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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New drug hope for rare childhood immune disorders
Disease control Recruiting nowThis early-stage trial tests a new medicine called IMSB301 in people with rare diseases where the immune system attacks the body. The study includes up to 6 participants aged 12 and older. The main goal is to check if the drug is safe and how it behaves in the body.
Phase 1 • Sponsor: ImmuneSensor Therapeutics Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Major study launches to unravel rare genetic diseases affecting immunity and the brain
Knowledge-focused Recruiting nowThis study aims to learn more about rare genetic diseases that affect both the immune system and the nervous system. Researchers will collect medical information and samples from 1,000 participants, including patients, their relatives, and healthy volunteers. The goal is to bette…
Sponsor: Imagine Institute • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC
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Scientists launch study to unravel RNA's role in rare brain diseases
Knowledge-focused Recruiting nowThis study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions…
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:00 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC