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 297. アラジール症候群 [臨床試験数:13,薬物数:13(DrugBank:8),標的遺伝子数:0,標的パスウェイ数:0] 

Searched query = "Alagille syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03082937January 31, 201716 December 2017An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy SubjectsAn Open Label, Single-Dose, Single Period Study Designed to Assess the Mass Balance Recovery, Metabolite Profile and Metabolite Identification of [14C]-A4250 in Healthy Male SubjectsOrphan Cholestatic Liver Diseases;Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome;Primary Biliary CirrhosisDrug: 3 mg [14C]-A4250 capsuleAlbireoNot recruiting30 Years65 YearsMale6Phase 1United Kingdom
2JPRN-jRCTs04118008801/12/201610 September 2019Influence of fatty acid metabolism for clinical course of biliary atresiaThe difference of the profile of fatty acids and eicosanoids in clinical course and the effect to the prognosis by collection of biliary atresiabiliary atresia, neonatal hepatitis, Alagille syndrome, PFIC, etc.oral administration of 30(+/- 10)mg/kg/day of eicosapentaenoic acidWataru SumidaHiroo UchidaRecruitingNot applicableNot applicableBoth30N/Anone
3EUCTR2015-000906-20-GB13/05/201528 September 2015An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
MedDRA version: 17.1 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1 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
Pharmaceutical Form: Oral solution
INN or Proposed INN: LUM001
CAS Number: 228113-66-4
Current Sponsor code: LUM001
Concentration unit: µg/kg microgram(s)/kilogram
Concentration type: range
Concentration number: 14-400
Shire Human Genetic Therapies IncAuthorisedFemale: yes
Male: yes
120United States;Canada;Australia;United Kingdom
4NCT02117713March 16, 20151 April 2019An 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 SyndromeA 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 SyndromeAlagille SyndromeDrug: LUM001Mirum Pharmaceuticals, Inc.Lumena Pharmaceuticals, Inc.;Childhood Liver Disease Research and Education NetworkNot recruiting1 Year18 YearsAll36Phase 2United States;Canada
5NCT02057692November 24, 20141 April 2019Evaluation of LUM001 in the Reduction of Pruritus in Alagille SyndromeThe Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver DiseaseAlagille SyndromeDrug: LUM001;Drug: PlaceboMirum Pharmaceuticals, Inc.Childhood Liver Disease Research and Education NetworkNot recruiting12 Months18 YearsAll37Phase 2United States;Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02160782October 28, 20141 April 2019Safety and Efficacy Study of LUM001 With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)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 SyndromeAlagille SyndromeDrug: LUM001;Drug: PlaceboMirum Pharmaceuticals, Inc.Not recruiting12 Months18 YearsAll30Phase 2Australia;Belgium;France;Poland;Spain;United Kingdom;Canada;Germany
7EUCTR2013-005373-43-ES05/08/201411 August 2014The purpose of this study is to evaluate a drug (LUM001) that may help treat the liver and control itching in Alagille Syndrome. In this study, all children who are eligible to enrol will take study drug for 18 weeks, followed by a 4 week period where they will either take LUM001 or placebo. After this 4 week period, all patients will go back on active study drug treatment for the remaining 26 weeks.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 - ICONICAlagille Syndrome
MedDRA version: 17.0 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
Pharmaceutical Form: Powder and solvent for oral solution
INN or Proposed INN: LUM001
CAS Number: 228113-66-4
Current Sponsor code: LUM001
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: range
Concentration number: 0.05-60
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
Lumena Pharmaceuticals IncAuthorisedFemale: yes
Male: yes
30Canada;Spain;Australia
8JPRN-UMIN0000127822014/02/012 April 2019Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.Group A; phenylbutyrate 6g (Child 100mg/kg)/day *7days
Group B; phenylbutyrate 6g (Child 100mg/kg)/day *3days and 12g (Child 200mg/kg)/day *4days
Group C; phenylbutyrate 6g (Child 100mg/kg)/day *1day, phenylbutyrate 12g (Child 200mg/kg)/day *2days and phenylbutyrate 21g (Child 300mg/kg)/day *4days
Juntendo UniversityNot RecruitingNot applicableNot applicableMale and Female2Not selectedJapan
9EUCTR2013-003832-54-GB28/11/201330 April 2018A 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 STUDYAlagille 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
Pharmaceutical Form: Powder and solvent for oral solution
INN or Proposed INN: maralixibat chloride
CAS Number: 228113-66-4
Current Sponsor code: LUM001
Concentration unit: µg/kg microgram(s)/kilogram
Concentration type: up to
Concentration number: 280-
Lumena Pharmaceuticals, LLC.AuthorisedFemale: yes
Male: yes
18Phase 2United Kingdom
10NCT02963077July 201328 November 2016A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384A Phase I, Double-Blind Single and Multiple Ascending Dose Study to Assess Safety and Pharmacokinetics of A4250 as Monotherapy, and in Combination With Colonic Release Cholestyramine (A3384) or Commercially Available Cholestyramine (Questran™) in Healthy SubjectsOrphan Cholestatic Liver Diseases;Primary Biliary Cirrhosis;Progressive Familial Intrahepatic Cholestasis;Alagille SyndromeDrug: A4250;Drug: CRC (A3384);Drug: Questran;Drug: PlaceboAlbireoNot recruiting18 Years60 YearsBoth94Phase 1
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2012-005346-38-GB11/06/20134 August 2015A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED 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 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF LUM001, AN APICAL SODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PAEDIATRIC PATIENTS WITH ALAGILLE SYNDROME - IMAGOAlagille 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: 14.1 Level: PT Classification code 10053870 Term: Alagille syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: LUM001
Pharmaceutical Form: Powder and solvent for oral solution
INN or Proposed INN: LUM001
Current Sponsor code: LUM001
Concentration unit: µg/kg microgram(s)/kilogram
Concentration type: range
Concentration number: 14-280
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
Lumena Pharmaceuticals, Inc.Not RecruitingFemale: yes
Male: yes
United Kingdom
12JPRN-UMIN0000038022010/04/012 April 2019Efficacy and safety of 4-phenylbutyrate in refractory cholestatic disease including progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.Progressive familial intrahepatic cholestasis, primary biliary cirrhosis, primary sclerosing cholangitis and Alagille syndrome.phenylbutyrate(Child 250mg/kg/day)for 1-4months : phenylbutyrate(Child 350mg/kg/day)for 1-4months : phenylbutyrate(Child 500mg/kg/day)for 1-4monthsSaiseikai Yokohama City Tobu HospitalLaboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo)RecruitingNot applicableNot applicableMale and Female15Not selectedJapan
13NCT00007033October 200019 February 2015Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver DiseaseAlagille Syndrome;Cholestasis;Biliary AtresiaDrug: magnesium gluconate;Drug: magnesium sulfateNational Center for Research Resources (NCRR)Children's Hospital Medical Center, CincinnatiNot recruiting3 Years18 YearsBoth25N/AUnited States

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