biostudies-literatureCrow YJMedical Research CouncilNational Institute for Health Research (NIHR)750-757https://www.ebi.ac.uk/biostudies/studies/S-EPMC7617231biostudies-literatureUnknown67(6)<h4>Aim</h4>To extend the findings of a previous clinical trial suggesting combined abacavir (ABC), lamivudine (3TC), and zidovudine (AZT) reduces type I interferon (IFN) signalling in Aicardi-Goutières syndrome (AGS).<h4>Method</h4>This was an open label, non-placebo-controlled phase II clinical trial (NCT04731103) in patients less than 16 years with any of five AGS genotypes. The effect of ABC or 3TC individually, or of combined ABC + 3TC + AZT, on IFN-stimulated gene (ISG) expression (primary outcome) and IFN-alpha protein (secondary outcome) in blood was assessed.<h4>Results</h4>Thirteen patients were recruited. Compliance was poor in the ABC + 3TC + AZT arm. No statistically significant effects were observed with ABC or 3TC, or with ABC + 3TC + AZT over 6 weeks. A statistically signifDevelopmental medicine and child neurologyReverse transcriptase inhibitors in Aicardi-Goutieres syndrome: A crossover clinical trial.PMC7617231MC_UU_00035/11NIHR203956MR/S034676/1Price-Kuehne FD'Arco FCrow YJNorrie JSudarsanam AMaclean ABondet VDuffy DMurphy LKennel TDonald AHemingway CReid KRWassmer EQuigley AJEl-Daher MTEleftheriou DLivingstone KSutherland FJEnsor HMunro AFRice GIBattison CJack KForbes DAGS‐RTIs Study GroupParker RAMoraitis EBriggs TAShetty JMcmaster PHunt DPGiddings AHong YBurleigh AParida AfalseReverse transcriptase inhibitors in Aicardi-Goutieres syndrome: A crossover clinical trial.<h4>Aim</h4>To extend the findings of a previous clinical trial suggesting combined abacavir (ABC), lamivudine (3TC), and zidovudine (AZT) reduces type I interferon (IFN) signalling in Aicardi-Goutières syndrome (AGS).<h4>Method</h4>This was an open label, non-placebo-controlled phase II clinical trial (NCT04731103) in patients less than 16 years with any of five AGS genotypes. The effect of ABC or 3TC individually, or of combined ABC + 3TC + AZT, on IFN-stimulated gene (ISG) expression (primary outcome) and IFN-alpha protein (secondary outcome) in blood was assessed.<h4>Results</h4>Thirteen patients were recruited. Compliance was poor in the ABC + 3TC + AZT arm. No statistically significant effects were observed with ABC or 3TC, or with ABC + 3TC + AZT over 6 weeks. A statistically signif2025-01-01T00:00:00Z2025 Jun2026-07-15T21:54:19.582Z2025-04-04T01:54:38.927ZS-EPMC76172313963093510.1111/dmcn.16199