Maralixibat (in precedenza SHP625 o LUM001) ( DrugBank: Maralixibat )

1 disease
告示番号疾患名（ページ内リンク）臨床試験数
338進行性家族性肝内胆汁うっ滞症1

338. 進行性家族性肝内胆汁うっ滞症

臨床試験数 : 60 /  薬物数 : 25 - (DrugBank : 6) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2019-001211-22-IT
(EUCTR)		07/10/201902/02/2021A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Product Name: Maralixibat (in precedenza SHP625 o LUM001)
Product Code: [Maralixibat]
INN or Proposed INN: maralixibat cloruro		Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany