Troponin measurements pre- and post- AAV gene therapy

• Research type

  Research Study

• Full title

  Troponin measurements pre- and post- AAV gene therapy

• IRAS ID

  306322

• Contact name

  Jonathan Foley

• Contact email

  jonathan.foley@freeline.life

• Sponsor organisation

  Freeline Therapeutics

• Duration of Study in the UK

  0 years, 6 months, 2 days

• Research summary

  Freeline has two in vivo AAV gene therapy programs in the clinic - for the treatment of haemophilia B and Fabry disease. Both programmes utilise the same recombinant capsid, AAVS3. We have observed elevated troponin levels in our Fabry patients after gene therapy. Heart involvement (and elevated troponin) is a classic component and part of the natural history of Fabry disease; however, given the temporality of elevated troponin levels with respect to the timing of gene therapy administration, we are interested in examining troponin levels in patients (i.e. haemophilia B) where elevated troponin levels are not typically a part of the natural history of that disease. This study will address whether AAVS3 administration is temporality associated with elevated troponin levels in haemophilia B patients.

• REC name

  Yorkshire & The Humber - Bradford Leeds Research Ethics Committee

• REC reference

  21/YH/0257

• Date of REC Opinion

  7 Oct 2021

• REC opinion

  Favourable Opinion