Progressive familial intrahepatic cholestasis
MONDO:0015762Progressive familial intrahepatic cholestasis (PFIC) refers to a heterogeneous group of autosomal recessive disorders of childhood that disrupt bile formation and present with cholestasis of hepatocellular origin.
Also known as: PFIC, cholestasis, progressive familial intrahepatic
23 clinical trials for this condition and its sub-types, 16 tagged with Progressive familial intrahepatic cholestasis itself.
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Sub-types of Progressive familial intrahepatic cholestasis
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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 …
Phase 3 • Sponsor: Albireo, an Ipsen Company • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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Hope for rare liver disease: drug aims to ease severe itching in children
Disease control CompletedThis study tests the long-term safety of maralixibat, an oral medication, in children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease that causes intense itching. Participants who completed a prior study will receive maralixibat twice daily. Resear…
Phase 3 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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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…
Phase 2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:00 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…
Phase 2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:02 UTC
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New drug shows promise for rare liver disease itch
Symptom relief CompletedThis study tested a drug called TAK-625 (maralixibat) in 5 Japanese children with progressive familial intrahepatic cholestasis (PFIC), a rare liver disease that causes severe itching. The main goal was to see if the drug reduces itching over 26 weeks. Researchers also checked sa…
Phase 3 • Sponsor: Takeda • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC
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Spanish study hunts for hidden liver disease genes
Knowledge-focused CompletedThis study looks for genetic changes linked to progressive familial intrahepatic cholestasis (PFIC) in adults with unexplained bile flow problems. Researchers will collect health information and blood samples from 150 participants across Spain during a single visit. The goal is t…
Sponsor: Ipsen • Aim: Knowledge-focused
Last updated Aug 05, 2026 00:00 UTC