296. 胆道閉鎖症 Biliary atresia Clinical trials / Disease details
臨床試験数 : 63 / 薬物数 : 65 - (DrugBank : 37) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 63
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05072626 (ClinicalTrials.gov) | October 11, 2021 | 13/9/2021 | High Medium-chain Triglyceride Nutritional Support in Infants With Biliary Atresia | Study on High Medium-chain Triglyceride Nutritional Support in Infants With Biliary Atresia After Kasai Portoenterostomy | Biliary Atresia;Infant;Nutrition Support;Medium-chain Triglyceride | Combination Product: high MCT formula;Dietary Supplement: MCT powder | Children's Hospital of Fudan University | NULL | Recruiting | N/A | 3 Months | All | 300 | N/A | China |
| 2 | NCT04524390 (ClinicalTrials.gov) | July 8, 2021 | 20/8/2020 | Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai | Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Biliary Atresia After Hepatoportoenterostomy | Biliary Atresia | Drug: Maralixibat;Other: Placebo | Mirum Pharmaceuticals, Inc. | NULL | Recruiting | 31 Days | 111 Days | All | 72 | Phase 2 | United States;Germany;Poland;United Kingdom |
| 3 | ChiCTR2100046985 | 2021-06-01 | 2021-06-06 | A clinical study of biliary atresia treatment with Belimumab | Immune pathogenesis and treatment of biliary atresia | biliary atresia | Control Group:Conventional therapy;Experimental group:Belimumab; | Guangzhou Women and Children Medical Center | NULL | Recruiting | 0.1 | 0.3 | Both | Control Group:6;Experimental group:6; | Phase 4 | China |
| 4 | ChiCTR2100044533 | 2021-06-01 | 2021-03-23 | Multicenter study of non-invasive diagnostic scoring system for biliary atresia | Multicenter study of non-invasive diagnostic scoring system for biliary atresia | Biliary atresia | Gold Standard:Intraoperative cholangiography;Index test:Non-invasive diagnostic scoring system for biliary atresia (Biochemicaltests, blood routine, CRP, blood ammonia, AFP, hepatobiliary B ultrasound); | Children's Hospital Affiliated to Zhejiang University Medical College | NULL | Recruiting | Both | Target condition:200;Difficult condition:0 | China | |||
| 5 | JPRN-jRCTs031210066 | 06/05/2021 | 06/05/2021 | bezafibrate for post operated bilirary atresia | Prospective study of bezafibrate for post operated bilirary atresia | Biliary Atresia Obstructive jaundice;D001656 | Bezafibrate 400 mg/day is orally administered to patients after biliary atresia in two divided doses after breakfast and dinner. | Terui Keita | NULL | Recruiting | >= 18age old | < 80age old | Both | 10 | Phase 2 | Japan |
| 6 | EUCTR2020-000974-22-IT (EUCTR) | 22/03/2021 | 24/05/2021 | Clinical study to evaluate the Efficacy and Safety of Maralixibat used in treatment of Biliary Atresia subjects after Hepatoportoenterostomy | Randomized, Double-blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy - EMBARK: Evaluation of Maralixibat in Biliary Atresia Response post Kasai | Biliary atresia (BA) is a rare, inflammatory condition of the biliary tree that presents in the first weeks of life and leads to bile duct obstruction and consequent liver injury, fibrosis and cirrhosis which lead to portal hypertension and a decline in hepatic synthetic function. Untreated, the outcome of BA is uniformly fatal. The 2 most important improvements inthe care of BA patients to date are the Kasai hepatoportoenterostomy (HPE; Kasai procedure) and orthotopic liver transplantation. MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE Product Name: Maralixibat Product Code: [Maralixibat] INN or Proposed INN: Maralixibat cloruro Other descriptive name: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | France;United States;Canada;Poland;Germany;United Kingdom;Italy | ||
| 7 | EUCTR2020-000974-22-FR (EUCTR) | 07/12/2020 | 25/09/2020 | Clinical study to evaluate the Efficacy and Safety of Maralixibat used in treatment of Biliary Atresia subjects after Hepatoportoenterostomy | Randomized, Double-blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy - EMBARK: Evaluation of Maralixibat in Biliary Atresia Response post Kasai | Biliary atresia (BA) is a rare, inflammatory condition of the biliary tree that presents in the first weeks of life and leads to bile duct obstruction and consequent liver injury, fibrosis and cirrhosis which lead to portal hypertension and a decline in hepatic synthetic function. Untreated, the outcome of BA is uniformly fatal. The 2 most important improvements in the care of BA patients to date are the Kasai hepatoportoenterostomy (HPE; Kasai procedure) and orthotopic liver transplantation. MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;France;Canada;Poland;Germany;United Kingdom;Italy | ||
