Alagille syndrome
MONDO:0007318Alagille (AGS) syndrome is variably characterized by chronic cholestasis due to paucity of intrahepatic bile ducts, peripheral pulmonary artery stenosis, vertebrae segmentation anomalies, characteristic facies, posterior embryotoxon/anterior segment abnormalities, pigmentary retinopathy, and dysplastic kidneys.
Also known as: Alagille syndrome, Alagille-Watson syndrome, Arteriohepatic dysplasia, syndromic bile duct paucity, Cardiovertebral syndrome, Hepatofacioneurocardiovertebral syndrome, Watson Alagille syndrome, Watson-Miller syndrome
15 clinical trials for this condition and its sub-types.
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Broader categories
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New hope for kids with rare liver disease: long-term drug study launched
Symptom relief OngoingThis study looks at the long-term safety and effectiveness of odevixibat in 70 people with Alagille syndrome, a rare genetic condition that affects the liver and other organs. The drug aims to reduce severe itching and improve bile flow. Participants who completed a prior 24-week…
Phase: PHASE3 • Sponsor: Albireo, an Ipsen Company • Aim: Symptom relief
Last updated Jul 04, 2026 00:00 UTC
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Major study on rare childhood liver diseases halted
Knowledge-focused TerminatedThis study followed children and young adults with genetic liver diseases that cause bile buildup. The goal was to track how these diseases progress over time, including the need for liver transplants or other complications. No treatments were tested; the aim was simply to learn …
Sponsor: Arbor Research Collaborative for Health • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:02 UTC