AAV:B - In Haemophilia B Patients
Research type
Research Study
Full title
AAV:B - Adenovirus-Associated Virus Antibodies in UK cohort of Haemophilia B Patients: A seroprevalence study of the presence of Adenovirus-Associated Virus Vector 6 neutralizing activity and antibodies in Patients with Haemophilia B
IRAS ID
246242
Contact name
Sara Boyce
Contact email
Sponsor organisation
Research and Development
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
0 years, 6 months, 2 days
Research summary
Haemophilia B is an X chromosome-linked bleeding disorders where patients have a deficiency of Factor IX (FIX) clotting protein leading to bleeding, either spontaneously or on trauma, depending on the severity. Treatment at present involves the infusion of FIX concentrate in order to prevent or treat bleeds. Although replacement of FIX has improved life expectancy and quality by reducing joint damage and spontaneous bleeding, there are limitations which include frequent infusions at high costs and the formation of inhibitors which makes this type of therapy ineffective. Therefore, gene-based therapies for haemophilia are being developed to achieve sustained high levels of clotting factor. Gene therapy is based on using an AAV vector with the human FIX genetic code inserted. These vectors home in on the liver in order to produce FIX constitutively. However, antibodies against these AAV serotypes often limit this form of therapy. This study therefore aims to assess AAV 6 antibody prevalence in the haemophilia B community in the UK to determine the potential numbers of patients for whom this therapy would be effective.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
18/EM/0313
Date of REC Opinion
6 Nov 2018
REC opinion
Further Information Favourable Opinion