Biliary atresia

This 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…

This 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…

This 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…

This study is a registry that will review medical records of 1,000 patients treated for benign or malignant gastrointestinal diseases between 2005 and 2030. Researchers will look at survival, symptoms like heartburn and swallowing trouble, and quality of life. The goal is to lear…

This 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…

This study watches 200 people with lung, gut, or hormone diseases over time using special brain scans (PET). It aims to see how these diseases change brain activity and whether those changes can predict tumor return, spread, or survival. No new treatment is tested—just observatio…