Thrombin generation assay (TGA)
Research type
Research Study
Full title
Evaluation of global haemostatic capacity in haemophilia B patient samples containing the FIX-Padua variant
IRAS ID
289825
Contact name
Jonathan Foley
Contact email
Sponsor organisation
Freeline Therapeutics
Duration of Study in the UK
0 years, 2 months, 5 days
Research summary
There are numerous Factor IX (FIX) activity assays in routine use in the clinic for the diagnosis and monitoring of patients with haemophilia B. Preliminary data has shown that FIX activity from a certain FIX variant (FIX-Padua) expressed by investigational AAV gene therapy products can vary by up to 3-fold depending on the FIX assay used. In the proposed study, we will evaluate haemophilia B patient samples containing the FIX-Padua variant with a global assay of haemostasis (thrombin generation assay, TGA) and 2 routine FIX assays. By comparing TGA results to the previously established normal range, we hope to establish which routine FIX assay are most appropriate for measuring FIX-Padua activity in the clinic. This pilot study would inform on the usefulness of this assay in a larger study.
REC name
South East Scotland REC 01
REC reference
20/SS/0126
Date of REC Opinion
2 Dec 2020
REC opinion
Favourable Opinion