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. | 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 | EUCTR2021-000996-36-NL (EUCTR) | 04/03/2022 | 02/09/2021 | A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | An 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 AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Malaysia;Belgium;Poland;Netherlands;Germany;New Zealand | ||
| 2 | EUCTR2021-000996-36-IT (EUCTR) | 09/11/2021 | 27/01/2022 | A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | An 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 AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Germany;Netherlands;New Zealand | ||
| 3 | NCT04729751 (ClinicalTrials.gov) | September 9, 2021 | 25/1/2021 | A 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 Syndrome | Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome;Cholestatic Liver Disease | Drug: Maralixibat | Mirum Pharmaceuticals, Inc. | NULL | Recruiting | N/A | 364 Days | All | 12 | Phase 2 | United States;Belgium;France;Poland;United Kingdom |
| 4 | NCT05035030 (ClinicalTrials.gov) | September 3, 2021 | 13/8/2021 | Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome | An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT) | Alagille Syndrome | Drug: Odevixibat | Albireo | NULL | Enrolling by invitation | N/A | N/A | All | 63 | Phase 3 | United States;France;Poland;United Kingdom |
| 5 | EUCTR2020-004011-28-DE (EUCTR) | 21/07/2021 | 15/04/2021 | An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | A 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 AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Germany;Netherlands;New Zealand | ||
| 6 | EUCTR2020-004011-28-NL (EUCTR) | 08/06/2021 | 16/12/2020 | An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | A 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 AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 3 | United States;France;Canada;Poland;Belgium;Denmark;Australia;Germany;Netherlands;United Kingdom;Italy | ||
| 7 | 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. | 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 Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | France;Poland;Belgium;United Kingdom | ||
| 8 | NCT04674761 (ClinicalTrials.gov) | March 19, 2021 | 10/12/2020 | Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome | A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT) | Alagille Syndrome | Drug: Odevixibat;Drug: Placebo | Albireo | NULL | Recruiting | N/A | N/A | All | 63 | Phase 3 | United States;Belgium;Canada;France;Germany;Israel;Italy;Malaysia;Netherlands;New Zealand;Poland;Turkey;United Kingdom |
| 9 | EUCTR2020-004011-28-IT (EUCTR) | 24/02/2021 | 20/05/2021 | An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | A 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 AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 3 | France;United States;Canada;Poland;Belgium;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy | ||
| 10 | EUCTR2020-004011-28-FR (EUCTR) | 09/02/2021 | 25/11/2020 | An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | A 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 AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 63 | Phase 3 | United States;France;Canada;Poland;Belgium;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy | ||
| 11 | 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. | 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 Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | France;Poland;Belgium;United Kingdom | ||
| 12 | NCT04530994 (ClinicalTrials.gov) | August 24, 2020 | 25/8/2020 | A 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 Syndrome | Alagille Syndrome | Drug: maralixibat | Mirum Pharmaceuticals, Inc. | Clinigen, Inc. | Approved for marketing | 12 Months | N/A | All | United States | ||
| 13 | 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. | 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. | 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 | ||
| 14 | 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. | 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. | 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 | ||
| 15 | 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. | 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. | NULL | Authorised-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): no | France;United States;Canada;Poland;Belgium;Spain;Australia;United Kingdom | ||
| 16 | NCT03082937 (ClinicalTrials.gov) | January 31, 2017 | 27/2/2017 | 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 | NULL | Completed | 30 Years | 65 Years | Male | 6 | Phase 1 | United Kingdom |
| 17 | JPRN-jRCTs041180088 | 01/12/2016 | 12/03/2019 | 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 | Sumida Wataru | Uchida Hiroo | Recruiting | Not applicable | Not applicable | Both | 30 | N/A | Japan |
| 18 | EUCTR2015-000906-20-GB (EUCTR) | 13/05/2015 | 01/04/2015 | 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 INN or Proposed INN: LUM001 | Shire Human Genetic Therapies Inc | NULL | Not 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): no | United States;Canada;Australia;United Kingdom | ||
| 19 | 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. | 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. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Belgium;Poland;Spain;Australia;United Kingdom | ||
| 20 | EUCTR2013-005373-43-BE (EUCTR) | 18/03/2015 | 05/02/2015 | 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: 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 LLC | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Poland;Spain;Belgium;Australia;United Kingdom | ||
