Prospective bleeding study in Glanzmann's thrombasthenia
| ISRCTN | ISRCTN15050281 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN15050281 |
| IRAS number | 313531 |
| Secondary identifying numbers | HMB-001-NH01, IRAS 313531 |
- Submission date
- 15/08/2023
- Registration date
- 28/09/2023
- Last edited
- 15/04/2024
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Plain English Summary
Background and study aims
Glanzmann's thrombasthenia (GT) is a rare, inherited bleeding disorder affecting both males and females. There are about 150 patients with GT in the UK. In normal blood clotting, platelet cells attach at the site of injury and group together via proteins on the platelet cell surface, forming a plug to stop bleeding. In GT a protein is missing or reduced (glycoprotein IIb/IIIa). The platelets don't plug injuries efficiently resulting in increased bleeding in patients with GT. Due to the rare nature of the condition, there has been limited knowledge regarding the types and frequency of bleeds in GT. Little is known about the impact of bleeding on patient symptoms and quality of life.
Hemab is a company developing HMB-001, a bispecific antibody, for the prophylactic treatment of GT. Understanding bleeding symptoms and impact will provide Hemab valuable information as it moves forward in developing HMB-001. The aim of this study is to collect information regarding bleeding episodes, quality of life, and the social and clinical impact of bleeds in participants with GT.
Who can participate?
Patients aged 16 years and over with GT
What does the study involve?
Participants undertake quality-of-life assessments to explore the effects of their symptoms. They will be asked to complete a detailed daily diary for 3 months to document all bleeding symptoms. This will help us learn about the types and frequency of bleeding that people experience. Participants will also be asked about the effects of bleeds. For example, participants will be asked to report pain, missed activities caused by bleeds or visits to hospital required. After 85 days, participants will be given the option to continue to complete the diary card and receive monthly follow-up calls for up to a further 3 months.
What are the possible benefits and risks of participating?
There are no direct benefits for participants. However, the study may help others in the future. Completing the questionnaires and the diary will require giving up some time every day for 12 weeks. Although the researchers do not think that answering the surveys or the diary completion will be distressing, weekly calls from will be undertaken to support the participants. Psychological support can be accessed via a GP or through the treatment centre if the participant feels this is required.
Where is the study run from?
Richmond Pharmacology Ltd (UK)
When is the study starting and how long is it expected to run for?
May 2022 to September 2023
Who is funding the study?
Hemab Therapeutics ApS (Denmark)
Who is the main contact?
Dr Catherine Rea, catherine@hemab.com
Contact information
Principal Investigator
Richmond Pharmacology Ltd
1A Newcomen Street
London Bridge
London
SE1 1YR
United Kingdom
 ORCID ID |
0000-0002-6316-5860 |
|---|---|
| Phone | +44 (0)2070425800 |
| u.lorch@richmondpharmacology.com |
Scientific
Richmond Pharmacology Ltd
1A Newcomen Street
London Bridge
London
SE1 1YR
United Kingdom
| ORCID ID |
0000-0002-6364-9822 |
|---|---|
| Phone | +44 (0)2070425800 |
| j.melo@richmondpharmacology.com |
Study information
| Study design | Observational cohort study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cohort study |
| Study setting(s) | Community, Home, Pharmaceutical testing facility |
| Study type | Diagnostic, Quality of life, Screening |
| Participant information sheet | Not available in web format, please use the contact details to request a participant information sheet |
| Scientific title | A prospective, observational study of bleeding and quality of life in subjects with Glanzmann thrombasthenia in the United Kingdom |
| Study hypothesis | Glanzmann's thrombasthenia (GT) is a rare, inherited bleeding disorder affecting both males and females. There are approximately 150 patients with GT in the UK. In normal blood clotting, platelet cells attach at the site of injury and group together via proteins on the platelet cell surface, forming a plug to stop bleeding. In GT a protein is missing or reduced (glycoprotein IIb/IIIa). The platelets don't plug injuries efficiently resulting in increased bleeding in patients with GT. Due to the rare nature of the condition, there has been limited knowledge regarding the types and frequency of bleeds in GT. Little is known about the impact of bleeding on patient symptoms and quality of life. In order to develop new treatments and ways of managing patients, it is important to first understand the types of symptoms and their impact on patients. The Glanzmann 360 project is being undertaken to improve understanding of GT and the effects that it has on individuals. |
