DDrare: Database of Drug Development for Rare Diseases

Searched query = "Biliary atresia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = A4250; Bicarbonate Ringer's solution; Cefoperazone; Chloride; Corticosteroids; Cysteine; Desflurane; Desflurane anesthetics; Dexamethasone; Dimethyl fumarate; Eicosapentaenoic acid; Epadel; Filgrastim; Gold; Granulocyte Colony-Stimulating Factor; ICG Pulsion; ICG-Pulsion 25mg; Indocyanine green; Intravenous immunoglobulin (IVIG); Lactated Ringer; Lactobacillus casei rhamnosus (Lcr35); MARALIXIBAT CHLORIDE; Magnesium; Magnesium gluconate; Magnesium sulfate; Maralixibat; Meloxicam; Methylprednisolone; N-Acetyl cysteine; Neomycin; Normal saline; OBETICHOLIC ACID; OCA; OCA 0.1 mg; OCA 1.5mg; OCA 5mg; Obeticholic acid; Ocaliva; Odevixibat; Oral Vancomycin; Other Crystalloid; Pentoxifylline; Prednisolone; Propanolol; Propofol; Rifaximin; Rituximab; Rocuronium; Sevoflurane; Sevoflurane anesthetics; Sulfate; UDCA; Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation; VO4CX; Vancomycin; [A4250]

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-GB (EUCTR)		21/10/2020	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 in the 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 INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat INN or Proposed INN: MARALIXIBAT CHLORIDE	Mirum Pharmaceuticals Inc.	NULL	NA			Female: yes Male: yes	72	Phase 2	United States;France;Canada;Poland;Germany;Italy;United Kingdom

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EUCTR2020-000974-22-GB
(EUCTR)

21/10/2020

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 in the 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
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE

Mirum Pharmaceuticals Inc.

NULL

Female: yes
Male: yes

Phase 2

United States;France;Canada;Poland;Germany;Italy;United Kingdom