338. 進行性家族性肝内胆汁うっ滞症 Progressive familial intrahepatic cholestasis Clinical trials / Disease details


臨床試験数 : 60 薬物数 : 25 - (DrugBank : 6) / 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
1NCT04729751
(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
2NCT04483531
(ClinicalTrials.gov)
June 1, 202113/7/2020Odevixibat for the Treatment of Progressive Familial Intrahepatic CholestasisOdevixibat (A4250) for the Treatment of Progressive Familial Intrahepatic Cholestasis (Expanded Access Program)Progressive Familial Intrahepatic CholestasisDrug: OdevixibatAlbireoNULLApproved for marketingN/AN/AAllUnited States
3EUCTR2020-004628-40-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
4EUCTR2020-004628-40-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
5EUCTR2019-003395-39-IT
(EUCTR)
20/05/202024/05/2021Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) - Not Applicable In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
Product Code: [Maralixibat]
INN or Proposed INN: Maralixibat cloruro
Product Name: Maralixibat
Product Code: [Maralixibat]
INN or Proposed INN: Maralixibat cloruro
Product Name: Maralixibat
Product Code: [Maralixibat]
INN or Proposed INN: Maralixibat cloruro
Product Name: Maralixibat
Product Code: [Maralixibat]
INN or Proposed INN: Maralixibat cloruro
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany
6EUCTR2019-003395-39-GB
(EUCTR)
30/03/202028/11/2019Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
7EUCTR2019-002755-42-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
8EUCTR2019-002755-42-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
9EUCTR2019-003395-39-AT
(EUCTR)
11/03/202008/01/2020Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Singapore;Germany
10EUCTR2019-003395-39-FR
(EUCTR)
09/03/202017/12/2019Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Singapore;Germany
11EUCTR2019-002755-42-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
12EUCTR2019-003395-39-HU
(EUCTR)
05/02/202007/01/2020Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
13NCT04185363
(ClinicalTrials.gov)
January 8, 20202/12/2019An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)Progressive Familial Intrahepatic Cholestasis (PFIC)Drug: MaralixibatMirum Pharmaceuticals, Inc.NULLEnrolling by invitation1 Year18 YearsAll30Phase 3United States;Argentina;Austria;Belgium;Brazil;Canada;Colombia;France;Germany;Italy;Lebanon;Mexico;Poland;Singapore;United Kingdom
14EUCTR2019-001211-22-DE
(EUCTR)
15/11/201903/06/2019A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany
15EUCTR2019-001211-22-AT
(EUCTR)
13/11/201911/07/2019A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany
16EUCTR2019-001211-22-GB
(EUCTR)
29/10/201930/05/2019A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC Progressive Familial Intrahepatic Cholestasis (PFIC)In patients with progressive familial intrahepatic cholestasis (PFIC), 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 acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Saudi Arabia;Lebanon;Turkey;Austria;Chile;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
17EUCTR2019-001211-22-FR
(EUCTR)
23/10/201919/06/2019A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC), 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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat (formerly SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Saudi Arabia;Lebanon;Turkey;Austria;Chile;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
18EUCTR2019-001211-22-IT
(EUCTR)
07/10/201902/02/2021A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat (in precedenza SHP625 o LUM001)
Product Code: [Maralixibat]
INN or Proposed INN: maralixibat cloruro
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany
19EUCTR2019-001211-22-HU
(EUCTR)
15/08/201920/06/2019A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC), 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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat (formerly SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
20NCT03905330
(ClinicalTrials.gov)
July 9, 20191/4/2019A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFICProgressive Familial Intrahepatic Cholestasis (PFIC)Drug: Maralixibat;Other: PlaceboMirum Pharmaceuticals, Inc.NULLRecruiting1 Year17 YearsAll30Phase 3United States;Argentina;Austria;Belgium;Brazil;Canada;Colombia;France;Germany;Hungary;Italy;Lebanon;Mexico;Poland;Singapore;Turkey;United Kingdom
21EUCTR2017-002325-38-BE
(EUCTR)
11/02/201904/09/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
22EUCTR2017-002325-38-ES
(EUCTR)
12/12/201817/09/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
60 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden
23EUCTR2017-002325-38-IT
(EUCTR)
09/11/201811/10/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
24EUCTR2017-002325-38-GB
(EUCTR)
02/11/201826/06/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden
25EUCTR2017-002325-38-NL
(EUCTR)
31/10/201801/08/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Netherlands;Germany;Sweden
26EUCTR2017-002325-38-SE
(EUCTR)
30/10/201807/06/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Odevixibat
Other descriptive name: A4250 (200 µg)
Albireo ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
27NCT03353454
(ClinicalTrials.gov)
October 25, 201814/11/2017A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat (SHP625) in the Treatment of Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)Progressive Familial Intrahepatic Cholestasis (PFIC)Drug: Maralixibat;Drug: PlaceboMirum Pharmaceuticals, Inc.NULLWithdrawnN/A18 YearsAll0Phase 3NULL
28NCT03659916
(ClinicalTrials.gov)
September 28, 201824/8/2018Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFICAn Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)Progressive Familial Intrahepatic CholestasisDrug: A4250 (odevixibat)AlbireoNULLRecruitingN/A100 YearsAll120Phase 3United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Saudi Arabia;Spain;Sweden;Turkey;United Kingdom
29EUCTR2017-002338-21-BE
(EUCTR)
13/09/201830/03/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
30EUCTR2017-002325-38-FR
(EUCTR)
11/09/201807/06/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
60 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden
31EUCTR2017-002338-21-IT
(EUCTR)
05/06/201814/01/2021A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) - A study to determine if A4250 is safe and can be used to treat children with Progressive Familial In Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ---
Product Code: [A4250]
Other descriptive name: A4250 (1200 µg)
Product Name: ---
Product Code: [A4250]
Other descriptive name: A4250 (600 µg)
Product Name: ---
Product Code: [A4250]
Other descriptive name: A4250 (400 µg)
Product Name: ---
Product Code: [A4250]
Other descriptive name: A4250 (200 µg)
ALBIREO ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
32EUCTR2017-002338-21-GB
(EUCTR)
04/06/201809/02/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