| Ethics approval(s) |
Approved 10/05/2022, London - Surrey Research Ethics Committee (The Old Chapel, Royal Standard Place, Nottingham, NG1 6FS, United Kingdom; +44 (0)2071048388; surrey.rec@hra.nhs.uk), ref: 22/PR/0374 |
| Condition | Glanzmann's thrombasthenia |
| Intervention | The researchers will ask participants to undertake quality-of-life assessments to explore the effects of their symptoms. They will then be asked to complete a detailed daily diary for 3 months to document all bleeding symptoms. This will help us learn about the types and frequency of bleeding that people experience. Participants will also be asked about the effects of bleeds. For example, participants will be asked to report pain, missed activities caused by bleeds or visits to the hospital required. |
| Intervention type | Other |
| Primary outcome measure | 1. Quantitative assessment of bleeding using the International Society on Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) at baseline 2. Clinical outcomes over 3 months of observation, including: 2.1. Number of bleeding events measured using daily electronic bleed diary 2.2. Sites of bleeding events measured using daily electronic bleed diary 2.3. Treatments administered for bleeding events measured using daily electronic bleed diary 2.4 Number of hospitalizations due to bleeding events measured using daily electronic bleed diary 4. Quality of life measured at baseline using: 4.1. Patient-Reported Outcomes Measurement Information System (PROMIS)-29 4.2. Menstrual Impact Questionnaire (MIQ) 5. Social outcomes, including missed work, school, and activities, measured using [daily electronic bleed diary] over 3 months of observation |
| Secondary outcome measures | There are no secondary outcome measures |
| Overall study start date | 02/05/2022 |
| Overall study end date | 01/09/2023 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Mixed |
| Lower age limit | 16 Years |
| Upper age limit | 99 Years |
| Sex | Both |
| Target number of participants | 30 |
| Total final enrolment | 30 |
| Participant inclusion criteria | Each participant must meet all of the following inclusion criteria to be eligible for enrollment in the study: 1. Participant has signed the informed consent form 2. ≥16 years of age 3. Participant has confirmed GT (documented diagnosis on patient held registry document or clinical record) 4. Participant has sufficient English language skills to complete the consent process, electronic questionnaires, and diary |
| Participant exclusion criteria | Each participant must not meet any of the following exclusion criteria to be eligible for enrolment in the study: 1. Participant is currently pregnant 2. Participant has a cancer diagnosis (excluding benign or localized disease eg. basal cell carcinoma of the skin) and is currently undergoing systemic treatment 3. Participant has renal impairment currently requiring renal replacement therapy 4. Participant has liver disease currently requiring treatment 5. Participant has heart disease currently requiring treatment 6. Participant is receiving current treatment with anticoagulation or anti-platelet therapies |
| Recruitment start date | 10/05/2022 |
| Recruitment end date | 01/05/2023 |
Locations
Countries of recruitment
- England
- United Kingdom
Study participating centre
London Bridge
London
SE1 1YR
United Kingdom
Sponsor information
Industry
125 Cambridge Park Drive
Cambridge
MA 02140
United States of America
| catherine@hemab.com | |
| Website | https://hemab.com/ |
Funders
Funder type
Industry
No information available
Results and Publications
| Intention to publish date | 01/10/2023 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | Peer-reviewed scientific journals; conference presentation; other publications. A final report summary is to be sent to patients on study completion, with their permission. |
| IPD sharing plan | The data-sharing plans for the current study are unknown and will be made available at a later date. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | 02/11/2023 | 15/04/2024 | Yes | No |
Editorial Notes
15/04/2024: Publication reference added.
30/08/2023: Study's existence confirmed by Hemab Therapeutics.
