ALAGILLE SYNDROME
Clinical trials for ALAGILLE SYNDROME explained in plain language.
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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…
Matched conditions: ALAGILLE SYNDROME
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 …
Matched conditions: ALAGILLE SYNDROME
Phase: PHASE4 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated Jun 27, 2026 12:00 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…
Matched conditions: ALAGILLE SYNDROME
Phase: PHASE2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC