PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2
Clinical trials for PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2 explained in plain language.
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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 TYPE 2
Phase: PHASE3 • Sponsor: Albireo, an Ipsen Company • Aim: Disease control
Last updated Jun 27, 2026 12:25 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…
Matched conditions: PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2
Phase: PHASE3 • Sponsor: Takeda • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC