297. アラジール症候群 Alagille syndrome Clinical trials / Disease details


臨床試験数 : 43 薬物数 : 19 - (DrugBank : 9) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2

  
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2021-000996-36-NL
(EUCTR)
04/03/202202/09/2021A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Malaysia;Belgium;Poland;Netherlands;Germany;New Zealand
2EUCTR2021-000996-36-IT
(EUCTR)
09/11/202127/01/2022A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT) - NA Alagille Syndrome
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: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250-015
Product Name: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
ALBIREO ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Germany;Netherlands;New Zealand
3NCT04729751
(ClinicalTrials.gov)
September 9, 202125/1/2021A 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).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 SyndromeProgressive Familial Intrahepatic Cholestasis;Alagille Syndrome;Cholestatic Liver DiseaseDrug: MaralixibatMirum Pharmaceuticals, Inc.NULLRecruitingN/A364 DaysAll12Phase 2United States;Belgium;France;Poland;United Kingdom
4NCT05035030
(ClinicalTrials.gov)
September 3, 202113/8/2021Long-term Safety and Efficacy of Odevixibat in Patients With Alagille SyndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT)Alagille SyndromeDrug: OdevixibatAlbireoNULLEnrolling by invitationN/AN/AAll63Phase 3United States;France;Poland;United Kingdom
5EUCTR2020-004011-28-DE
(EUCTR)
21/07/202115/04/2021An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeA Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Germany;Netherlands;New Zealand
6EUCTR2020-004011-28-NL
(EUCTR)
08/06/202116/12/2020An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeA Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 3United States;France;Canada;Poland;Belgium;Denmark;Australia;Germany;Netherlands;United Kingdom;Italy
7EUCTR2020-004628-40-FR
(EUCTR)
25/03/202116/12/2020Clinical 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: 20.0;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]
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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 2France;Poland;Belgium;United Kingdom
8NCT04674761
(ClinicalTrials.gov)
March 19, 202110/12/2020Efficacy and Safety of Odevixibat in Patients With Alagille SyndromeA Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)Alagille SyndromeDrug: Odevixibat;Drug: PlaceboAlbireoNULLRecruitingN/AN/AAll63Phase 3United States;Belgium;Canada;France;Germany;Israel;Italy;Malaysia;Netherlands;New Zealand;Poland;Turkey;United Kingdom
9EUCTR2020-004011-28-IT
(EUCTR)
24/02/202120/05/2021An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeA Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT) - NA Alagille Syndrome
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: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: [A4250]
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
ALBIREO ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 3France;United States;Canada;Poland;Belgium;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy
10EUCTR2020-004011-28-FR
(EUCTR)
09/02/202125/11/2020An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeA Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
63Phase 3United States;France;Canada;Poland;Belgium;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy
11EUCTR2020-004628-40-BE
(EUCTR)
29/01/202115/12/2020Clinical 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). 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]
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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 2France;Poland;Belgium;United Kingdom
12NCT04530994
(ClinicalTrials.gov)
August 24, 202025/8/2020A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille SyndromeAlagille SyndromeDrug: maralixibatMirum Pharmaceuticals, Inc.Clinigen, Inc.Approved for marketing12 MonthsN/AAllUnited States
13EUCTR2019-002755-42-FR
(EUCTR)
24/03/202016/01/2020MERGE: 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: 20.0;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]
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
14EUCTR2019-002755-42-GB
(EUCTR)
19/03/202023/12/2019MERGE: 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: 20.0;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]
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
15EUCTR2019-002755-42-ES
(EUCTR)
07/02/202006/02/2020MERGE: 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: 20.0;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]
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 finished Female: yes
Male: yes
53 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Canada;Poland;Belgium;Spain;Australia;United Kingdom
16NCT03082937
(ClinicalTrials.gov)
January 31, 201727/2/2017An 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 capsuleAlbireoNULLCompleted30 Years65 YearsMale6Phase 1United Kingdom
17JPRN-jRCTs041180088
01/12/201612/03/2019Influence 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 atresia biliary atresia, neonatal hepatitis, Alagille syndrome, PFIC, etc.oral administration of 30(+/- 10)mg/kg/day of eicosapentaenoic acidSumida WataruUchida HirooRecruitingNot applicableNot applicableBoth30N/AJapan
18EUCTR2015-000906-20-GB
(EUCTR)
13/05/201501/04/2015An 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
INN or Proposed INN: LUM001
Shire Human Genetic Therapies IncNULLNot Recruiting Female: yes
Male: yes
120 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Australia;United Kingdom
19EUCTR2013-005373-43-GB
(EUCTR)
21/04/201506/02/2015The 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: 20.0;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
INN or Proposed INN: Maralixibat chloride
Mirum Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Belgium;Poland;Spain;Australia;United Kingdom
20EUCTR2013-005373-43-BE
(EUCTR)
18/03/201505/02/2015The 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: 18.1;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
INN or Proposed INN: LUM001
