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.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Part of
Sub-types of Progressive familial intrahepatic cholestasis
-
New registry tracks Odevixibat's Long-Term impact on rare liver disease
Disease control Recruiting nowThis registry will follow 10 people with progressive familial intrahepatic cholestasis (PFIC) in South Korea who are taking odevixibat (Bylvay). The goal is to see how safe and effective the drug is over the long term in real-world use. Researchers will track side effects and how…
Sponsor: Ipsen • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
-
New registry tracks Real-World use of PFIC drug odevixibat
Disease control Recruiting nowThis registry will follow 20 people in China with progressive familial intrahepatic cholestasis (PFIC) who are taking odevixibat (Bylvay) as part of their normal care. The goal is to see how safe the drug is over the long term and how well it controls symptoms like severe itching…
Sponsor: Ipsen • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
-
New drug livmarli tracked for Long-Term safety in kids with rare liver disorders
Disease control Recruiting nowThis study follows 230 children with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC) who are taking Livmarli, a drug to reduce bile buildup and itching. Researchers will monitor side effects, liver function, and long-term outcomes like need for transplan…
Phase 4 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
-
Baby jaundice may leave lasting marks on teeth, new study investigates
Knowledge-focused Recruiting nowThis study looks at how common tooth staining is in children who had very high bilirubin levels (jaundice) early in life. Researchers will check 120 children with certain liver conditions to see if their teeth are discolored and whether that affects their daily life. The goal is …
Sponsor: University Hospital, Toulouse • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:32 UTC
-
New national registry aims to unlock secrets of rare childhood liver diseases
Knowledge-focused Recruiting nowThis study creates a Canadian registry for children under 18 with Alagille syndrome or genetic intrahepatic cholestasis, rare liver conditions that cause bile buildup. Researchers will collect health data from medical records over time to understand how these diseases progress an…
Sponsor: Children's Hospital of Eastern Ontario • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:28 UTC
-
Gene hunt for mysterious adult liver disease
Knowledge-focused Recruiting nowThis study is using advanced whole-genome sequencing to find hidden genetic causes of cholestatic liver disease in adults whose condition remains unexplained after standard tests. Researchers will compare the DNA of 60 affected adults to a large group of healthy controls. The goa…
Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:01 UTC
-
New safety study monitors liver risks in kids taking livmarli
Knowledge-focused Recruiting nowThis study is monitoring 50 children in Japan with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis (PFIC) who are taking the drug Livmarli (maralixibat). Researchers are using a national registry to track how many children develop liver problems and how …
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:07 UTC