33EUCTR2017-002338-21-NL
(EUCTR)
28/05/201819/02/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Sweden;Germany;Netherlands;Australia;Poland;Belgium;Canada;France;Italy;United Kingdom;Israel;Turkey;Spain
34NCT03566238
(ClinicalTrials.gov)
May 16, 201825/5/2018This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1)PFIC1;PFIC2Drug: A4250 (odevixibat);Drug: PlaceboAlbireoNULLCompleted6 Months18 YearsAll62Phase 3United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Saudi Arabia;Spain;Sweden;Turkey;United Kingdom
35EUCTR2017-002338-21-ES
(EUCTR)
29/04/201808/03/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
36EUCTR2017-002338-21-FR
(EUCTR)
23/04/201801/02/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
37NCT03082937
(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
38JPRN-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
39EUCTR2015-000906-20-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
40NCT02057718
(ClinicalTrials.gov)
March 1, 20145/2/2014Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic CholestasisOpen Label Study of the Efficacy and Long Term Safety of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic CholestasisProgressive Familial Intrahepatic Cholestasis (PFIC)Drug: LUM001Mirum Pharmaceuticals, Inc.NULLCompleted12 Months18 YearsAll33Phase 2United States;France;Poland;United Kingdom
41JPRN-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
42EUCTR2013-003833-14-GB
(EUCTR)
30/01/201414/11/2013AN OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001 IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASISOPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001, AN APICALSODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS - INDIGO STUDY In patients with progressive familial intrahepatic cholestasis (PFIC), 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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: LUM001
INN or Proposed INN: maralixibat chloride
Mirum Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2France;Poland;United Kingdom
43JPRN-UMIN000017823
2013/07/1705/06/2015A study to assess the safety and efficacy of rifampicin for progressive familial intrahepatic cholestasis (PFIC) and benign recurrent intrahepatic cholestasis (BRIC) Progressive familial intrahepatic cholestasisBenign recurrent intrahepatic cholestasisRifampicin (RFP, 10 mg/kg/day) are given for 4-8 weeks.
When RFP is effective against cholestasis, RFP will be tapered 2.5mg/kg per a week and withdrawn after 4 weeks.
When bilirubin level does not decrease or the continuation of RFP is difficult for some side effects, RFP will be withdrawn.
Department of Pediatrics, Graduate School of Medical Sciences, Kyushu UniversityNULLComplete: follow-up completeNot applicableNot applicableMale and Female3Not selectedJapan
44NCT02963077
(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
45JPRN-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
46EUCTR2020-004628-40-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
47EUCTR2019-002755-42-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
48EUCTR2013-003833-14-FR
(EUCTR)
22/06/2015AN OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001 IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASISAN OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001, AN APICAL SODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS - INDIGO STUDY In patients with progressive familial intrahepatic cholestasis (PFIC), 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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 18.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;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
12Phase 2United States;France;Poland;United Kingdom
49EUCTR2017-002338-21-DE
(EUCTR)
30/01/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
50EUCTR2017-002325-38-DE
(EUCTR)
06/06/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNAFemale: yes
Male: yes
120Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
51EUCTR2019-003395-39-DE
(EUCTR)
19/11/2019Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany
52EUCTR2017-002338-21-PL
(EUCTR)
29/10/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden
53EUCTR2017-002325-38-PL
(EUCTR)
19/11/2018A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNAFemale: yes
Male: yes
120Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden
54EUCTR2019-003395-39-PL
(EUCTR)
11/12/2019Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
55EUCTR2019-001211-22-PL
(EUCTR)
28/06/2019A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC),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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Singapore;Germany
56EUCTR2019-001211-22-BE
(EUCTR)
22/07/2019A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC).MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) – MARCH-PFIC. - MARCH-PFIC In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: MRX Drug Substance (formerly Maralixibat , SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: MRX Drug Substance (formerly Maralixibat, SHP625 or LUM001)
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
30Phase 3United States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
57EUCTR2019-003395-39-BE
(EUCTR)
10/12/2019Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) In patients with progressive familial intrahepatic cholestasis (PFIC), 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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Product Name: Maralixibat
INN or Proposed INN: MARALIXIBAT CHLORIDE
Mirum Pharmaceuticals Inc.NULLNA Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Lebanon;Turkey;Austria;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Singapore;Germany
58EUCTR2017-002338-21-SE
(EUCTR)
29/01/2018A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) Progressive Familial Intrahepatic Cholestasis Types 1 and 2
MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (1200 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (600 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (400 µg)
Product Code: A4250
INN or Proposed INN: Not applicable
Other descriptive name: A4250 (200 µg)
Albireo ABNULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden
59EUCTR2019-002755-42-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
60EUCTR2013-003833-14-PL
(EUCTR)
19/08/2014AN OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001 IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASISOPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001, AN APICALSODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS - INDIGO STUDY In patients with progressive familial intrahepatic cholestasis (PFIC), 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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: LUM001
INN or Proposed INN: maralixibat chloride
Mirum Pharmaceuticals, LLC.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2France;Poland;United Kingdom