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

    jonathan.foley@freeline.life

  • 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