Maralixibat cloruro ( DrugBank: Maralixibat )

2 diseases

ID	Disease name (Link within this page)	Number of trials
296	Biliary atresia	1
338	Progressive familial intrahepatic cholestasis	2

296. Biliary atresia

Clinical trials : 71 / Drugs : 70 - (DrugBank : 39) / Drug target genes : 35 - Drug target pathways : 60

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2020-000974-22-IT (EUCTR)	22/03/2021	24/05/2021	Clinical study to evaluate the Efficacy and Safety of Maralixibat used in treatment of Biliary Atresia subjects after Hepatoportoenterostomy	Randomized, Double-blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy - EMBARK: Evaluation of Maralixibat in Biliary Atresia Response post Kasai	Biliary atresia (BA) is a rare, inflammatory condition of the biliary tree that presents in the first weeks of life and leads to bile duct obstruction and consequent liver injury, fibrosis and cirrhosis which lead to portal hypertension and a decline in hepatic synthetic function. Untreated, the outcome of BA is uniformly fatal. The 2 most important improvements inthe care of BA patients to date are the Kasai hepatoportoenterostomy (HPE; Kasai procedure) and orthotopic liver transplantation. MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE	Mirum Pharmaceuticals Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	72	Phase 2	France;United States;Canada;Poland;Germany;United Kingdom;Italy

338. Progressive familial intrahepatic cholestasis

Clinical trials : 60 / Drugs : 26 - (DrugBank : 6) / Drug target genes : 2 - Drug target pathways : 2

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2019-003395-39-IT (EUCTR)	20/05/2020	24/05/2021	Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).	MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) - Not Applicable	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 stagesof 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 Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro	Mirum Pharmaceuticals Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 3	United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany
2	EUCTR2019-001211-22-IT (EUCTR)	07/10/2019	02/02/2021	A 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.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 3	United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany