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 drug shows promise for babies with rare liver disorders
Disease control CompletedThis study tested the safety of a drug called maralixibat in 27 infants under 12 months old with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC), two rare liver diseases that cause bile buildup. The drug is given as an oral solution and aims to reduce bi…
Phase: PHASE2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Can a common cholesterol drug shrink skin lumps in kids with rare liver disease?
Disease control CompletedThis study tested the safety and effectiveness of atorvastatin (Lipitor) in 15 children with Alagille syndrome who had skin cholesterol deposits called xanthomas. The children took the drug for 6 months, and doctors graded the size and number of the lumps. The goal was to see if …
Phase: PHASE4 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New drug TAK-625 shows promise for rare liver disease in kids
Disease control CompletedThis phase 3 study tested TAK-625 (maralixibat) in 7 Japanese children with Alagille syndrome, a rare genetic liver disorder. The goal was to see if the drug reduces bile acid levels and itching, and to find the best dose. Participants took the drug daily for up to 34 months, wit…
Phase: PHASE3 • Sponsor: Takeda • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Liver drug maralixibat tested for Long-Term safety in rare disease patients
Disease control CompletedThis study looked at the long-term safety of a drug called maralixibat in 52 people with cholestatic liver disease, including Alagille Syndrome and other rare conditions. Participants had already taken maralixibat in an earlier study. Researchers tracked side effects and how well…
Phase: PHASE2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC
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Can a chest X-Ray and blood test quickly rule out TB?
Diagnosis CompletedThis study tested two new tools for diagnosing tuberculosis (TB): a software that analyzes chest X-rays (CAD4TB) and a blood test that measures C-reactive protein (CRP). Researchers enrolled 1,400 adults with TB symptoms in Lesotho and South Africa. They compared these tests to s…
Sponsor: Klaus Reither • Aim: Diagnosis
Last updated Jun 27, 2026 07:51 UTC