Edinburgh Clinical Trials


Inhibition of reverse transcription in type I interferon mediated neuropathology

Aicardi-Goutières syndrome (AGS) is a disease of children, particularly affecting the brain and the skin. There is a close link between AGS and increased amounts of a chemical called interferon. Normally humans only produce interferon when they are infected with a virus. In AGS, there is no viral infection. Instead, the cells in the cells of affected patients are confused into thinking that their own genetic material is coming from a virus. As a result they produce interferon all the time, which acts as a poison that damages the cells. The Investigators wish to treat AGS patients with drugs called reverse transcriptase inhibitors (RTIs), used to fight the HIV-1 virus that causes AIDS. The investigators will monitor the effect of treatment on interferon levels, and look at other markers which might give us clues to how the drugs are working. The trial is funded by the Medical Research Council, and involves experts based in Edinburgh, Birmingham, Manchester and London.


Chief Investigator: Professor Yanick Crow

Number and location of participating sites (by region/ country): 4 sites in the UK  Edinburgh, London, Manchester, Birmingham


EudraCT number: 2020-003502-31 

ISRCTN number: ClincialTrials.Gov - NCT04731103   https://clinicaltrials.gov/ct2/show/NCT04731103


Funder: Medical Research Council

Start and End date

Of grant award: 01 February 2020 - 31 July 2024

Of recruitment:

Current Status: Recruiting

Website: Yanick Crow research group: https://www.ed.ac.uk/mrc-human-genetics-unit/research/crow-research-group/aicardi-goutieres-syndrome

Email: agsrti.trial@ed.ac.uk


UK GDPR Privacy Statement: Information included in Participant Information Leaflet


Sponsor: The University of Edinburgh & Lothian Health Board; ACCORD https://accord.scot/


Chief Investigator:

Professor Yanick Crow

MRC Institute of Genetics and Molecular Medicine, University of Edinburgh, Crew Road, Edinburgh.  Email: Yanick.Crow@ed.ac.uk

Trial Manager:

Claire Battison, ECTU, University of Edinburgh, Level 2, NINE Edinburgh BioQuarter 9 Little France Road

Assistant Trial Manager:

Annabel Giddings, ECTU, University of Edinburgh, Level 2, NINE Edinburgh BioQuarter 9 Little France Road


ECTU involvement: Trial Management, Statistics and Database