Ataxia telangiectasia
MONDO:0008840Ataxia-telangiectasia is the association of severe combined immunodeficiency (affecting mainly the humoral immune response) with progressive cerebellar ataxia. It is characterized by neurological signs, telangiectasias, increased susceptibility to infections and a higher risk of cancer.
Also known as: Louis-Bar syndrome, ataxia - telangiectasia, ataxia telangiectasia, ataxia telangiectasia syndrome, AT, AT, complementation group A, AT, complementation group C, AT, complementation group D
35 clinical trials for this condition and its sub-types, 11 tagged with Ataxia telangiectasia itself.
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Sub-types of Ataxia telangiectasia
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Could red blood cells deliver a steroid to ease a rare brain disorder?
Disease control CompletedThis Phase 3 trial tested a treatment called EryDex for people with ataxia telangiectasia (A-T), a rare genetic disease that affects movement and coordination. EryDex is a steroid (dexamethasone) packaged inside the patient's own red blood cells and given as an IV infusion every …
Phase 3 • Sponsor: Quince Therapeutics S.p.A. • Aim: Disease control
Last updated Jun 27, 2026 12:25 UTC
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Can an immune drug slow a rare childhood brain disease?
Symptom relief CompletedAtaxia-telangiectasia is a rare, inherited disease that damages the nervous system, skin, and immune system, and it has no cure. Researchers are testing sirolimus, an immune-suppressing drug, in children under 17 with the condition. The six participants take sirolimus by mouth fo…
Phase 2 • Sponsor: Sara Elva Espinosa Padilla, MD • Aim: Symptom relief
Last updated Sep 17, 2026 00:00 UTC
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Large study looks at how a 25-Gene cancer test affects patients and families
Knowledge-focused CompletedThis study looked at how a genetic test that checks 25 genes linked to hereditary cancers is used in clinics. Over 1,500 people with a personal or family history of cancer took part. Researchers collected blood samples and had participants fill out questionnaires over five years …
Sponsor: University of Southern California • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:06 UTC