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 Image 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: 28 July 2023 Current Status: Closed to recruitment, follow up complete. 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, Edinburgh Clinical Trials Unit, Usher Building, The University of Edinburgh, 5‒7 Little France Road, Edinburgh BioQuarter ‒ Gate 3, Edinburgh EH16 4UX Assistant Trial Manager: Annabel Giddings, Edinburgh Clinical Trials Unit, Usher Building, The University of Edinburgh, 5‒7 Little France Road, Edinburgh BioQuarter ‒ Gate 3, Edinburgh EH16 4UX ECTU involvement: Trial Management, Statistics and Database This article was published on 2024-09-24
