JAK Inhibitor Treatment in AGS

Summary

The primary objective of this study is to assess safety as well as efficacy of baricitinib, a Janus Kinase (JAK) inhibitor, in patients with Aicardi Goutières Syndrome (AGS), a multisystem heritable disorder of the innate immunity resulting in excessive interferon production

Timeline

Start

2019-06-03

Primary completion

2024-01-04

Completion

2024-03-25

Outcome

Met primary endpoint

registry p <0.0005; Estimated change (52 weeks - baseline) 0.9 (95% CI 0.5 to 1.2) NCT03921554 ↗

Publications

• Adang LA, Gavazzi F, D'Aiello R, Isaacs D, Bronner N, Arici ZS, Flores Z, Jan A, Scher C, Sherbini O, Behrens EM, Goldbach-Mansky R, Olson TS, Lambert MP, Sullivan KE, Teachey DT, Witmer C, Vanderver A, Shults J. Hematologic abnormalities in Aicardi Goutieres Syndrome. Mol Genet Metab. 2022 Aug;136(4):324-329. doi: 10.1016/j.ymgme.2022.06.003. Epub 2022 Jun 16.

Drugs

Indications