Hereditary angioedema (HAE) multi-national survey study
| ISRCTN | ISRCTN85479564 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN85479564 |
| Secondary identifying numbers | ICON 0238-0556 |
- Submission date
- 31/10/2022
- Registration date
- 31/10/2022
- Last edited
- 31/10/2022
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Circulatory System
Plain English Summary
Background and study aims
Takeda (a pharmaceutical company sponsoring this study) is conducting this study to understand the impact of hereditary angioedema (HAE; a disorder characterized by recurrent episodes of severe swelling) on patients and their caregivers and the burden of illness for individuals with HAE and their caregivers.
Who can participate?
Adult patients with any type of HAE, caregivers of adult patients, or caregivers of pediatric patients.
What does the study involve?
HAE Patients - If you agree to participate, you will complete an online survey. Patients will be asked questions about their experiences living with HAE as well as questions regarding socio-demographic background and clinical history. These questions give us some basic information about you (e.g. age, employment status, highest level of education), in addition to obtaining more specific information about your clinical condition (e.g. symptoms, type of diagnosis, current medications, etc.). The survey will take approximately 40 minutes to complete.
Caregivers of Adults - If you agree to participate, you will complete an online survey. Caregivers will be asked questions about their experiences as a caregiver to an adult patient diagnosed with HAE, as well as questions regarding your socio-demographic background (e.g. age, employment status, highest level of education). These questions give us some basic information about you. The survey will take approximately 30 minutes to complete.
Caregivers of Children - If you agree to participate, you will complete an online survey. Caregivers will be asked questions about their child/adolescent’s experiences living with HAE as well as questions about your experiences as a caregiver, as well as questions regarding socio-demographic background. These questions give us some basic information about you (e.g. age, employment status, highest level of education). The survey will take approximately 30 minutes to complete.
What are the possible benefits and risks of participating?
There are no known physical risks to participating in this study. There may be questions that make you uncomfortable while completing the online survey. This is not a test—there are no “right” or “wrong” answers; however, if you are uncomfortable answering a question you can leave it blank. There is little risk of loss of confidentiality since we will not collect any identifying information from you on the survey.
Where is the study run from?
Takeda Pharmaceuticals (Switzerland)
When is the study starting and how long is it expected to run for?
September 2019 to February 2023.
Who is funding the study?
Takeda Pharmaceuticals (Switzerland)
Who is the main contact?
Dr Niall Conlon, conlonn1@tcd.ie
Contact information
Scientific
4131 Parklake Ave.
Suite 600
Raleigh
27612
United States of America
| Phone | +1 240-243-3299 |
|---|---|
| Marie.delaCruz@iconplc.com |
Study information
| Study design | Non-interventional cross-sectional web-based survey |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cross sectional study |
| Study setting(s) | Home |
| Study type | Quality of life |
| Participant information sheet | Not available in web format, please use contact details to request a participant information sheet |
| Scientific title | Burden of hereditary angioedema (HAE) and impact on quality of life: a multi-national survey of patients and caregivers |
| Study hypothesis | Non-interventional cross-sectional web-based survey of 1) adult (≥18 years of age) patients with a self-reported diagnosis of HAE; 2) caregivers of pediatric patients (≤17 years of age) diagnosed with HAE; and 3) caregivers of adult (≥18 years of age) patients diagnosed with HAE in the following countries: Argentina, Brazil, Colombia, Croatia, Denmark, Hungary, Ireland, Norway, Poland, Portugal, Romania, and Sweden. |
| Ethics approval(s) | Approved 05/09/2022, SJH/TUH Research Ethics Committee (Tallaght University Hospital, Tallaght, Dublin 24, D24NR0A; +353 (01) 414 2199; researchethics@tuh.ie), ref: 1067 |
