Cholestasis

This expanded access program offers the experimental drug odevixibat to people with progressive familial intrahepatic cholestasis (PFIC) who have severe itching and high bile acid levels but cannot join the main clinical trial. The drug works by blocking a bile acid transporter i…

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 study tests a medicine called volixibat to see if it can reduce severe itching in people with primary sclerosing cholangitis (PSC), a rare liver disease. About 182 participants will receive either volixibat or a placebo, and researchers will track itch scores using a daily d…

This study tests the long-term safety of linerixibat, a drug for severe itching caused by primary biliary cholangitis (PBC). All 242 participants receive the drug openly. The goal is to see if it remains safe over time and improves quality of life.

This study follows up to 90 children and young adults with mitochondrial liver diseases to learn how these conditions progress over time. Researchers will collect medical data and samples to better understand the diseases and find markers that predict outcomes. The goal is to imp…

This 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 …

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…