Progressive familial intrahepatic cholestasis type 1
MONDO:0008892PFIC1, a type of progressive familial intrahepathic cholestasis (PFIC), is an infantile hereditary disorder in bile formation that is hepatocellular in origin and associated with extrahepatic features.
Also known as: Byler disease, FIC1 deficiency, PFIC1, cholestasis, progressive familial intrahepatic 1, cholestasis, progressive familial intrahepatic, type 1, Byler's disease, cholestasis, fatal intrahepatic, cholestasis, progressive familial intrahepatic, 1
11 clinical trials for this condition and its sub-types, 4 tagged with Progressive familial intrahepatic cholestasis type 1 itself.
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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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New drug shows promise for rare childhood liver disease
Disease control CompletedThis study tested a drug called odevixibat (A4250) in 62 children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2, a rare liver disease that causes severe itching and bile buildup. The drug works by blocking the reabsorption of bile acids in the gut. The g…
Phase 3 • Sponsor: Albireo • Aim: Disease control
Last updated Jun 27, 2026 08:00 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