Exploring the hidden burden of living with Glanzmann thrombasthenia

ISRCTN	ISRCTN16256452
DOI	https://doi.org/10.1186/ISRCTN16256452
Integrated Research Application System (IRAS)	368693
Sponsor	Equipath Analytics Limited
Funder	Hemab ApS

Submission date: 27/01/2026
Registration date: 28/01/2026
Last edited: 28/01/2026

Recruitment status: Not yet recruiting
Overall study status: Ongoing
Condition category: Genetic Diseases

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Background and study aims
Glanzmann thrombasthenia (GT) is a very rare inherited bleeding disorder. Doctors know how to treat bleeding episodes, but much less is known about how living with GT affects people’s everyday lives. This includes emotional wellbeing, mental health, relationships, education, work, and the impact on families and caregivers.
The aim of this study is to better understand the real, lived experiences of adults with GT and of people who care for someone with GT. By listening directly to their stories, the study hopes to uncover the “hidden burden” of the condition that is often not captured in medical records. The findings will be used to improve understanding, support, communication, and future research for people affected by GT.

Who can participate?
People can take part if they are aged 16 or over and have a self reported diagnosis of Glanzmann thrombasthenia. Self reported diagnosis is acceptable because GT is very rare and the study focuses on personal experiences rather than medical tests.
Caregivers can also take part. This includes parents, partners, siblings, or other family members who support someone with GT.
Young people under the age of 16 may take part if a parent or legal guardian provides consent and the young person agrees to take part where appropriate.

What does the study involve? (for participants)
Participants will be invited to take part in a single interview lasting about 45 to 60 minutes. The interview will take place by video call or telephone, depending on what the participant prefers.
During the interview, participants will be asked to talk about their experiences of living with GT or caring for someone with GT. Topics may include daily life, emotional wellbeing, school or work, relationships, experiences with healthcare, and challenges they face.
Taking part is voluntary. Participants can skip any questions they do not want to answer and can stop the interview at any time. With permission, the interview may be audio recorded to help the research team accurately capture what is said.
As a thank you for their time, participants will receive a £50 digital voucher.

What are the possible benefits and risks of participating?
There are no direct medical benefits from taking part. However, many people may find it helpful to share their experiences and know that their views could help improve understanding and support for others with GT in the future.
There are no physical risks. Some people may find it emotionally upsetting to talk about difficult experiences. The interviewers are trained to handle sensitive topics and can pause or stop the interview if needed. Participants will also be given information about support resources if they feel distressed.

Where is the study run from?
The study is sponsored and run by EquiPath Analytics Limited, based in the United Kingdom. Although the study is run from the UK, people from many countries around the world are able to take part because interviews are conducted remotely.

When is the study starting and how long is it expected to run for?
The study is expected to start recruiting participants in February 2026. Recruitment is planned to continue until December 2026. The overall study is expected to run until February 2027.

Who is funding the study?
The study is funded by Hemab ApS. The funder has no involvement in how the study is designed, how interviews are carried out, how data is analysed, or how results are reported.

Who is the main contact?
The main contacts for the study are the principal investigators, Amy Owen-Wyard and Stacey McGeown, at EquiPath Analytics Limited in the United Kingdom. They can be contacted by email at <amy@epalimited.com> or <stacey@epalimited.com>, or by telephone using the contact details provided in the study information.

