AGS-RTI
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