| 8 | ChiCTR2000040505 | 2020-12-01 | 2020-12-01 | N-acetylcysteine in children with biliary atresia: a clinical trial | Treatment of biliary atresia with N-acetylcysteine and its mechanism | biliary atresia | Preoperative intervention group:N-Acetylcysteine;Postoperative intervention group:N-Acetylcysteine;Preoperative control group:Conventional therapy;postoperative control group:Conventional therapy; | Guangzhou Women and Children Medical Center | NULL | Pending | Both | Preoperative intervention group:20;Postoperative intervention group:20;Preoperative control group:20;postoperative control group:20; | Phase 4 | China | ||
| 9 | EUCTR2019-003807-37-NL (EUCTR) | 01/12/2020 | 28/04/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Malaysia;Belgium;Australia;Germany;Netherlands;China;New Zealand;Korea, Republic of | ||
| 10 | ChiCTR2000035667 | 2020-10-01 | 2020-08-16 | Efficacy and safety study of dimethyl fumarate in adjuvant therapy for biliary atresia | Efficacy and safety study of dimethyl fumarate in adjuvant therapy for biliary atresia | Biliary Atresia | control group:Take a placebo;Experimental group:Take dimethyl fumarate; | Children's Hospital of Fudan University | NULL | Pending | Both | control group:120;Experimental group:120; | Phase 4 | China | ||
| 11 | EUCTR2019-003807-37-IT (EUCTR) | 10/09/2020 | 21/10/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) - BOLD | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: [A4250] INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: [A4250] INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: [A4250] INN or Proposed INN: Odevixibat | ALBIREO AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Belgium;Malaysia;Australia;Denmark;Germany;Netherlands;New Zealand;China;Korea, Republic of | ||
| 12 | EUCTR2019-003807-37-FR (EUCTR) | 08/09/2020 | 27/04/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Belgium;Malaysia;Australia;Denmark;Germany;Netherlands;New Zealand;China;Korea, Republic of | ||
| 13 | NCT04494763 (ClinicalTrials.gov) | August 15, 2020 | 19/4/2018 | Primary Prophylaxis for Variceal Bleed in Biliary Atresia | Propanolol for Primary Prophylaxis for Variceal Bleed in Biliary Atresia - An Open Label Randomized Controlled Study | Portal Hypertension, Biliary Atresia | Drug: Propanolol;Other: Placebo | Institute of Liver and Biliary Sciences, India | NULL | Recruiting | 6 Months | 5 Years | All | 92 | N/A | India |
| 14 | EUCTR2019-003807-37-GB (EUCTR) | 11/08/2020 | 17/04/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) - Biliary Atresia Odevixibat Liver Disease (BOLD) | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | New Zealand;United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Belgium;Malaysia;Australia;Denmark;Netherlands;Germany;China;Korea, Republic of | ||
| 15 | ChiCTR2000032983 | 2020-08-01 | 2020-05-17 | Development of Biomarkers for Biliary Atresia and Establishment of Artificial Intelligence Diagnostic Model | Development of Biomarkers for Biliary Atresia and Establishment of Artificial Intelligence Diagnostic Model | Biliary atresia | Gold Standard:Cholangiography combined with liver biopsy;Index test:MMP7;bile acid profile; | Children's Hospital of Fudan University | NULL | Recruiting | Both | Target condition:600;Difficult condition:600 | China | |||
| 16 | NCT04506021 (ClinicalTrials.gov) | August 2020 | 3/8/2020 | A Real World Study on Clinical Efficacy of Bicarbonate Ringer's Solution in Biliary Atresia Children | A Multicenter Real World Study on Clinical Efficacy of Sodium Bicarbonate Ringer's Solution in Children With Biliary Atresia | Biliary Atresia | Drug: Bicarbonate Ringer's solution;Drug: Other Crystalloid | Tongji Hospital | NULL | Not yet recruiting | N/A | 90 Days | All | 110 | NULL | |
| 17 | EUCTR2019-003807-37-HU (EUCTR) | 29/07/2020 | 17/04/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Belgium;Malaysia;Australia;Denmark;Germany;Netherlands;New Zealand;China;Korea, Republic of | ||
| 18 | ChiCTR2000034127 | 2020-07-20 | 2020-06-25 | Application of MMP-7 in early diagnosis of neonatal biliary atresia | Application of MMP-7 in early diagnosis of neonatal biliary atresia | Biliary atresia disease | Gold Standard:Laparoscopic cholangiography;Index test:Serum MMP-7; | Department of Pediatric Surgery, Union Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology | NULL | Pending | 0.1 | Both | Target condition:300;Difficult condition:0 | China | ||
| 19 | NCT04336722 (ClinicalTrials.gov) | July 10, 2020 | 27/3/2020 | Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD) | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy | Biliary Atresia | Drug: Odevixibat;Drug: Placebo | Albireo | NULL | Recruiting | N/A | 111 Days | All | 200 | Phase 3 | United States;Australia;Belgium;Canada;China;France;Germany;Hungary;Israel;Italy;Korea, Republic of;Malaysia;Netherlands;New Zealand;Poland;Spain;Taiwan;Turkey;United Kingdom |
| 20 | NCT04373941 (ClinicalTrials.gov) | June 1, 2020 | 13/8/2019 | Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia | Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia: Part II of a Prospective, Randomized Controlled, Multi-Institutional Trial | Biliary Atresia | Drug: Filgrastim | Holterman, Ai-Xuan, M.D. | T Rose Clinical, Inc.;Big Leap Research | Recruiting | N/A | 180 Days | All | 400 | Phase 2 | Pakistan;Vietnam |
| 21 | ChiCTR2000031738 | 2020-02-18 | 2020-04-08 | A clinical study for Rituximab in the treatment of biliary atresia | Immune pathogenesis and treatment of biliary atresia | biliary atresia | Control:Conventional therapy;Experimental group:Rituximab; | Guangzhou Women and Children Medical Center | NULL | Recruiting | 0.1 | 0.3 | Both | Control:6;Experimental group:6; | Phase 4 | China |
| 22 | NCT04522869 (ClinicalTrials.gov) | August 10, 2019 | 4/9/2019 | Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Biliary Atresia | Evaluation Safety and Efficacy of Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia: A Matched Control Prospective Study | Primary Biliary Cirrhosis | Biological: Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation | Vinmec Research Institute of Stem Cell and Gene Technology | Children's Hospital Number 2, Ho Chi Minh City, Vietnam | Recruiting | 5 Months | 2 Years | All | 34 | Phase 1/Phase 2 | Vietnam |
| 23 | ChiCTR1900023843 | 2019-07-01 | 2019-06-14 | Effects of intestinal flora distribution and mucosal structure on pediatric liver transplantation with biliary atresia | Effects of intestinal flora distribution and mucosal structure on pediatric liver transplantation with biliary atresia | Biliary Atresia | 1:No;2:probiotics;3:Rifaximin; | Tianjin First Center Hospital | NULL | Pending | 0 | 18 | Both | 1:20;2:10;3:10; | China | |
| 24 | ChiCTR1800019866 | 2019-01-01 | 2018-12-02 | Development and validation of novel diagnostic model for biliary atresia | Development and validation of novel diagnostic model for biliary atresia: a multicenter diagnostic test | biliary atresia | Gold Standard:cholangiography combined with liver biopsy;Index test:Nomogram; | Children's Hospital of Fudan University | NULL | Pending | Both | Target condition:1000;Difficult condition:200 | China | |||
| 25 | EUCTR2014-004693-42-FI (EUCTR) | 02/11/2018 | 20/07/2018 | A study to look at how the study drug Obeticholic Acid (OCA), when given as a single dose and multiple doses, is tolerated in children who have biliary atresia and to look at the effects that the study drug has on the body. | A Multicenter, Open-Label, Single- and Multiple-Dose, Dose-Finding Study to Assess the Effects of Obeticholic Acid in Pediatric Subjects with Biliary Atresia | Biliary atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10004654;Term: Biliary atresia, congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: OCA 1.5mg Product Code: OCA INN or Proposed INN: obeticholic acid Trade Name: Ocaliva Product Name: OCA 5mg Product Code: OCA INN or Proposed INN: obeticholic acid Product Name: OCA 0.1 mg Product Code: OCA INN or Proposed INN: obeticholic acid Other descriptive name: OBETICHOLIC ACID | Intercept Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 72 | Phase 2 | United States;Finland;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 26 | ChiCTR1800017428 | 2018-08-15 | 2018-07-30 | Machine learning based on the gallbladder morphology for screening biliary atresia among infants with conjugated hyperbilirubinemia | Machine learning based on the gallbladder morphology for screening biliary atresia among infants with conjugated hyperbilirubinemia | Biliary atresia | Gold Standard:Surgical exploration, puncture biopsy, intraoperative cholangiography or percutaneous cholecystography;Index test:machine learning based on the gallbladder morphology; | Department of Medical Ultrasonics, the First Affiliated Hospital of Sun Yat-sen University | NULL | Pending | Both | Target condition:500;Difficult condition:100 | China | |||