Lumena Pharmaceuticals LLCNULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Poland;Spain;Belgium;Australia;United Kingdom
21NCT02117713
(ClinicalTrials.gov)
March 16, 201516/4/2014An 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: LUM001 (Maralixibat)Mirum Pharmaceuticals, Inc.Lumena Pharmaceuticals, Inc.;Childhood Liver Disease Research and Education NetworkCompleted1 Year18 YearsAll34Phase 2United States;Canada
22NCT02057692
(ClinicalTrials.gov)
November 24, 20145/2/2014Evaluation 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 NetworkCompleted12 Months18 YearsAll37Phase 2United States;Canada
23NCT02160782
(ClinicalTrials.gov)
October 28, 20149/6/2014Safety 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.NULLCompleted12 Months18 YearsAll31Phase 2Australia;Belgium;France;Poland;Spain;United Kingdom;Canada;Germany
24EUCTR2013-005373-43-ES
(EUCTR)
05/08/201422/05/2014The 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 - 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
INN or Proposed INN: LUM001
Lumena Pharmaceuticals IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Canada;Belgium;Poland;Spain;Australia;United Kingdom
25JPRN-UMIN000012782
2014/02/0101/02/2014Efficacy 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 UniversityNULLPendingNot applicableNot applicableMale and Female2Not selectedJapan
26NCT02047318
(ClinicalTrials.gov)
December 20, 201323/1/2014An 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)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 SyndromeAlagille SyndromeDrug: LUM001 (Maralixibat)Mirum Pharmaceuticals, Inc.NULLCompleted12 Months18 YearsAll19Phase 2United Kingdom
27EUCTR2013-003832-54-GB
(EUCTR)
28/11/201309/12/2013A 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: 20.0;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
INN or Proposed INN: maralixibat chloride
Mirum Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2United Kingdom
28NCT01903460
(ClinicalTrials.gov)
August 201316/7/2013Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in 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 SyndromeAlagille SyndromeDrug: LUM001;Drug: PlaceboMirum Pharmaceuticals, Inc.NULLCompleted12 Months18 YearsAll20Phase 2United Kingdom
29NCT02963077
(ClinicalTrials.gov)
July 20131/11/2016A 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: PlaceboAlbireoNULLCompleted18 Years60 YearsBoth94Phase 1NULL
30EUCTR2012-005346-38-GB
(EUCTR)
11/06/201307/05/2013A 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 - 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 diseasethat 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
INN or Proposed INN: LUM001
Lumena Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
United Kingdom
31JPRN-UMIN000003802
2010/04/0122/06/2010Efficacy 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
32NCT00007033
(ClinicalTrials.gov)
October 20006/12/2000Study 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, CincinnatiCompleted3 Years18 YearsBoth25N/AUnited States
33EUCTR2019-002755-42-BE
(EUCTR)
11/12/2019MERGE: 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) 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]
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
109Phase 2France;United States;Canada;Spain;Poland;Belgium;Australia;United Kingdom
34EUCTR2021-000996-36-PL
(EUCTR)
01/09/2021A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLNAFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Poland;Malaysia;Belgium;Germany;Netherlands;New Zealand
35EUCTR2021-000996-36-DE
(EUCTR)
02/11/2021A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLNAFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Malaysia;Belgium;Poland;Germany;Netherlands;New Zealand
36EUCTR2020-004011-28-PL
(EUCTR)
07/12/2020An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeA Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLNAFemale: yes
Male: yes
60Phase 3United Kingdom;Italy;United States;France;Canada;Belgium;Poland;Denmark;Australia;Netherlands;Germany
37EUCTR2021-000996-36-FR
(EUCTR)
25/06/2021A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLNAFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Malaysia;Belgium;Poland;Germany;Netherlands;New Zealand
38EUCTR2019-002755-42-PL
(EUCTR)
08/01/2020MERGE: 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) 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]
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
109Phase 2France;United States;Canada;Spain;Belgium;Poland;Australia;United Kingdom
39EUCTR2013-005373-43-FR
(EUCTR)
10/07/2015The 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: 18.0;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
INN or Proposed INN: LUM001
Lumena Pharmaceuticals IncNULLNot RecruitingFemale: yes
Male: yes
30Phase 2France;Canada;Belgium;Poland;Spain;Australia;United Kingdom
40EUCTR2020-004628-40-PL
(EUCTR)
20/01/2021Clinical 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). 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]
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;Belgium;Poland;United Kingdom
41EUCTR2013-005373-43-PL
(EUCTR)
09/07/2014The 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: 20.0;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
INN or Proposed INN: Maralixibat chloride
Mirum Pharmaceuticals,Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 2United Kingdom;Australia;Poland;Spain;Belgium;France
42EUCTR2020-004011-28-BE
(EUCTR)
19/01/2021An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeA Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLNAFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Malaysia;Australia;Denmark;Germany;Netherlands;New Zealand
43EUCTR2021-000996-36-BE
(EUCTR)
01/09/2021A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndromeAn Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT) Alagille Syndrome
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: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Product Name: Odevixibat
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250
Albireo ABNULLNAFemale: yes
Male: yes
60Phase 3United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Germany;Netherlands;New Zealand