Aicardi-Goutières syndrome (AGS) is a rare genetic disorder that affects the brain, spinal cord and immune system. It is a type of leukodystrophy, a group of conditions that affect the white matter of the brain. This disorder also results in excessive interferon production, which is thought to cause injury not only to the brain, but also the skin, liver, lungs, heart, and other organs.

Researchers at Children’s Hospital of Philadelphia recently launched a Phase II clinical trial to investigate the safety and efficacy of baricitinib, a Janus kinase (JAK) inhibitor thought to decrease interferon signaling, potentially alleviating the inflammatory symptoms associated with this disorder.

Individuals who are interested in participating in the clinical trial will need to visit Children’s Hospital of Philadelphia for an initial 7-10 day screening visit, as well as follow-up visits every six (6) months. If you are interested in learning more about the study, you may either submit our referral survey or contact the study team at agsclinicaltrials@email.chop.edu.

You may also visit the official ClinicalTrials.gov listing for more information about the study.