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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | ||
| 11 | 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 | ||
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 | |||
| 15 | 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 | |||
| 16 | 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 | |||
| 17 | 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 | |||
| 18 | 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 |