338. 進行性家族性肝内胆汁うっ滞症 Progressive familial intrahepatic cholestasis Clinical trials / Disease details
臨床試験数 : 60 / 薬物数 : 25 - (DrugBank : 6) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2
| No. | 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 | NCT04483531 (ClinicalTrials.gov) | June 1, 2021 | 13/7/2020 | Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis | Odevixibat (A4250) for the Treatment of Progressive Familial Intrahepatic Cholestasis (Expanded Access Program) | Progressive Familial Intrahepatic Cholestasis | Drug: Odevixibat | Albireo | NULL | Approved for marketing | N/A | N/A | All | United States | ||
| 2 | EUCTR2017-002325-38-BE (EUCTR) | 11/02/2019 | 04/09/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 3 | EUCTR2017-002325-38-ES (EUCTR) | 12/12/2018 | 17/09/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 4 | EUCTR2017-002325-38-IT (EUCTR) | 09/11/2018 | 11/10/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 5 | EUCTR2017-002325-38-GB (EUCTR) | 02/11/2018 | 26/06/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 6 | EUCTR2017-002325-38-NL (EUCTR) | 31/10/2018 | 01/08/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;Italy;United Kingdom;France;Canada;Belgium;Poland;Australia;Netherlands;Germany;Sweden | ||
| 7 | EUCTR2017-002325-38-SE (EUCTR) | 30/10/2018 | 07/06/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Odevixibat Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Odevixibat Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Odevixibat Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Odevixibat Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 8 | NCT03659916 (ClinicalTrials.gov) | September 28, 2018 | 24/8/2018 | Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis | Drug: A4250 (odevixibat) | Albireo | NULL | Recruiting | N/A | 100 Years | All | 120 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Saudi Arabia;Spain;Sweden;Turkey;United Kingdom |
| 9 | EUCTR2017-002338-21-BE (EUCTR) | 13/09/2018 | 30/03/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 10 | EUCTR2017-002325-38-FR (EUCTR) | 11/09/2018 | 07/06/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 11 | EUCTR2017-002338-21-IT (EUCTR) | 05/06/2018 | 14/01/2021 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) - A study to determine if A4250 is safe and can be used to treat children with Progressive Familial In | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: --- Product Code: [A4250] Other descriptive name: A4250 (1200 µg) Product Name: --- Product Code: [A4250] Other descriptive name: A4250 (600 µg) Product Name: --- Product Code: [A4250] Other descriptive name: A4250 (400 µg) Product Name: --- Product Code: [A4250] Other descriptive name: A4250 (200 µg) | ALBIREO AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 12 | EUCTR2017-002338-21-GB (EUCTR) | 04/06/2018 | 09/02/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 13 | EUCTR2017-002338-21-NL (EUCTR) | 28/05/2018 | 19/02/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Sweden;Germany;Netherlands;Australia;Poland;Belgium;Canada;France;Italy;United Kingdom;Israel;Turkey;Spain | ||
| 14 | NCT03566238 (ClinicalTrials.gov) | May 16, 2018 | 25/5/2018 | This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2 | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | PFIC1;PFIC2 | Drug: A4250 (odevixibat);Drug: Placebo | Albireo | NULL | Completed | 6 Months | 18 Years | All | 62 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Saudi Arabia;Spain;Sweden;Turkey;United Kingdom |
| 15 | EUCTR2017-002338-21-ES (EUCTR) | 29/04/2018 | 08/03/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 16 | EUCTR2017-002338-21-FR (EUCTR) | 23/04/2018 | 01/02/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | ||
| 17 | NCT03082937 (ClinicalTrials.gov) | January 31, 2017 | 27/2/2017 | An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects | An Open Label, Single-Dose, Single Period Study Designed to Assess the Mass Balance Recovery, Metabolite Profile and Metabolite Identification of [14C]-A4250 in Healthy Male Subjects | Orphan Cholestatic Liver Diseases;Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome;Primary Biliary Cirrhosis | Drug: 3 mg [14C]-A4250 capsule | Albireo | NULL | Completed | 30 Years | 65 Years | Male | 6 | Phase 1 | United Kingdom |
| 18 | NCT02963077 (ClinicalTrials.gov) | July 2013 | 1/11/2016 | A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384 | A Phase I, Double-Blind Single and Multiple Ascending Dose Study to Assess Safety and Pharmacokinetics of A4250 as Monotherapy, and in Combination With Colonic Release Cholestyramine (A3384) or Commercially Available Cholestyramine (Questran™) in Healthy Subjects | Orphan Cholestatic Liver Diseases;Primary Biliary Cirrhosis;Progressive Familial Intrahepatic Cholestasis;Alagille Syndrome | Drug: A4250;Drug: CRC (A3384);Drug: Questran;Drug: Placebo | Albireo | NULL | Completed | 18 Years | 60 Years | Both | 94 | Phase 1 | NULL |
| 19 | EUCTR2017-002325-38-DE (EUCTR) | 06/06/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | NA | Female: yes Male: yes | 120 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | |||
| 20 | EUCTR2017-002325-38-PL (EUCTR) | 19/11/2018 | A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | NA | Female: yes Male: yes | 120 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden | |||
| 21 | EUCTR2017-002338-21-SE (EUCTR) | 29/01/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | |||
| 22 | EUCTR2017-002338-21-DE (EUCTR) | 30/01/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Germany;Netherlands;Sweden | |||
| 23 | EUCTR2017-002338-21-PL (EUCTR) | 29/10/2018 | A study to determine if A4250 is safe and can be used to treat children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2. | A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1) | Progressive Familial Intrahepatic Cholestasis Types 1 and 2 MedDRA version: 20.0;Level: PT;Classification code 10076033;Term: Progressive familial intrahepatic cholestasis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (1200 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (600 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (400 µg) Product Code: A4250 INN or Proposed INN: Not applicable Other descriptive name: A4250 (200 µg) | Albireo AB | NULL | Not Recruiting | Female: yes Male: yes | 60 | Phase 3 | United States;Saudi Arabia;Spain;Turkey;Israel;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Germany;Netherlands;Sweden |