Could a rheumatoid arthritis drug calm a rare brain disease?
NCT ID NCT03921554
First seen Jan 07, 2026 · Last updated Jun 23, 2026 · Updated 25 times
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.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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.
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.