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

  
30 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
Inclusion_
agemax
Inclusion_
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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
2EUCTR2020-004628-40-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
3EUCTR2020-004628-40-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
4EUCTR2019-003395-39-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
5EUCTR2019-003395-39-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
6EUCTR2019-002755-42-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
7EUCTR2019-002755-42-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
8EUCTR2019-003395-39-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
9EUCTR2019-003395-39-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
10EUCTR2019-002755-42-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
11EUCTR2019-003395-39-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
12NCT04185363
(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
13EUCTR2019-001211-22-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
14EUCTR2019-001211-22-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
15EUCTR2019-001211-22-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
16EUCTR2019-001211-22-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
17EUCTR2019-001211-22-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
18EUCTR2019-001211-22-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
19NCT03905330
(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
20NCT03353454
(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
21EUCTR2013-003833-14-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
22EUCTR2019-003395-39-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
23EUCTR2019-001211-22-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
24EUCTR2019-002755-42-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
25EUCTR2019-003395-39-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
26EUCTR2013-003833-14-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
27EUCTR2019-002755-42-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
28EUCTR2020-004628-40-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
29EUCTR2019-001211-22-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
30EUCTR2019-003395-39-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