| Condition | Hereditary angioedema quality of life for patients and caregivers |
| Intervention | ICON, on behalf of Takeda, is conducting an online survey assessing the quality of life of patients with hereditary angioedema and their caregivers. The HAE multi-national survey study is an online survey and will target at least 300 adult persons with a self-reported diagnosis of HAE (type I, II, normal functioning C1-INH, and unknown) and up to 250 caregivers of pediatric HAE patients and up to 200 caregivers of adult HAE patients (type I, II, normal functioning C1-INH, and unknown) combined in the following countries: Argentina, Brazil, Colombia, Croatia, Denmark, Hungary, Ireland, Norway, Poland, Portugal, Romania, and Sweden. |
| Intervention type | Other |
| Primary outcome measure | Measured using an online survey at a single time point: 1. Perceived control of angioedema measured with the Angioedema Control Test (AECT) 2. Quality of Life measured with the Angioedema Quality-of-Life Questionnaire (AE-QoL) 3. Work impacts measured with the Work Productivity and Activity Impairment Questionnaire - General Health (WPAI-GH) 4. Overall health measured with the Medical Outcomes Study Short Form 12-Item Questionnaire (SF-12) 5. Anxiety and Depression measured with the Hospital Anxiety and Depression Scale (HADS) 6. De novo questions about HAE attacks, medications used, and life impacts of the condition |
| Secondary outcome measures | There are no secondary outcome measures |
| Overall study start date | 19/09/2019 |
| Overall study end date | 28/02/2023 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 750 |
| Participant inclusion criteria | 1. Self-reported diagnosis of HAE (Types I, II, normal functioning C1-INH, or unknown type) 2. Age 18 years or older 3. Experienced at least 1 episode of angioedema or prodromal symptoms within the last year 4. Has been treated with a prescription medication for an angioedema attack within the last 2 years 5. Able to understand and provide consent 6. Willing to complete an web-based survey 7. Adequate fluency in the target language in which the survey is designed: 7.1. Spanish (Argentina & Colombia) 7.2. Brazilian Portuguese (Brazil) 7.3. Croatian (Croatia) 7.4. Danish (Denmark) 7.5. Hungarian (Hungary) 7.6. English (Ireland) 7.7. Norwegian (Norway) 7.8. Polish (Poland) 7.9. Portuguese (Portugal) 7.10. Romanian (Romania) 7.11. Swedish (Sweden) |
| Participant exclusion criteria | Does not meet inclusion criteria |
| Recruitment start date | 01/08/2022 |
| Recruitment end date | 31/01/2023 |
Locations
Countries of recruitment
- Argentina
- Brazil
- Colombia
- Croatia
- Denmark
- Germany
- Hungary
- Ireland
- Norway
- Poland
- Portugal
- Romania
- Sweden
Study participating centres
Buenos Aires
B1631BUV
Argentina
Santo André/SP
CEP 09060-870
Brazil
San José de Cúcuta
540001
Colombia
Dublin
DO8 NHY1
Ireland
Lisbon
1649-028
Portugal
Budapest
1088
Hungary
Kolding
6000
Denmark
Šibenik
22000
Croatia
Stavanger
4068
Norway
Timișoara
300681
Romania
Warszawa
04-141
Poland
Mörfelden Walldorf
64546
Germany
Falun
791 82
Sweden
Sponsor information
Industry
Thurgauerstrasse 130
Glattpark-Opfikon
Zurich
8152
Switzerland
| Phone | +41 44 555 10 00 |
|---|---|
| maureen.watt@takeda.com | |
| Website | takeda.com |
"ROR" |
https://ror.org/002ysmy84 |
Funders
Funder type
Industry
Government organisation / For-profit companies (industry)
- Alternative name(s)
- Takeda, Takeda Pharmaceutical Company Limited, Chobei Takeda & Co., Ltd., Takeda Pharmaceutical Industries, Ltd., Takeda Chemical Industries, Ltd., 武田薬品工業株式会社, TPC
- Location
- Japan
Results and Publications
| Intention to publish date | 31/03/2024 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not expected to be made available |
| Publication and dissemination plan | Planned publication in a high-impact peer-reviewed journal |
| IPD sharing plan | The datasets generated during and/or analysed during the current study are not expected to be made available due to the data being the proprietary information of Takeda Pharmaceuticals. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Protocol file | version 2.0 | 25/03/2022 | 31/10/2022 | No | No |
Additional files
Editorial Notes
31/10/2022: Trial's existence confirmed by Tallaght University Hospital.
