Aicardi-Goutieres syndrome
MONDO:0018866Aicardi-Goutieres syndrome (AGS) is an inherited, subacute encephalopathy characterized by the association of basal ganglia calcification, leukodystrophy and cerebrospinal fluid (CSF) lymphocytosis.
Also known as: Aicardi Goutieres syndrome, Aicardi-Goutières Syndrome, Cree encephalitis, encephalopathy with basal ganglia calcification, encephalopathy with intracranial calcification and chronic lymphocytosis of cerebrospinal fluid, AGS, Aicardi-Goutières syndrome, encephalopathy, familial infantile, with calcification of basal ganglia and chronic cerebrospinal fluid lymphocytosis
60 clinical trials for this condition and its sub-types, 9 tagged with Aicardi-Goutieres syndrome itself.
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Sub-types of Aicardi-Goutieres syndrome
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Aicardi-Goutieres syndrome 1 2 trials
1 sub-type
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Aicardi-Goutieres syndrome 2 0 trials
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Aicardi-Goutieres syndrome 3 0 trials
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Aicardi-Goutieres syndrome 4 0 trials
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Aicardi-Goutieres syndrome 5 0 trials
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Aicardi-Goutieres syndrome 6 0 trials
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Aicardi-Goutieres syndrome 7 0 trials
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Aicardi-Goutieres syndrome 8 0 trials
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Aicardi-Goutieres syndrome 9 0 trials
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Could a rheumatoid arthritis drug calm a rare brain disease?
Disease control CompletedThis 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 improv…
Phase 2 • Sponsor: Adeline Vanderver, MD • Aim: Disease control
Last updated Jun 27, 2026 08:07 UTC
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Could HIV drugs tame a rare immune disease in kids?
Disease control CompletedThis small pilot study tested whether drugs normally used for HIV could help children with Aicardi-Goutières syndrome (AGS), a rare genetic disorder that causes severe brain inflammation. The trial gave 11 children a combination of three reverse transcriptase inhibitors to see if…
Phase 2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 26, 2026 13:51 UTC
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Could a single DNA test solve the mystery of rare brain diseases in kids?
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete read of a person's DNA) can help diagnose leukodystrophies, a group of rare brain diseases that are hard to identify. Researchers enrolled 236 children with white matter abnormalities on brain scans but no known gen…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:56 UTC