Prospective bleeding study in Glanzmann's Thrombasthenia
Research type
Research Study
Full title
A Prospective, Observational Study of Bleeding and Quality of Life in Subjects with Glanzmann Thrombasthenia in the United Kingdom
IRAS ID
313531
Contact name
Simon Fletcher
Contact email
Sponsor organisation
Hemab Therapeutics
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
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.
In this specific study we 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 hospital required.
Haemnet are a non-profit research organisation focused on understanding and articulating the lived experience of people with bleeding disorders. Hemab Therapeutics are developing a treatment designed to prevent bleeds in GT and data from this study will support treatment development.
REC name
London - Surrey Research Ethics Committee
REC reference
22/PR/0374
Date of REC Opinion
10 May 2022
REC opinion
Further Information Favourable Opinion