| 27 | NCT03563378 (ClinicalTrials.gov) | July 28, 2018 | 21/5/2018 | Effects of Intraoperative Normal Saline vs Lactated Ringer on Outcomes in Pediatric Liver Transplantation | Effects of Intraoperative Normal Saline vs Lactated Ringer on Outcomes in Pediatric Liver Transplantation: A Double-blind Randomized Trial | Biliary Atresia Intrahepatic Syndromic Form | Drug: Lactated Ringer;Drug: Normal saline | RenJi Hospital | NULL | Unknown status | 3 Months | 6 Years | All | 200 | N/A | China |
| 28 | ChiCTR-IOR-17013585 | 2018-06-14 | 2017-11-24 | Comparison of renal injury in children with liver transplantation under combined anesthesia with sevoflurane and propofol | The influence of sevoflurane and propofol on acute kidney injury in pediatric living donor liver transplantion | Congenital biliary atresia | P:Propofol compound anesthesia;Se:Sevoflurane compound anesthesia; | Tianjin First Central Hospital | NULL | Recruiting | 0.5 | 2 | Both | P:60;Se:60; | China | |
| 29 | NCT03499249 (ClinicalTrials.gov) | May 18, 2018 | 4/4/2018 | N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy | A Phase 2 Trial of N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy | Biliary Atresia | Drug: N-Acetyl cysteine | Baylor College of Medicine | NULL | Active, not recruiting | N/A | 90 Days | All | 16 | Phase 2 | United States |
| 30 | ChiCTR1800015778 | 2018-04-20 | 2018-04-20 | Myocardial protection in pediatric patients undergoing liver transplantation | Myocardial protective strategies of pediatric liver transplantation with biliary atresia | Congenital biliary atresia | Propofol:Propofol compound anesthesia;Sev:Sev compound anesthesia; | Tianjin First Center Hospital | NULL | Recruiting | 0 | 2 | Both | Propofol:60;Sev:60; | China | |
| 31 | NCT03395028 (ClinicalTrials.gov) | January 15, 2018 | 22/12/2017 | GCSF Adjunct Therapy for Biliary Atresia | Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia | Biliary Atresia | Drug: Granulocyte Colony-Stimulating Factor | Holterman, Ai-Xuan, M.D. | T. Rose Clinical, Inc., United States;Children's National Health System;Big Leap Research, Vietnam | Completed | N/A | 180 Days | All | 6 | Early Phase 1 | United States;Vietnam |
| 32 | ChiCTR1800015250 | 2018-01-01 | 2018-03-19 | The Effect of Adjuvant UDCA Therapy Post-Kasai Portoenterostomy in Biliary Atresia: a multicenter randomized controlled trial | The Effect of Adjuvant UDCA Therapy Post-Kasai Portoenterostomy in Biliary Atresia: a multicenter randomized controlled trial | biliary atresia | high-dose:UDCA 25mg/kg po;low-dose:UDCA 15mg/kg po; | Children's Hospital of Fudan University | NULL | Recruiting | Both | high-dose:100;low-dose:100; | China | |||
| 33 | JPRN-UMIN000028549 | 2017/08/03 | 05/08/2017 | The efficacy of navigation surgery using indocyanine green for visualizing bile duct | Biliary atresia, Congenital biliary dilation, Cholelithiasis, hepato-biliary-pancreatic tumor | Injection of indocyanine green | Nagoya University Graduate School of Medicine | NULL | Pending | Not applicable | Not applicable | Male and Female | 100 | Not selected | Japan | |
| 34 | JPRN-UMIN000027972 | 2017/01/01 | 01/07/2017 | Assessment of Indocyanine Green excretion using pinpoint endoscopic fluorescence imaging system for diagnosis of biliary atresia; prospective study. | Biliary atresia | To inject the "Indocyanine Green" (0.5mg/kg) within 30 seconds. | Juntendo University School of Medicine | NULL | Complete: follow-up complete | 1months-old | 4months-old | Male and Female | 10 | Phase 2 | Japan | |
