296. 胆道閉鎖症 Biliary atresia Clinical trials / Disease details
臨床試験数 : 63 / 薬物数 : 65 - (DrugBank : 37) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 63
| 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 | ||
| 2 | EUCTR2020-000974-22-FR (EUCTR) | 07/12/2020 | 25/09/2020 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;France;Canada;Poland;Germany;United Kingdom;Italy | ||
| 3 | EUCTR2019-002755-42-PL (EUCTR) | 08/01/2020 | MERGE: Maralixibat Extension Safety Study Providing Long-term Treatment to Subjects with Cholestatic Liver Disease. | MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study. | Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease including, but not limited to, Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia. MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10053870;Term: Alagille syndrome;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | NA | Female: yes Male: yes | 109 | Phase 2 | France;United States;Canada;Spain;Belgium;Poland;Australia;United Kingdom | |||
| 4 | EUCTR2020-000974-22-PL (EUCTR) | 09/10/2020 | 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 | France;United States;Canada;Poland;Germany;United Kingdom;Italy | |||
| 5 | 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 | |||
| 6 | EUCTR2019-002755-42-BE (EUCTR) | 11/12/2019 | MERGE: Maralixibat Extension Safety Study Providing Long-term Treatment to Subjects with Cholestatic Liver Disease. | MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study. | Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease including, but not limited to, Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia. MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10053870;Term: Alagille syndrome;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | NA | Female: yes Male: yes | 109 | Phase 2 | France;United States;Canada;Spain;Poland;Belgium;Australia;United Kingdom |