Contact information

Mrs Amy Owen-Wyard
Public, Principal investigator, Scientific

EquiPath Analytics Limited
8 FALMOUTH
Worcester
WR4 0TE
United Kingdom

ORCID ID	0009-0008-7468-4042
Phone	+44 7510310324
Email	amy@epalimited.com

Mrs Stacey McGeown
Public, Scientific

8 Falmouth
Worcester
WR4 0TE
United Kingdom

Phone	+44 7850910227
Email	stacey@epalimited.com

Study information

Primary study design	Observational
Observational study design	Cross sectional study
Participant information sheet	48915 Participant Information Sheet interview.pdf
Scientific title	Exploring the hidden burden of living with Glanzmann thrombasthenia: a qualitative study
Study objectives	Primary Objective: 1. To explore the lived emotional, psychological, social, and practical experiences of adults with GT. Secondary Objectives: 1. To describe the demographic and sociodemographic profile of PwGT, carriers, and caregivers, including age range, gender identity, geographic region, and care roles. 2. To understand caregiver experiences, including emotional labor, care management, stress, and coping strategies. 3. To identify unmet support needs, barriers to access, and structural or systemic obstacles affecting quality of life. 4. To generate evidence to inform clinical communication, resource development, and future patient-centered interventions. This study is designed to generate rich, real world accounts of the lived experience of GT that cannot be captured through quantitative methods. Findings will be used to inform patient support resources, clinical communication, and future research priorities.
Ethics approval(s)	Not yet submitted
Health condition(s) or problem(s) studied	Glanzmann thrombasthenia
Intervention	This is a non interventional, observational, qualitative study. The study is funded by Hemab ApS, who have no involvement in study design, data access, analysis, or reporting. The Sponsor (EquiPath Analytics Limited) retains full independence and oversight. This study will use a qualitative, exploratory design appropriate for capturing nuanced experiences. s allow flexibility while ensuring coverage of core areas of interest. The study will follow an interpretivist paradigm, acknowledging that participants’ experiences are shaped by social context. Interviews will be analyzed using Braun & Clarke’s six-phase thematic analysis method, allowing identification of patterns both within and across participant groups. No quantitative data will be collected. No experimental treatments or changes to standard clinical management will occur. Given the rarity of GT and the global geographical distribution of individuals, self reported diagnosis is accepted. This is consistent with qualitative rare disease research, where clinical confirmation is often impractical and unnecessary for experiential exploration.
Intervention type	Other
Primary outcome measure(s)	Explore the experience of living with GT measured using semi-structured interviews, analyzed using Braun & Clarke’s six-phase thematic analysis method at a single time point
Key secondary outcome measure(s)
Completion date	24/02/2027

Eligibility

Participant type(s)
Age group	Mixed
Lower age limit	16 Years
Upper age limit	120 Years
Sex	All
Target sample size at registration	45
Key inclusion criteria	Participants will be eligible if they meet one or more of the following: 1. Individuals with a self-reported diagnosis of Glanzmann’s Thrombasthenia (GT). (Given the rarity of GT and the exploratory qualitative nature of the study, self-reported diagnosis is considered appropriate and proportionate) 2. Primary or secondary caregivers of an individual with GT AND: 3. Can read and understand the participant information sheet 4. Provide informed consent electronically prior to beginning the survey 5. Are aged ≥16 years, or have a caregiver completing the form on their behalf if under 16 6. For participants aged 16–17 years, informed consent will be obtained directly, as they are considered competent to consent to low risk qualitative research. For participants under 16, a parent or legal guardian will provide consent, and the young person will provide assent where appropriate
Key exclusion criteria	1. Decline or withdraw consent at any point during the study 2. Inability to comply with study procedures 3. Behavioural concerns that may compromise participant safety or data integrity, as assessed by the research team 4. Individuals unable to provide informed consent
Date of first enrolment	24/02/2026
Date of final enrolment	21/12/2026

