MARALIXIBAT CHLORIDE ( DrugBank: Chloride, Maralixibat )
3 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 296 | 胆道閉鎖症 | 5 |
| 297 | アラジール症候群 | 11 |
| 338 | 進行性家族性肝内胆汁うっ滞症 | 24 |
296. 胆道閉鎖症
臨床試験数 : 71 / 薬物数 : 70 - (DrugBank : 39) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 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 | ||
| 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 | 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 | |||
| 4 | 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 | |||
| 5 | 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 |
297. アラジール症候群
臨床試験数 : 45 / 薬物数 : 21 - (DrugBank : 10) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 5
| 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 | EUCTR2020-004628-40-FR (EUCTR) | 25/03/2021 | 16/12/2020 | Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. | Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation (RISE). | Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | France;Poland;Belgium;United Kingdom | ||
| 2 | EUCTR2020-004628-40-BE (EUCTR) | 29/01/2021 | 15/12/2020 | Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. | Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation (RISE). | Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | France;United States;Mexico;Poland;Brazil;Belgium;United Kingdom | ||
| 3 | EUCTR2019-002755-42-FR (EUCTR) | 24/03/2020 | 16/01/2020 | 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). 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 2 | United States;France;Canada;Spain;Poland;Belgium;Australia;United Kingdom | ||
| 4 | EUCTR2019-002755-42-GB (EUCTR) | 19/03/2020 | 23/12/2019 | 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). 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 2 | United States;France;Canada;Spain;Poland;Belgium;Australia;United Kingdom | ||
| 5 | EUCTR2019-002755-42-ES (EUCTR) | 07/02/2020 | 06/02/2020 | 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). 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 2 | Canada;Belgium;United States;Poland;United Kingdom;Australia;France;Spain | ||
| 6 | EUCTR2013-005373-43-GB (EUCTR) | 21/04/2015 | 06/02/2015 | The purpose of this study is to evaluate a drug (LUM001 also known as SHP625) that may help treat the liver and control itching in Alagille Syndrome. In the Optional Follow-up Treatment Period (after Week 48), all eligible children treated in the LUM001-304 study will be offered to continue the study drug treatment until the subjects are eligible to enter another LUM001 study or LUM001 is available commercially, or the sponsorstops the program or development in this indication. | Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome - ICONIC | Alagille syndrome (ALGS) is an autosomal dominant with variable penetration genetic multisystem disorder. The clinical diagnosis is based on the presence of intrahepatic bile duct paucity on liver biopsy in association with at least three of the major clinical features: chronic cholestasis, cardiac disease, skeletal abnormalities, ocular abnormalities and characteristic facial features. MedDRA version: 20.0;Level: PT;Classification code 10053870;Term: Alagille syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LUM001 Product Code: LUM001 INN or Proposed INN: Maralixibat chloride | Mirum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Poland;Spain;Australia;United Kingdom | ||
| 7 | EUCTR2013-003832-54-GB (EUCTR) | 28/11/2013 | 09/12/2013 | A MULTICENTRE CLINICAL STUDY TO EVALUATE THE SAFETY AND EFFICACY OF LUM001, AN AGENT THAT INHIBITS BILE ACID REUPTAKE FROM THE INTESTINE, IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PAEDIATRIC PATIENTS WITH ALAGILLE SYNDROME | A MULTICENTRE EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND DURABILITY OF THE THERAPEUTIC EFFECT OF LUM001, AN APICAL SODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTI), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC SUBJECTS WITH ALAGILLE SYNDROME - IMAGINE STUDY | Alagille syndrome (ALGS). This is an example of cholestatic liver disease in children. In patients with Alagille syndrome, impairment of the egress of bile acids from the liver leads to cholestasis, hepatocellular injury and damage, and progressive liver disease that may ultimately lead to the need for liver transplantation. Itch is the archetypal symptom of cholestasis, occurring at all stages of cholestatic liver disease, with or without jaundice. MedDRA version: 20.0;Level: PT;Classification code 10053870;Term: Alagille syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LUM001 INN or Proposed INN: maralixibat chloride | Mirum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | United Kingdom | ||
| 8 | 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). 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;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 | 53 | Phase 2 | Canada;Belgium;United States;Poland;United Kingdom;Australia;France;Spain | |||
| 9 | EUCTR2013-005373-43-PL (EUCTR) | 09/07/2014 | The purpose of this study is to evaluate a drug (LUM001 also known as SHP625) that may help treat the liver and control itching in Alagille Syndrome. In the Optional Follow-up Treatment Period (after Week 48), all eligible children treated in the LUM001-304 study will be offered to continue the study drug treatment until the subjects are eligible to enter another LUM001 study or LUM001 is available commercially, or the sponsorstops the program or development in this indication. | Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome - ICONIC | Alagille syndrome (ALGS) is an autosomal dominant with variable penetration genetic multisystem disorder. The clinical diagnosis is based on the presence of intrahepatic bile duct paucity on liver biopsy in association with at least three of the major clinical features: chronic cholestasis, cardiac disease, skeletal abnormalities, ocular abnormalities and characteristic facial features. MedDRA version: 20.0;Level: PT;Classification code 10053870;Term: Alagille syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: LUM001 Product Code: LUM001 INN or Proposed INN: Maralixibat chloride | Mirum Pharmaceuticals,Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Spain;Poland;Australia;United Kingdom | |||
| 10 | 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 | |||
| 11 | EUCTR2020-004628-40-PL (EUCTR) | 20/01/2021 | Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. | Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation (RISE). | Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. 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;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 | 20 | Phase 2 | France;United States;Mexico;Belgium;Brazil;Poland;United Kingdom |
338. 進行性家族性肝内胆汁うっ滞症
臨床試験数 : 60 / 薬物数 : 26 - (DrugBank : 6) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 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 | EUCTR2020-004628-40-FR (EUCTR) | 25/03/2021 | 16/12/2020 | Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. | Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation (RISE). | Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | France;Poland;Belgium;United Kingdom | ||
| 2 | EUCTR2020-004628-40-BE (EUCTR) | 29/01/2021 | 15/12/2020 | Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. | Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation (RISE). | Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | France;United States;Mexico;Poland;Brazil;Belgium;United Kingdom | ||
