PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC)
Clinical trials for PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC) explained in plain language.
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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…
Matched conditions: PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC)
Phase: PHASE3 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 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…
Matched conditions: PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS (PFIC)
Phase: PHASE3 • Sponsor: Takeda • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC