297. アラジール症候群 Alagille syndrome Clinical trials / Disease details
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
Showing 1 to 10 of 15 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 | NCT04530994 (ClinicalTrials.gov) | August 24, 2020 | 25/8/2020 | A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS) A MaralixibatExpanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille ... | A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome A MaralixibatExpanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille ... | Alagille Syndrome | Drug: maralixibat | Mirum Pharmaceuticals, Inc. | Clinigen, Inc. | Approved for marketing | 12 Months | N/A | All | United States | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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. The purpose of this study is to evaluate a drug (LUM001 also known as SHP625) that may help treat th ... | 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 Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Peri ... | 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] Alagille syndrome (ALGS) is an autosomal dominant with variable penetration genetic multisystem diso ... | 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 | ||
| 9 | NCT02117713 (ClinicalTrials.gov) | March 16, 2015 | 16/4/2014 | An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatm ... | A Multicenter 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 A Multicenter Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Eff ... | Alagille Syndrome | Drug: LUM001 (Maralixibat) | Mirum Pharmaceuticals, Inc. | Lumena Pharmaceuticals, Inc.;Childhood Liver Disease Research and Education Network | Completed | 1 Year | 18 Years | All | 34 | Phase 2 | United States;Canada |
| 10 | NCT02047318 (ClinicalTrials.gov) | December 20, 2013 | 23/1/2014 | An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Subjects With Alagille Syndrome (ALGS) An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatm ... | A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001 Also Known as Maralixibat (MRX), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Eff ... | Alagille Syndrome | Drug: LUM001 (Maralixibat) | Mirum Pharmaceuticals, Inc. | NULL | Completed | 12 Months | 18 Years | All | 19 | Phase 2 | United Kingdom |