297. Alagille syndrome
[
13 clinical trials,
13 drugs（DrugBank:
8 drugs),
0 target gene / 0 target pathway ]
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.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03082937 | January 31, 2017 | 16 December 2017 | An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects | An 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 Subjects | Orphan Cholestatic Liver Diseases;Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome;Primary Biliary Cirrhosis | Drug: 3 mg [14C]-A4250 capsule | Albireo | Not recruiting | 30 Years | 65 Years | Male | 6 | Phase 1 | United Kingdom | |
| 2 | JPRN-jRCTs041180088 | 01/12/2016 | 10 September 2019 | Influence of fatty acid metabolism for clinical course of biliary atresia | The difference of the profile of fatty acids and eicosanoids in clinical course and the effect to the prognosis by collection of biliary atresia | biliary atresia, neonatal hepatitis, Alagille syndrome, PFIC, etc. | oral administration of 30(+/- 10)mg/kg/day of eicosapentaenoic acid | Wataru Sumida | Hiroo Uchida | Recruiting | Not applicable | Not applicable | Both | 30 | N/A | none |
| 3 | EUCTR2015-000906-20-GB | 13/05/2015 | 28 September 2015 | 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) | 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 Inc | Authorised | Female: yes Male: yes | 120 | United States;Canada;Australia;United Kingdom | ||||
| 4 | NCT02117713 | March 16, 2015 | 1 April 2019 | 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 | 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 | Alagille Syndrome | Drug: LUM001 | Mirum Pharmaceuticals, Inc. | Lumena Pharmaceuticals, Inc.;Childhood Liver Disease Research and Education Network | Not recruiting | 1 Year | 18 Years | All | 36 | Phase 2 | United States;Canada |
| 5 | NCT02057692 | November 24, 2014 | 1 April 2019 | Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome | The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease | Alagille Syndrome | Drug: LUM001;Drug: Placebo | Mirum Pharmaceuticals, Inc. | Childhood Liver Disease Research and Education Network | Not recruiting | 12 Months | 18 Years | All | 37 | Phase 2 | United States;Canada |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT02160782 | October 28, 2014 | 1 April 2019 | Safety 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 Syndrome | Alagille Syndrome | Drug: LUM001;Drug: Placebo | Mirum Pharmaceuticals, Inc. | Not recruiting | 12 Months | 18 Years | All | 30 | Phase 2 | Australia;Belgium;France;Poland;Spain;United Kingdom;Canada;Germany | |
| 7 | EUCTR2013-005373-43-ES | 05/08/2014 | 11 August 2014 | The 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 - ICONIC | Alagille 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 Inc | Authorised | Female: yes Male: yes | 30 | Canada;Spain;Australia | ||||
| 8 | JPRN-UMIN000012782 | 2014/02/01 | 2 April 2019 | Efficacy 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 University | Not Recruiting | Not applicable | Not applicable | Male and Female | 2 | Not selected | Japan | ||
| 9 | EUCTR2013-003832-54-GB | 28/11/2013 | 30 April 2018 | 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 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: 20.0 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. | Authorised | Female: yes Male: yes | 18 | Phase 2 | United Kingdom | |||
| 10 | NCT02963077 | July 2013 | 28 November 2016 | A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384 | A 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 Subjects | Orphan Cholestatic Liver Diseases;Primary Biliary Cirrhosis;Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome | Drug: A4250;Drug: CRC (A3384);Drug: Questran;Drug: Placebo | Albireo | Not recruiting | 18 Years | 60 Years | Both | 94 | Phase 1 | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2012-005346-38-GB | 11/06/2013 | 4 August 2015 | A 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 SYNDROME | A 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 - IMAGO | 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: 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 Recruiting | Female: yes Male: yes | United Kingdom | |||||
| 12 | JPRN-UMIN000003802 | 2010/04/01 | 2 April 2019 | Efficacy 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 1-4months | Saiseikai Yokohama City Tobu Hospital | Laboratory of Molecular Pharmacokinetics, Graduate School of Pharmaceutical Sciences, The University of Tokyo (Tokyo) | Recruiting | Not applicable | Not applicable | Male and Female | 15 | Not selected | Japan | |
| 13 | NCT00007033 | October 2000 | 19 February 2015 | Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease | Alagille Syndrome;Cholestasis;Biliary Atresia | Drug: magnesium gluconate;Drug: magnesium sulfate | National Center for Research Resources (NCRR) | Children's Hospital Medical Center, Cincinnati | Not recruiting | 3 Years | 18 Years | Both | 25 | N/A | United States |