Aicardi-Goutieres syndrome

MONDO:0018866

Aicardi-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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Where it sits in the disease tree

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Part of

↑ Hereditary disorder of connective tissue (1314) ↑ Autosomal recessive disease (999) ↑ Inborn error of immunity (345) ↑ Leukodystrophy (72) ↑ Type 1 interferonopathy of childhood (19)

Sub-types of Aicardi-Goutieres syndrome

Aicardi-Goutieres syndrome 1 2 trials
1 sub-type
- TREX1-related autosomal dominant Aicardi-Goutieres syndrome 0 trials
Aicardi-Goutieres syndrome 2 0 trials
Aicardi-Goutieres syndrome 3 0 trials
Aicardi-Goutieres syndrome 4 0 trials
Aicardi-Goutieres syndrome 5 0 trials
Aicardi-Goutieres syndrome 6 0 trials
Aicardi-Goutieres syndrome 7 0 trials
Aicardi-Goutieres syndrome 8 0 trials
Aicardi-Goutieres syndrome 9 0 trials
Basal ganglia calcification, idiopathic, childhood-onset 0 trials

Including sub-types (60) Tagged with Aicardi-Goutieres syndrome (9)

Trials to join now! 4 Not yet finished but already full! 1 Completed 3 Terminated 1

New pill aims to tame rare immune diseases

Disease control Ongoing

This early-stage trial tests an experimental drug called BI 3000202 in 16 adults with rare type 1 interferonopathies, such as Aicardi-Goutières syndrome. Participants take a low dose for 4 weeks, then a higher dose for 36 weeks. The main goal is to see if the drug is safe and how…

Phase 1 • Sponsor: Boehringer Ingelheim • Aim: Disease control

Last updated Sep 17, 2026 00:00 UTC

Aicardi-Goutieres syndrome

Where it sits in the disease tree

New pill aims to tame rare immune diseases