| 35 | JPRN-UMIN000024625 | 2016/12/01 | 30/10/2016 | The difference of the profile of fatty acids and eicosanoids in clinical course and the effect to the prognosis by collection of biliary atresia | The difference of the profile of fatty acids and eicosanoids in clinical course and the effect to the prognosis by collection of biliary atresia - The effect of collection of fatty acids in biliary atresia | biliary atresia | EPADEL 20-40 mg/kg/d | Nagoya University | NULL | Recruiting | Not applicable | Not applicable | Male and Female | 15 | Not selected | Japan |
| 36 | JPRN-jRCTs041180088 | 01/12/2016 | 12/03/2019 | Influence of fatty acid metabolism for clinical course of biliary atresia | The difference of the profile of fatty acids and eicosanoids in clinical course and the effect to the prognosis by collection of biliary atresia | biliary atresia, neonatal hepatitis, Alagille syndrome, PFIC, etc. | oral administration of 30(+/- 10)mg/kg/day of eicosapentaenoic acid | Sumida Wataru | Uchida Hiroo | Recruiting | Not applicable | Not applicable | Both | 30 | N/A | Japan |
| 37 | ChiCTR-IPR-16008609 | 2016-07-01 | 2016-06-07 | Therapeutic effect of prophylactic intravenous antibiotics after Kasai procedure in biliary atresia | A clinical randomized controlled study to investigate the preventive effect of prophylactic intravenous antibiotics against cholangitis after Kasai procedure in biliary atresia | biliary atresia | Short-term Group:Cefoperazone 5 days;Long-term Group:Cefoperazone 14 days; | Children's Hospital of Fudan University | NULL | Completed | 0 | 1 | Both | Short-term Group:86;Long-term Group:86; | China | |
| 38 | ChiCTR-OOC-16008707 | 2016-06-01 | 2016-06-22 | Study on pharmacodynamics of continuous infusion of rocuronium during transplantation of living donor liver transplantation for infants | Study on pharmacodynamics of continuous infusion of rocuronium during transplantation of living donor liver transplantation for infants | congenital biliary atresia | obstructive jaundice group:continuous infusion of rocuronium;after Kasai portoenterostomy group :continuous infusion of rocuronium; | Ren Ji Hospital, School of Medicine, Shanghai Jiao Tong University | NULL | Recruiting | Both | obstructive jaundice group:15;after Kasai portoenterostomy group :15; | China | |||
| 39 | EUCTR2014-004693-42-NL (EUCTR) | 05/01/2016 | 09/09/2015 | A study to look at how the study drug Obeticholic Acid (OCA), when given as a single dose and multiple doses, is tolerated in children who have biliary atresia and to look at the effects that the study drug has on the body. | A Multicenter, Randomized, Open Label, Single- and Multiple-Dose, Dose Finding Study and Open-Label Extension to Assess the Effects of Obeticholic Acid in Pediatric Subjects with Biliary Atresia | Biliary atresia MedDRA version: 18.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850 MedDRA version: 18.0;Classification code 10004654;Term: Biliary atresia, congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: OCA 1.5mg Product Code: OCA INN or Proposed INN: obeticholic acid Product Name: OCA 5mg Product Code: OCA INN or Proposed INN: obeticholic acid | Intercept Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | United States;Canada;Germany;Netherlands;United Kingdom | |||
| 40 | EUCTR2014-004693-42-DE (EUCTR) | 06/11/2015 | 01/06/2015 | A study to look at how the study drug Obeticholic Acid (OCA), when given as a single dose and multiple doses, is tolerated in children who have biliary atresia and to look at the effects that the study drug has on the body. | A Multicenter, Open-Label, Single- and Multiple-Dose, Dose-Finding Study to Assess the Effects of Obeticholic Acid in Pediatric Subjects with Biliary Atresia- P/0204/2019 | Biliary atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10004654;Term: Biliary atresia, congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: OCA 1.5mg Product Code: OCA INN or Proposed INN: obeticholic acid Trade Name: Ocaliva Product Name: OCA 5mg Product Code: OCA INN or Proposed INN: obeticholic acid Product Name: OCA 0.1 mg Product Code: OCA INN or Proposed INN: obeticholic acid Other descriptive name: OBETICHOLIC ACID | Intercept Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Finland;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 41 | EUCTR2014-004693-42-BE (EUCTR) | 27/08/2015 | 26/10/2015 | A study to look at how the study drug Obeticholic Acid (OCA), when given as a single dose and multiple doses, is tolerated in children who have biliary atresia and to look at the effects that the study drug has on the body including an optional long-term