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

Exploring Symptom Profile and Treatment Patterns in Korean HAE Patients Using Digital Patient Diary: A Retrospective Descriptive Study (SPEAKUP Study)

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.

Study Overview

• Status: Completed
• Conditions: Hereditary Angioedema (HAE)
• Intervention / Treatment: Other: No Intervention

• Study Type: Observational
• Enrollment (Actual): 19

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul,, Korea, Republic of, 03080
    • Seoul National University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

Inclusion Criteria:

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

Exclusion Criteria:

- 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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

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; This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Categorized Based on Demographic Characteristics	Number of participants will be reported by their demographic characteristics (age, sex, disease duration and baseline danazol use).	At Baseline
Icatibant-treated HAE Attack Rate	The icatibant-treated HAE attack rate will be calculated as a PPPM rate. The PPPM rate will be calculated by dividing the total number of icatibant-treated attacks reported per each HAE participant during their observation periods by their duration of observation periods in months.	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	The number of icatibant administration in a single attack will be reported as either none, once, or twice.	Up to 11 months

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): October 16, 2024
• Primary Completion (Actual): December 18, 2024
• Study Completion (Actual): December 18, 2024

Study Registration Dates

• First Submitted: September 4, 2024
• First Submitted That Met QC Criteria: September 4, 2024
• First Posted (Actual): September 19, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 6, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immunologic Deficiency Syndromes; Skin Diseases; Urticaria; Skin Diseases, Vascular; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: TAK-667-4004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No