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: 28 July 2023

Current Status: Published.

To see the results of the trial, please see the publication and the lay summary of study results below:

Reverse transcriptase inhibitors in Aicardi–Goutières syndrome: A crossover clinical trial (143.43 KB / PDF)

AGS-RTI Study - Lay summary of results (131.83 KB / PDF)

Website: Yanick Crow research group: https://institute-genetics-cancer.ed.ac.uk/research/research-groups-a-z/crow-group

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

Trial Management:

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 / Database

This article was published on 2024-09-24