extension. | A Multicenter, Open-Label, Single- and Multiple-Dose, Dose-Finding Study, with an Optional Open-Label Extension to Assess the Safety, Tolerability, and Pharmacokinetics of Obeticholic Acid in Pediatric Subjects with Biliary Atresia - ObetiCholic Acid in Pediatric Subjects with BiliaRy AtrEsia (CARE) | Biliary atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10004654;Term: Biliary atresia, congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: OCA 1.5mg Product Code: OCA INN or Proposed INN: obeticholic acid Trade Name: Ocaliva Product Name: OCA 5mg Product Code: OCA INN or Proposed INN: obeticholic acid Product Name: OCA 0.1 mg Product Code: OCA INN or Proposed INN: obeticholic acid Other descriptive name: OBETICHOLIC ACID | Intercept Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 2 | France;Finland;Poland;Belgium;Netherlands;Germany;United Kingdom | ||
| 42 | EUCTR2014-004693-42-GB (EUCTR) | 29/06/2015 | 23/12/2014 | A study to look at how the study drug Obeticholic Acid (OCA), when given as a single dose and multiple doses, is tolerated in children who have biliary atresia and to look at the effects that the study drug has on the body. | A Multicenter, Open Label, Single- and Multiple-Dose, Dose Finding Study to Assess the Effects of Obeticholic Acid in Pediatric Subjects with Biliary Atresia | Biliary atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10004654;Term: Biliary atresia, congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Digestive System Diseases [C06] | Product Name: OCA 1.5mg Product Code: OCA INN or Proposed INN: obeticholic acid Trade Name: Ocaliva Product Name: OCA 5mg Product Code: OCA INN or Proposed INN: obeticholic acid Product Name: OCA 0.1 mg Product Code: OCA INN or Proposed INN: obeticholic acid Other descriptive name: OBETICHOLIC ACID | Intercept Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 72 | Phase 2 | United States;Finland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 43 | ChiCTR-DPD-15005847 | 2014-11-19 | 2015-01-19 | Evidence-based guideline of diagnosis and treatment of biliary atresia | Evidence-based guideline of diagnosis and treatment of biliary atresia | biliary atresia | Gold Standard:;Index test:; | Children's Hospital of Fudan University | NULL | Pending | 0 | 18 | Both | Target condition:0;Difficult condition:0 | I (Phase 1 study) | China |
| 44 | NCT01854827 (ClinicalTrials.gov) | October 2013 | 27/9/2012 | Safety Study of Intravenous Immunoglobulin (IVIG) Post-Portoenterostomy in Infants With Biliary Atresia | A Phase 1/2A Trial of Intravenous Immunoglobulin (IVIG) Therapy Following Portoenterostomy in Infants With Biliary Atresia | Biliary Atresia | Drug: Intravenous immunoglobulin (IVIG) | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | NULL | Completed | N/A | 120 Days | All | 30 | Phase 1/Phase 2 | United States;Canada |
| 45 | NCT01774487 (ClinicalTrials.gov) | January 2013 | 22/1/2013 | Pentoxifylline Therapy in Biliary Atresia | A Phase II Trial of Pentoxifylline in Newly-Diagnosed Biliary Atresia | Biliary Atresia | Drug: Pentoxifylline | Baylor College of Medicine | NULL | Recruiting | N/A | 180 Days | All | 60 | Phase 2 | United States |
| 46 | NCT02471209 (ClinicalTrials.gov) | January 2012 | 3/6/2015 | Biliary Atresia, Hepatic Buffer Response and Sevoflurane | Biliary Atresia | Drug: Sevoflurane | Children's Hospital of Fudan University | NULL | Completed | 1 Month | 3 Months | Both | 25 | N/A | NULL | |
| 47 | NCT01443572 (ClinicalTrials.gov) | September 2011 | 16/9/2011 | The Comparison of Desflurane and Sevoflurane on Postoperative Recovery and Hepatic Function of Biliary Atresia Patients During Kasai Operation | Kasai Operation;Biliary Atresia | Drug: desflurane anesthetics;Drug: sevoflurane anesthetics | Yonsei University | NULL | Completed | N/A | 1 Year | Both | 40 | N/A | Korea, Republic of | |
| 48 | NCT02137668 (ClinicalTrials.gov) | July 2010 | 12/5/2014 | Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin | The Human Gastrointestinal Tract Microbiota in the Setting of Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin | Primary Sclerosing Cholangitis;Biliary Atresia | Drug: Oral Vancomycin | Sacramento Pediatric Gastroenterology | NULL | Recruiting | N/A | 40 Years | Both | 200 | Phase 1 | United States |