| 3 | EUCTR2019-003395-39-GB (EUCTR) | 30/03/2020 | 28/11/2019 | 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) | 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 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 | 30 | Phase 3 | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany | ||
| 4 | EUCTR2019-002755-42-FR (EUCTR) | 24/03/2020 | 16/01/2020 | 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). 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 2 | United States;France;Canada;Spain;Poland;Belgium;Australia;United Kingdom | ||
| 5 | EUCTR2019-002755-42-GB (EUCTR) | 19/03/2020 | 23/12/2019 | 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). 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 2 | United States;France;Canada;Spain;Poland;Belgium;Australia;United Kingdom | ||
| 6 | EUCTR2019-003395-39-AT (EUCTR) | 11/03/2020 | 08/01/2020 | 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) | 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 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 | 30 | Phase 3 | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany | ||
| 7 | EUCTR2019-003395-39-FR (EUCTR) | 09/03/2020 | 17/12/2019 | 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) | 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 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 | 30 | Phase 3 | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Singapore;Germany | ||
| 8 | EUCTR2019-002755-42-ES (EUCTR) | 07/02/2020 | 06/02/2020 | 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). 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;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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 2 | Canada;Belgium;United States;Poland;United Kingdom;Australia;France;Spain | ||
| 9 | EUCTR2019-003395-39-HU (EUCTR) | 05/02/2020 | 07/01/2020 | 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) | 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 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 | 30 | Phase 3 | Colombia;Argentina;Singapore;Hungary;United States;United Kingdom;Lebanon;Canada;Austria;Turkey;Belgium;Brazil;Poland;Italy;Mexico;France;Germany | ||
| 10 | EUCTR2019-001211-22-DE (EUCTR) | 15/11/2019 | 03/06/2019 | 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: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | 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 | ||
| 11 | EUCTR2019-001211-22-AT (EUCTR) | 13/11/2019 | 11/07/2019 | 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: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | 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;Belgium;Brazil;Poland;Singapore;Germany | ||
| 12 | EUCTR2019-001211-22-GB (EUCTR) | 29/10/2019 | 30/05/2019 | 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 | Progressive Familial Intrahepatic Cholestasis (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 that may 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: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | United States;Saudi Arabia;Lebanon;Turkey;Austria;Chile;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany | ||
| 13 | EUCTR2019-001211-22-FR (EUCTR) | 23/10/2019 | 19/06/2019 | 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 that may ultimately lead to the need for liver transplantation. Itch is a common 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 (formerly SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Saudi Arabia;Lebanon;Turkey;Austria;Chile;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany | ||
| 14 | EUCTR2019-001211-22-HU (EUCTR) | 15/08/2019 | 20/06/2019 | 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 that may ultimately lead to the need for liver transplantation. Itch is a common 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 (formerly SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany | ||
| 15 | EUCTR2013-003833-14-GB (EUCTR) | 30/01/2014 | 14/11/2013 | AN OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001 IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS | OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001, AN APICALSODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS - INDIGO STUDY | 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 that may ultimately lead to the need for liver transplantation. Itch is a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice. MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: LUM001 INN or Proposed INN: maralixibat chloride | Mirum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;Poland;United Kingdom | ||
| 16 | EUCTR2019-001211-22-PL (EUCTR) | 28/06/2019 | 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 that may ultimately lead to the need for liver transplantation. Itch is a common 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: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Brazil;Singapore;Germany | |||
| 17 | EUCTR2019-001211-22-BE (EUCTR) | 22/07/2019 | 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: MRX Drug Substance (formerly Maralixibat , SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | NA | Female: yes Male: yes | 30 | Phase 3 | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany | |||
| 18 | EUCTR2020-004628-40-PL (EUCTR) | 20/01/2021 | Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. | Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants with Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. - Maralixibat Infant Safety Evaluation (RISE). | Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome. 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;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 | 20 | Phase 2 | France;United States;Mexico;Belgium;Brazil;Poland;United Kingdom | |||
| 19 | EUCTR2019-003395-39-DE (EUCTR) | 19/11/2019 | 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) | 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 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 | 30 | Phase 3 | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany | |||
| 20 | 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). 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;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 | 53 | Phase 2 | Canada;Belgium;United States;Poland;United Kingdom;Australia;France;Spain | |||
| 21 | 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 | |||
| 22 | EUCTR2019-003395-39-BE (EUCTR) | 10/12/2019 | 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) | 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 that may ultimately lead to the need for liver transplantation. Itch is a common 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 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 | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany | |||
| 23 | EUCTR2019-003395-39-PL (EUCTR) | 11/12/2019 | 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) | 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 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 | 30 | Phase 3 | United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany | |||
| 24 | EUCTR2013-003833-14-PL (EUCTR) | 19/08/2014 | AN OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001 IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS | OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001, AN APICALSODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS - INDIGO STUDY | 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 that may ultimately lead to the need for liver transplantation. Itch is a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice. MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: LUM001 INN or Proposed INN: maralixibat chloride | Mirum Pharmaceuticals, LLC. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;Poland;United Kingdom |