Biliary atresia
MONDO:0008867A rare, biliary tract disease characterized by progressive obliterative cholangiopathy of the intra- and extrahepatic bile ducts, occurring in the embryonic/ perinatal period, leading to severe and persistent neonatal jaundice and acholic stool.
Also known as: atresia of bile ducts, biliary atresia, congenital, congenital biliary atresia, isolated atresia of bile ducts, non-syndromic biliary atresia, isolated biliary atresia
30 clinical trials for this condition and its sub-types.
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Broader categories
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New drug may help kids with rare liver disease avoid transplant
Disease control ENROLLING_BY_INVITATIONThis study tests a drug called odevixibat in 180 children with biliary atresia, a rare liver disease. The goal is to see if the drug helps children stay alive without needing a liver transplant over the long term. Children who completed a previous study can join this open-label e…
Phase: PHASE3 • Sponsor: Albireo, an Ipsen Company • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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Special milk powder aimed to help sick infants – but study never started
Disease control TerminatedThis study planned to test whether a milk powder rich in medium-chain fatty acids could improve nutrition and health in infants with cholestatic liver disease. Researchers aimed to compare the special formula to regular milk powder in 64 infants under 6 months old. However, the s…
Phase: NA • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated Jun 26, 2026 17:00 UTC
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A drop of blood could spot a deadly liver disease in newborns
Diagnosis TerminatedThis trial is testing whether a dry blood spot test—using a tiny sample from a newborn's heel—can screen for biliary atresia, a serious liver condition that blocks bile flow. The test measures certain biomarkers (direct bilirubin, γ-GT, and MMP-7) that may be elevated in affected…
Phase: NA • Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine • Aim: Diagnosis
Last updated Aug 02, 2026 00:00 UTC
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New drug could soothe unbearable itch in rare liver conditions
Symptom relief OngoingThis Phase 3 trial tests whether maralixibat, an oral solution, can safely reduce severe itching (pruritus) in children and adults with rare cholestatic liver diseases. The study enrolls 90 participants aged 6 months and older who have not responded to other treatments. Over 20 w…
Phase: PHASE3 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Symptom relief
Last updated Aug 13, 2026 00:00 UTC
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Tiny blood cells may reveal clues about liver disease in kids
Knowledge-focused OngoingThis pilot study looks at red blood cells in 20 children with cholestatic liver disease and compares them to healthy children. Researchers use a special test called ektacytometry to measure how fragile and flexible the red blood cells are. The goal is to see if liver problems cha…
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:02 UTC