| 49 | EUCTR2009-014886-21-NL (EUCTR) | 26/01/2010 | 21/09/2009 | Intraoperative bile duct visualisation in children with biliary atresia using a fluorescence camera system: a pilot study - Bile duct mapping with fluorescence in BA | Intraoperative bile duct visualisation in children with biliary atresia using a fluorescence camera system: a pilot study - Bile duct mapping with fluorescence in BA | Biliary atresia | Trade Name: ICG-Pulsion 25mg Product Name: ICG Pulsion Product Code: VO4CX | University Medical Centre Groningen | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 4 | Netherlands | |||
| 50 | NCT02298218 (ClinicalTrials.gov) | September 2009 | 19/11/2014 | Clinical Study About the Role of COX-2 Inhibitor in Liver Cirrhosis With Biliary Atresia | Biliary Atresia, Kasai Portoenterostomy Status | Drug: Meloxicam | Yonsei University | NULL | Completed | 2 Years | 17 Years | Both | 50 | Phase 4 | NULL | |
| 51 | NCT01322386 (ClinicalTrials.gov) | May 2007 | 10/2/2011 | Gastrointestinal Microbiota in Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin | The Human Gastrointestinal Tract Microbiota in the Setting of Treating Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin. | Primary Sclerosing Cholangitis;Biliary Atresia | Drug: Vancomycin | Stanford University | NULL | Completed | 1 Month | 20 Years | All | 32 | Phase 1 | NULL |
| 52 | ChiCTR-TNRC-09000331 | 2007-01-01 | 2009-02-11 | Intervention and Efficacy Evaluation Study of Combined Modality Therapy in Patients with Biliary Atrisia after Kasai Operationru | Intervention and Efficacy Evaluation Study of Combined Modality Therapy in Patients with Biliary Atrisia after Kasai Operationru | Biliary Atresia | group 1:Given Yinzhihuang with Ligustrazine from the day 3 after operation;group 2:Given Yinzhihuang with placebo from the day 3;group A:Divide two groups according different glucocorticoid, group A is given dexamethasone from the seventh day after operation;group B:Divide two groups according different glucocorticoid, group B is given methylprednisolone from the seventh day after operation; | The First Affiliated Hospital of Sun Yat Sen University | NULL | Completed | Both | group 1:50;group 2:50;group A:50;group B:50; | China | |||
| 53 | NCT00294684 (ClinicalTrials.gov) | November 2005 | 21/2/2006 | A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy | A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy in Infants With Biliary Atresia | Biliary Atresia | Drug: Corticosteroids;Drug: Placebo | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | NULL | Completed | N/A | 6 Months | All | 141 | N/A | United States |
| 54 | NCT00166868 (ClinicalTrials.gov) | December 2003 | 12/9/2005 | Use of Probiotics to Prevent Cholangitis in Children With Biliary Atresia After the Kasai Portoenterostomy | Use of Probiotics to Prevent Cholangitis in Children With Biliary Atresia | Biliary Atresia | Drug: Lactobacillus casei rhamnosus (Lcr35);Drug: Neomycin | National Taiwan University Hospital | NULL | Completed | 4 Months | 3 Years | Both | 30 | N/A | Taiwan |
| 55 | NCT00007033 (ClinicalTrials.gov) | October 2000 | 6/12/2000 | Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease | Alagille Syndrome;Cholestasis;Biliary Atresia | Drug: magnesium gluconate;Drug: magnesium sulfate | National Center for Research Resources (NCRR) | Children's Hospital Medical Center, Cincinnati | Completed | 3 Years | 18 Years | Both | 25 | N/A | United States | |
| 56 | NCT00539565 (ClinicalTrials.gov) | January 2000 | 3/10/2007 | RCT of Steroids Following Kasai Portoenterostomy for Biliary Atresia. | Phase IIIb Study of Corticosteroids as Post-Operative Adjuvant Therapy in Biliary Atresia | Biliary Atresia | Drug: prednisolone;Drug: placebo | King's College Hospital NHS Trust | NULL | Enrolling by invitation | N/A | 100 Days | Both | 100 | Phase 3 | United Kingdom |
| 57 | EUCTR2019-003807-37-PL (EUCTR) | 05/05/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat | Albireo AB | NULL | NA | Female: yes Male: yes | 200 | Phase 3 | United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Malaysia;Belgium;Australia;Netherlands;Germany;China;New Zealand;Korea, Republic of | |||
| 58 | EUCTR2019-003807-37-BE (EUCTR) | 18/06/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat | Albireo AB | NULL | NA | Female: yes Male: yes | 200 | Phase 3 | United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Belgium;Malaysia;Australia;Netherlands;Germany;China;New Zealand;Korea, Republic of | |||
