A Multicentre, Seroprevalence Study of Subjects With Haemophilia B
Research type
Research Study
Full title
A Multicentre, Seroprevalence Study of Subjects With Haemophilia B
IRAS ID
216558
Contact name
Pratima Chowdary
Contact email
Sponsor organisation
Freeline Therapeutics Ltd
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
This is a single visit research study testing antibodies against different types of virus called adeno-associated virus (AAV) in people with hemophilia B. 1000 patients are planned to be enrolled.
Haemophilia B is a genetic disorder caused by low levels of Factor IX (coagulation factor) in the blood. It is manifested by frequent spontaneous bleedings, inside the body which can cause long term disability or even death.
The current treatment for Haemophilia B is intravenous infusions of Factor IX concentrates when bleeding occurs or on a regular basis to prevent bleeding (2 to 3 times a week). This is invasive treatment and cost for regular treatment is also a significant burden.. These unmet needs have driven interest in gene therapy of Haemophilia B.
The sponsor is developing a new gene therapy for Haemophilia B, using a technology which involves a virus called adeno-associated virus (AAV) to transfer the gene into human body. There are many different types of adeno-associated virus (AAV) virus that occur naturally in the environment and it is likely that participants have been infected with one or more of them over their lifetime. If they have been infected, they will have antibodies that will remain in their body in order to help fight off another infection. This new gene treatment for Haemophilia B will only be successful if the treated patients do not have antibodies to the AAV that is being used.
This study will involve a blood sample being taken to test for these antibodies to get a better understanding of how common the antibodies against each of the types of adeno-associated virus (AAV) are in Haemophilia B patients in different parts of the world.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
16/EM/0454
Date of REC Opinion
14 Nov 2016
REC opinion
Further Information Favourable Opinion