297. アラジール症候群 [臨床試験数:26,薬物数:16(DrugBank:8),標的遺伝子数:2,標的パスウェイ数:2

Searched query = "Alagille syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
8 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-002755-42-FR24/03/202016/01/2020MERGE: 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: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
53Phase 2United States;France;Canada;Spain;Poland;Belgium;Australia;United Kingdom
2EUCTR2019-002755-42-GB19/03/202023/12/2019MERGE: 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: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
53Phase 2United States;France;Canada;Spain;Poland;Belgium;Australia;United Kingdom
3EUCTR2013-005373-43-GB21/04/201506/02/2015The 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: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Belgium;Poland;Spain;Australia;United Kingdom
4EUCTR2013-003832-54-GB28/11/201309/12/2013A 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 SYNDROMEA 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: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2United Kingdom
5EUCTR2020-004628-40-BE15/12/2020Clinical 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: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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 IncNULLNAFemale: yes
Male: yes
12Phase 2France;Poland;Belgium;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-002755-42-PL08/01/2020MERGE: 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: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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.NULLNAFemale: yes
Male: yes
53Phase 2France;United States;Canada;Belgium;Spain;Poland;Australia;United Kingdom
7EUCTR2020-004628-40-FR16/12/2020Clinical 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: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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 IncNULLNAFemale: yes
Male: yes
12Phase 2France;Poland;Belgium;United Kingdom
8EUCTR2013-005373-43-PL09/07/2014The 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: 20.0Level: PTClassification code 10053870Term: Alagille syndromeSystem 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Belgium;Spain;Poland;Australia;United Kingdom