| 21 | NCT02117713 (ClinicalTrials.gov) | March 16, 2015 | 16/4/2014 | 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 (Maralixibat) | Mirum Pharmaceuticals, Inc. | Lumena Pharmaceuticals, Inc.;Childhood Liver Disease Research and Education Network | Completed | 1 Year | 18 Years | All | 34 | Phase 2 | United States;Canada |
| 22 | NCT02057692 (ClinicalTrials.gov) | November 24, 2014 | 5/2/2014 | 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 | Completed | 12 Months | 18 Years | All | 37 | Phase 2 | United States;Canada |
| 23 | NCT02160782 (ClinicalTrials.gov) | October 28, 2014 | 9/6/2014 | 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. | NULL | Completed | 12 Months | 18 Years | All | 31 | Phase 2 | Australia;Belgium;France;Poland;Spain;United Kingdom;Canada;Germany |
| 24 | EUCTR2013-005373-43-ES (EUCTR) | 05/08/2014 | 22/05/2014 | 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 INN or Proposed INN: LUM001 | Lumena Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Canada;Belgium;Poland;Spain;Australia;United Kingdom | ||
| 25 | JPRN-UMIN000012782 | 2014/02/01 | 01/02/2014 | 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 | NULL | Pending | Not applicable | Not applicable | Male and Female | 2 | Not selected | Japan | |
| 26 | NCT02047318 (ClinicalTrials.gov) | December 20, 2013 | 23/1/2014 | An 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 Syndrome | Alagille Syndrome | Drug: LUM001 (Maralixibat) | Mirum Pharmaceuticals, Inc. | NULL | Completed | 12 Months | 18 Years | All | 19 | Phase 2 | United Kingdom |
| 27 | 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 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. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | United Kingdom | ||
| 28 | NCT01903460 (ClinicalTrials.gov) | August 2013 | 16/7/2013 | Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in 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 | Alagille Syndrome | Drug: LUM001;Drug: Placebo | Mirum Pharmaceuticals, Inc. | NULL | Completed | 12 Months | 18 Years | All | 20 | Phase 2 | United Kingdom |
| 29 | NCT02963077 (ClinicalTrials.gov) | July 2013 | 1/11/2016 | 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 | NULL | Completed | 18 Years | 60 Years | Both | 94 | Phase 1 | NULL |
| 30 | EUCTR2012-005346-38-GB (EUCTR) | 11/06/2013 | 07/05/2013 | 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 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. | NULL | Not Recruiting | Female: yes Male: yes | United Kingdom | ||||
| 31 | JPRN-UMIN000003802 | 2010/04/01 | 22/06/2010 | 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 | |
| 32 | NCT00007033 (ClinicalTrials.gov) | October 2000 | 6/12/2000 | 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 | Completed | 3 Years | 18 Years | Both | 25 | N/A | United States | |
| 33 | 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. | 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 Inc | NULL | NA | Female: yes Male: yes | 12 | Phase 2 | France;Belgium;Poland;United Kingdom | |||
| 34 | EUCTR2020-004011-28-BE (EUCTR) | 19/01/2021 | An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | A 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 AB | NULL | NA | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Malaysia;Australia;Denmark;Germany;Netherlands;New Zealand | |||
| 35 | EUCTR2021-000996-36-FR (EUCTR) | 25/06/2021 | A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | An 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 AB | NULL | NA | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Malaysia;Belgium;Poland;Germany;Netherlands;New Zealand | |||
| 36 | EUCTR2019-002755-42-PL (EUCTR) | 08/01/2020 | MERGE: 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. | NULL | NA | Female: yes Male: yes | 109 | Phase 2 | France;United States;Canada;Spain;Belgium;Poland;Australia;United Kingdom | |||
| 37 | EUCTR2021-000996-36-BE (EUCTR) | 01/09/2021 | A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | An 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 AB | NULL | NA | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Malaysia;Poland;Germany;Netherlands;New Zealand | |||
| 38 | EUCTR2021-000996-36-DE (EUCTR) | 02/11/2021 | A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | An 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 AB | NULL | NA | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Malaysia;Belgium;Poland;Germany;Netherlands;New Zealand | |||
| 39 | 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. | 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. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United Kingdom;Australia;Poland;Spain;Belgium;France | |||
| 40 | EUCTR2021-000996-36-PL (EUCTR) | 01/09/2021 | A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | An 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 AB | NULL | NA | Female: yes Male: yes | 60 | Phase 3 | United States;Turkey;Israel;Italy;United Kingdom;France;Canada;Poland;Malaysia;Belgium;Germany;Netherlands;New Zealand | |||
| 41 | EUCTR2013-005373-43-FR (EUCTR) | 10/07/2015 | 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: 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 Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France;Canada;Belgium;Poland;Spain;Australia;United Kingdom | |||
| 42 | EUCTR2019-002755-42-BE (EUCTR) | 11/12/2019 | MERGE: 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. | NULL | NA | Female: yes Male: yes | 109 | Phase 2 | France;United States;Canada;Spain;Poland;Belgium;Australia;United Kingdom | |||
| 43 | EUCTR2020-004011-28-PL (EUCTR) | 07/12/2020 | An Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome | A 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 AB | NULL | NA | Female: yes Male: yes | 60 | Phase 3 | United Kingdom;Italy;United States;France;Canada;Belgium;Poland;Denmark;Australia;Netherlands;Germany |