| 59 | EUCTR2019-002755-42-BE (EUCTR) | 11/12/2019 | MERGE: Maralixibat Extension Safety Study Providing Long-term Treatment to Subjects with Cholestatic Liver Disease. | MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study. | Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease including, but not limited to, Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia. MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10053870;Term: Alagille syndrome;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | NA | Female: yes Male: yes | 109 | Phase 2 | France;United States;Canada;Spain;Poland;Belgium;Australia;United Kingdom | |||
| 60 | EUCTR2020-000974-22-PL (EUCTR) | 09/10/2020 | Clinical study to evaluate the Efficacy and Safety of Maralixibat used in treatment of Biliary Atresia subjects after Hepatoportoenterostomy | Randomized, Double-blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy - EMBARK: Evaluation of Maralixibat in Biliary Atresia Response post Kasai | Biliary atresia (BA) is a rare, inflammatory condition of the biliary tree that presents in the first weeks of life and leads to bile duct obstruction and consequent liver injury, fibrosis and cirrhosis which lead to portal hypertension and a decline in hepatic synthetic function. Untreated, the outcome of BA is uniformly fatal. The 2 most important improvements in the care of BA patients to date are the Kasai hepatoportoenterostomy (HPE; Kasai procedure) and orthotopic liver transplantation. MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;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. | NULL | NA | Female: yes Male: yes | 72 | Phase 2 | France;United States;Canada;Poland;Germany;United Kingdom;Italy | |||
| 61 | EUCTR2019-003807-37-DE (EUCTR) | 15/05/2020 | This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE | A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD) | Biliary Atresia MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat Product Name: Odevixibat Product Code: A4250 INN or Proposed INN: Odevixibat | Albireo AB | NULL | NA | Female: yes Male: yes | 200 | Phase 3 | United States;Taiwan;Spain;Turkey;Israel;United Kingdom;Italy;France;Hungary;Canada;Poland;Malaysia;Belgium;Australia;Netherlands;Germany;China;New Zealand;Korea, Republic of | |||
| 62 | EUCTR2020-000974-22-GB (EUCTR) | 21/10/2020 | Clinical study to evaluate the Efficacy and Safety of Maralixibat used in treatment of Biliary Atresia subjects after Hepatoportoenterostomy | Randomized, Double-blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Biliary Atresia after Hepatoportoenterostomy - EMBARK: Evaluation of Maralixibat in Biliary Atresia Response post Kasai | Biliary atresia (BA) is a rare, inflammatory condition of the biliary tree that presents in the first weeks of life and leads to bile duct obstruction and consequent liver injury, fibrosis and cirrhosis which lead to portal hypertension and a decline in hepatic synthetic function. Untreated, the outcome of BA is uniformly fatal. The 2 most important improvements in the care of BA patients to date are the Kasai hepatoportoenterostomy (HPE; Kasai procedure) and orthotopic liver transplantation. MedDRA version: 20.0;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 100000004850;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. | NULL | NA | Female: yes Male: yes | 72 | Phase 2 | United States;France;Canada;Poland;Germany;Italy;United Kingdom | |||
| 63 | EUCTR2019-002755-42-PL (EUCTR) | 08/01/2020 | MERGE: Maralixibat Extension Safety Study Providing Long-term Treatment to Subjects with Cholestatic Liver Disease. | MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study. | Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease including, but not limited to, Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia. MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10053870;Term: Alagille syndrome;Level: LLT;Classification code 10004653;Term: Biliary atresia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE Product Name: Maralixibat (formely SHP625 or LUM001) INN or Proposed INN: MARALIXIBAT CHLORIDE | Mirum Pharmaceuticals Inc. | NULL | NA | Female: yes Male: yes | 109 | Phase 2 | France;United States;Canada;Spain;Belgium;Poland;Australia;United Kingdom |