Reverse transcriptase inhibitors in Aicardi-Goutieres syndrome: A crossover clinical trial.

ABSTRACT:

Aim

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).

Method

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.

Results

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 signif

SUBMITTER: Crow YJ 

PROVIDER: S-EPMC7617231 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature