A Study to Explore Hereditary Angioedema (HAE) Symptoms and Treatment Patterns in Korean People

NCT06587464 | Completed

• 1 other identifier: TAK-667-4004
• Study Type: observational
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

This study intends to gather information on HAE attack symptoms, how often the attacks occur, and the number of times treatment is required to manage the attacks in Korean people over a period of up to 11 months. The main aims of this study are the following:

• To describe the patterns of HAE attacks, including how often they occur, where they happen in the body, how severe they are, the time between attacks, and any warning signs that appear before an attack.
• To assess how HAE is treated, particularly looking at how on-demand treatments are used during HAE attacks.
• To learn about the people who suffer from HAE attacks (e.g. age, gender, for how long they have been suffering from HAE, and if they use danazol to manage HAE). In this study, already existing data will be collected and reviewed from a mobile application called "MyHAE Story". The study will only review data collected as part of the clinical routine practice. The study will not impact the standard medical care and treatment of participants.

Conditions

Hereditary Angioedema (HAE)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (5)

• HAE Attack Rate

  The HAE attack rate will be calculated as a per participant per month (PPPM) rate. The PPPM rate will be calculated by dividing the total number of attacks reported per each HAE participant during their observation periods by their duration of observation periods in months.

  Up to 11 months

• Percentage of Participants With HAE Attack Based on Location

  Number of participants with HAE attack based on anatomical location (face, throat, arm, hand, abdomen, genital, leg, foot) will be evaluated.

  Up to 11 months

• Percentage of Participants With HAE Attack Based on Attack Severity

  The HAE attack based on severity will be determined using following definitions: mild (temporarily feels somewhat uncomfortable, swelling is alleviated within 48 hours), moderate (slight disruption in daily life), severe (symptoms that make it impossible to carry out daily life).

  Up to 11 months

• Average Time Interval Between HAE Attacks in Days

  Up to 11 months

• Percentage of Participants With Prodromal Symptom

  The presence of prodromal symptoms among participants, a response of "No" indicates the absence of any symptoms and a response of "Yes" suggests the presence of one or more symptoms from the following list will be reported: tight and stinging feeling in the skin, anxiety, nausea, sudden emotional changes, non-pruritic rash, severe fatigue, or other symptoms.

  Up to 11 months

Secondary Outcomes (4)

• Icatibant-treated HAE Attack Rate

  Up to 11 months

• Percentage of Icatibant-Treated HAE Attacks Among All HAE Attacks

  Up to 11 months

• Number of HAE Attacks Categorized Based on the Icatibant Administration in a Single Attack

  Up to 11 months

• Number of Participants Categorized Based on Demographic Characteristics

  At Baseline

Study Arms (1)

HAE Participants (Type I or II)

Participants with HAE type I or II who are prescribed icatibant, enrolled in the patient support program (PSP), and registered in the "MyHAE Story" mobile app will be included and their data will be collected retrospectively from the first documented date of app until the date of the last recorded attack, participant withdrawal, loss to follow-up, or the end of the study, whichever occurs first.

Other: No Intervention

Interventions

No Intervention OTHER

This is a non-interventional study.

HAE Participants (Type I or II)

Eligibility Criteria

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

Study Population

HAE type I or II participants who are prescribed icatibant, enrolled in the OnHeart PSP, and registered in and actively utilized mobile app application for at least 1 month will be included in this study.

You may qualify if:

• Participants who are participants in the OnHeart PSP program.
• Participants who have registered in "MyHAE Story" mobile application.

You may not qualify if:

• \- Participants who neither have made input to at least one attack nor have run the app by clicking 'myHAE story' tab within the Kakaotalk chat message at least once after app registration.

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Related Links

• To obtain more information about this study, click this link

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

Study Design

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

Study Record Dates

• First Submitted: September 4, 2024
• First Posted: September 19, 2024
• Study Start: October 16, 2024
• Primary Completion: December 18, 2024
• Study Completion: December 18, 2024
• Last Updated: February 10, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, 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

KR (1)