338. 進行性家族性肝内胆汁うっ滞症 Progressive familial intrahepatic cholestasis Clinical trials / Disease details
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
Showing 1 to 10 of 29 diseases
| 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 | NCT04729751 (ClinicalTrials.gov) | September 9, 2021 | 25/1/2021 | A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). A Study to Evaluate the Safety and Tolerability of Maralixibatin Infant Participants With Cholestati ... | 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 Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibatin the Treatment of ... | Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome;Cholestatic Liver Disease | Drug: Maralixibat | Mirum Pharmaceuticals, Inc. | NULL | Recruiting | 0 Days | 364 Days | All | 12 | Phase 2 | United States;Belgium;Brazil;France;Mexico;Poland;United Kingdom |
| 2 | 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. Clinical study to Evaluate the Safety and Tolerability of Maralixibatin the Treatment of Infants wit ... | 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). Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibatin the Treatment of ... | 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] Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Synd ... | 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 Product Name: Maralixibat(formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBATCHLORIDE Product N ... | Mirum Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | France;Poland;Belgium;United Kingdom | ||
| 3 | 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. Clinical study to Evaluate the Safety and Tolerability of Maralixibatin the Treatment of Infants wit ... | 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). Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibatin the Treatment of ... | 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] Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome MedDRA version: 20.0;Level: PT;C ... | 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 Product Name: Maralixibat(formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBATCHLORIDE Product N ... | 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 | ||
| 4 | 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). Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibatin Subjects with Progres ... | 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 MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibati ... | 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] In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bi ... | 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 Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibatcloruro Product N ... | 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 United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Arge ... | ||
| 5 | 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). Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibatin Subjects with Progres ... | 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) MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibati ... | 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] In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bi ... | 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 Product Name: Maralixibat INN or Proposed INN: MARALIXIBATCHLORIDE Product Name: Maralixibat INN or Pro ... | 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 United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Arge ... | ||
| 6 | 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. MERGE: MaralixibatExtension Safety Study Providing Long-term Treatment to Subjects with Cholestatic ... | 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. MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter I ... | 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] Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease inc ... | 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 Product Name: Maralixibat(formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBATCHLORIDE Product N ... | 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 | ||
| 7 | 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. MERGE: MaralixibatExtension Safety Study Providing Long-term Treatment to Subjects with Cholestatic ... | 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. MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter I ... | 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] Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease inc ... | 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 Product Name: Maralixibat(formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBATCHLORIDE Product N ... | 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 | ||
| 8 | 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). Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibatin Subjects with Progres ... | 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) MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibati ... | 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] In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bi ... | 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 Product Name: Maralixibat INN or Proposed INN: MARALIXIBATCHLORIDE Product Name: Maralixibat INN or Pro ... | 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 United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Arge ... | ||
| 9 | 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). Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibatin Subjects with Progres ... | 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) MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibati ... | 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] In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bi ... | 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 Product Name: Maralixibat INN or Proposed INN: MARALIXIBATCHLORIDE Product Name: Maralixibat INN or Pro ... | 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 United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Arge ... | ||
| 10 | 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. MERGE: MaralixibatExtension Safety Study Providing Long-term Treatment to Subjects with Cholestatic ... | 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. MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter I ... | 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] Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease inc ... | 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 Product Name: Maralixibat(formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBATCHLORIDE Product N ... | 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 |