clinical and/or Lab Assessment for HAE diagnosis

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Clinical Assessment for HAE Diagnosis With Symptoms Description for the Patient, their Descendants, or Relatives

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Laboratory Testing for the Participant, Their Descendants, or Relatives

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Follow-up Visits Routinely Performed (Average per Year) During the Retrospective and Prospective Phase

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants Who Missed Days at Work or School and Social Life due to HAE Attack

Time frame: Up to 5 months retrospectively, up to 12 months prospectively

Number of Participants With Treatment Discontinuation

Participants who discontinued the treatment due to reasons such as lack of efficacy, safety concerns, compliance issues, or others.

Time frame: Up to 5 months retrospectively, up to 12 months prospectively