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 11 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 | 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 Maralixibat in the Treatment of Infants wi ... | 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 Maralixibat in 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 | ||
| 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. Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants wi ... | 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 Maralixibat in 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 | ||
| 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. MERGE: Maralixibat Extension 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 | ||
| 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. MERGE: Maralixibat Extension 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 | ||
| 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. MERGE: Maralixibat Extension 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 | ||
| 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. 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 | ||
| 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 CLINICAL STUDY TO EVALUATE THE SAFETY AND EFFICACY OF LUM001, AN AGENT THAT INHIBITS B ... | 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 A MULTICENTRE EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND DURABILITY OF THE THERAPEUTIC EFF ... | 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] Alagille syndrome (ALGS). This is an example of cholestatic liver disease in children. In patients w ... | 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-BE (EUCTR) | 11/12/2019 | MERGE: Maralixibat Extension Safety Study Providing Long-term Treatment to Subjects with Cholestatic Liver Disease. MERGE: Maralixibat Extension 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) 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] 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 | NA | Female: yes Male: yes | 109 | Phase 2 | France;United States;Canada;Spain;Poland;Belgium;Australia;United Kingdom | |||
| 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. 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;Spain;Poland;Australia;United Kingdom | |||
| 10 | 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. Clinical study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants wi ... | 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 Maralixibat in 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; ... | 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 | NA | Female: yes Male: yes | 20 | Phase 2 | France;United States;Mexico;Belgium;Brazil;Poland;United Kingdom |