Locations

Countries of recruitment

United Kingdom
Afghanistan
Åland Islands
Albania
Algeria
American Samoa
Andorra
Angola
Anguilla
Antarctica
Antigua and Barbuda
Argentina
Armenia
Aruba
Australia
Austria
Azerbaijan
Bahamas
Bahrain
Bangladesh
Barbados
Belarus
Belgium
Belize
Benin
Bermuda
Bhutan
Bolivia
Bonaire Saint Eustatius and Saba
Bosnia and Herzegovina
Botswana
Bouvet Island
Brazil
British Indian Ocean Territory
Brunei Darussalam
Bulgaria
Burkina Faso
Burundi
Cabo Verde
Cambodia
Cameroon
Canada
Cayman Islands
Central African Republic
Chad
Chile
China
Christmas Island
Cocos (Keeling) Islands
Colombia
Comoros
Congo
Congo, Democratic Republic
Cook Islands
Costa Rica
Croatia
Cuba
Curaçao
Cyprus
Czech Republic
Côte d'Ivoire
Denmark
Djibouti
Dominica
Dominican Republic
Ecuador
Egypt
El Salvador
Equatorial Guinea
Eritrea
Estonia
Eswatini
Ethiopia
Falkland Islands
Faroe Islands
Fiji
Finland
France
French Guiana
French Polynesia
French Southern Territories
Gabon
Gambia
Georgia
Germany
Ghana
Gibraltar
Greece
Greenland
Grenada
Guadeloupe
Guam
Guatemala
Guernsey
Guinea
Guinea-Bissau
Guyana
Haiti
Heard Island and McDonald Islands
Holy See (Vatican City State)
Honduras
Hong Kong
Hungary
Iceland
India
Indonesia
Iran
Iraq
Ireland
Isle of Man
Israel
Italy
Jamaica
Japan
Jersey
Jordan
Kazakhstan
Kenya
Kiribati
Korea, North
Korea, South
Kosovo
Kuwait
Kyrgyzstan
Lao People's Democratic Republic
Latvia
Lebanon
Lesotho
Liberia
Libya
Liechtenstein
Lithuania
Luxembourg
Macao
Madagascar
Malawi
Malaysia
Maldives
Mali
Malta
Marshall Islands
Martinique
Mauritania
Mauritius
Mayotte
Mexico
Micronesia, Federated States of
Moldova
Monaco
Mongolia
Montenegro
Montserrat
Morocco
Mozambique
Myanmar
Namibia
Nauru
Nepal
Netherlands
New Caledonia
New Zealand
Nicaragua
Niger
Nigeria
Niue
Norfolk Island
North Macedonia
Northern Mariana Islands
Norway
Oman
Pakistan
Palau
Palestine, State of
Panama
Papua New Guinea
Paraguay
Peru
Philippines
Pitcairn
Poland
Portugal
Puerto Rico
Qatar
Romania
Russian Federation
Rwanda
Réunion
Saint Barthélemy
Saint Helena, Ascension and Tristan da Cunha
Saint Kitts and Nevis
Saint Lucia
Saint Martin (French part)
Saint Pierre and Miquelon
Saint Vincent and the Grenadines
Samoa
San Marino
Sao Tome and Principe
Saudi Arabia
Senegal
Serbia
Seychelles
Sierra Leone
Singapore
Sint Maarten (Dutch part)
Slovakia
Slovenia
Solomon Islands
Somalia
South Africa
South Georgia and the South Sandwich Islands
South Sudan
Spain
Sri Lanka
Sudan
Suriname
Svalbard and Jan Mayen
Sweden
Switzerland
Syria
Taiwan
Tajikistan
Tanzania
Thailand
Timor-Leste
Togo
Tokelau
Tonga
Trinidad and Tobago
Tunisia
Turkmenistan
Turks and Caicos Islands
Tuvalu
Türkiye
Uganda
Ukraine
United Arab Emirates
United States Minor Outlying Islands
United States of America
Uruguay
Uzbekistan
Vanuatu
Venezuela
Viet Nam
Virgin Islands, British
Virgin Islands, U.S.
Wallis and Futuna
Western Sahara
Yemen
Zambia
Zimbabwe

Study participating centre

Online study

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England

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not expected to be made available
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Other files	Plain language summary		28/01/2026	No	No
Participant information sheet			28/01/2026	No	Yes

Additional files

48915 Participant Information Sheet interview.pdf: Participant information sheet
48915 Plain language summary.pdf: Plain language summary

Editorial Notes

28/01/2026: Trial's existence confirmed by the National Institute for Health and Care Research (NIHR) (UK).