Could a rheumatoid arthritis drug calm a rare brain disease?
NCT ID NCT03921554
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tested baricitinib (Olumiant), a drug used for rheumatoid arthritis, in 54 people with Aicardi Goutières Syndrome (AGS), a rare genetic disorder that causes brain inflammation and developmental problems. The goal was to see if the drug could stabilize or improve neurologic function over 52 weeks. Participants took the drug by mouth or feeding tube, and researchers measured changes using a special neurologic scale.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Baricitinib (Olumiant), a JAK inhibitor drug taken by mouth or feeding tube
- What this could lead to
- If it works, this could point toward a treatment that slows or stabilizes neurologic decline in Aicardi Goutières Syndrome.
- What could go wrong
- This is a small, early-phase trial with no placebo group, so results may not prove the drug works. The disease is rare and variable, making it hard to measure improvement.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.