PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
Clinical trials for PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS 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: PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
Phase: PHASE2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Promising long-term results for rare liver disease drug in kids
Disease control CompletedThis study looked at the long-term safety and effectiveness of a drug called A4250 (odevixibat) in 116 children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease. The children took the drug for up to 72 weeks. The study measured changes in bile acid …
Matched conditions: PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
Phase: PHASE3 • Sponsor: Albireo, an Ipsen Company • Aim: Disease control
Last updated Jun 27, 2026 12:25 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: PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
Phase: PHASE2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC