299. 嚢胞性線維症
［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］
Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | EUCTR2014-005355-83-GR (EUCTR) | 05/04/2016 | 08/02/2016 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 2 | EUCTR2014-005355-83-GB (EUCTR) | 22/03/2016 | 15/02/2016 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | |||
| 3 | EUCTR2014-005355-83-BG (EUCTR) | 19/02/2016 | 16/12/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 4 | EUCTR2014-005355-83-NL (EUCTR) | 22/01/2016 | 25/11/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 5 | EUCTR2014-005355-83-FR (EUCTR) | 08/12/2015 | 04/12/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 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 |
| 6 | EUCTR2014-005355-83-IT (EUCTR) | 16/10/2015 | 26/08/2015 | Confirmatory extension study of ataluren (PTC124) in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis - NA | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 7 | EUCTR2014-005355-83-ES (EUCTR) | 08/10/2015 | 02/09/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 8 | EUCTR2013-004581-34-BE (EUCTR) | 01/10/2015 | 31/07/2014 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 17.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany | ||
| 9 | EUCTR2014-005355-83-BE (EUCTR) | 01/10/2015 | 24/08/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 10 | EUCTR2013-005449-35-FR (EUCTR) | 25/08/2015 | 18/06/2015 | Extension study of ataluren in patients with Cystic Fibrosis | An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 76 | Phase 3 | United States;France;Spain;Belgium;Israel;Germany;Italy;Sweden | ||
| 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 |
| 11 | EUCTR2013-004581-34-GR (EUCTR) | 29/07/2015 | 23/07/2015 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany | ||
| 12 | EUCTR2013-004581-34-NL (EUCTR) | 26/02/2015 | 06/08/2014 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany | ||
| 13 | EUCTR2013-004581-34-BG (EUCTR) | 05/02/2015 | 06/01/2015 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany | ||
| 14 | EUCTR2013-004581-34-DE (EUCTR) | 25/11/2014 | 05/08/2014 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany | ||
| 15 | EUCTR2013-005449-35-DE (EUCTR) | 01/10/2014 | 04/08/2014 | Extension study of ataluren in patients with Cystic Fibrosis | An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) | Nonsense Mutation Cystic Fibrosis MedDRA version: 17.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 76 | Phase 3 | France;United States;Belgium;Spain;Israel;Germany;Italy;Sweden | ||
| 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 |
| 16 | EUCTR2013-005449-35-IT (EUCTR) | 16/09/2014 | 25/06/2014 | Extension study of ataluren in patients with Cystic Fibrosis | An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) | Nonsense Mutation Cystic Fibrosis MedDRA version: 17.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 76 | Phase 3 | France;United States;Belgium;Spain;Israel;Germany;Italy;Sweden | ||
| 17 | EUCTR2013-004581-34-IT (EUCTR) | 15/09/2014 | 10/06/2014 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 17.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 210 | Phase 3 | United States;Greece;Spain;Turkey;Israel;Russian Federation;United Kingdom;Italy;Canada;Argentina;Brazil;Belgium;Australia;Bulgaria;Germany;Netherlands | ||
| 18 | EUCTR2013-004581-34-GB (EUCTR) | 12/09/2014 | 25/09/2014 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany | ||
| 19 | EUCTR2013-004581-34-ES (EUCTR) | 01/09/2014 | 14/08/2014 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 17.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 210 | Phase 3 | United States;Greece;Spain;Turkey;Israel;Russian Federation;United Kingdom;Italy;Canada;Argentina;Brazil;Belgium;Australia;Bulgaria;Germany;Netherlands | ||
| 20 | EUCTR2013-005449-35-ES (EUCTR) | 01/09/2014 | 11/08/2014 | Extension study of ataluren in patients with Cystic Fibrosis | An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) | Nonsense Mutation Cystic Fibrosis MedDRA version: 17.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 76 | Phase 3 | France;United States;Belgium;Spain;Israel;Germany;Italy;Sweden | ||
| 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 |
| 21 | EUCTR2013-005449-35-BE (EUCTR) | 26/08/2014 | 04/06/2014 | Extension study of ataluren in patients with Cystic Fibrosis | An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | France;United States;Spain;Belgium;Israel;Germany;Italy;Sweden | ||
| 22 | EUCTR2013-005449-35-SE (EUCTR) | 06/08/2014 | 05/06/2014 | Extension study of ataluren in patients with Cystic Fibrosis | An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) | Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | France;United States;Spain;Belgium;Israel;Germany;Italy;Sweden | ||
| 23 | NCT02139306 (ClinicalTrials.gov) | August 2014 | 13/5/2014 | Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis | Cystic Fibrosis | Drug: Ataluren (PTC124®);Drug: Placebo | PTC Therapeutics | Cystic Fibrosis Foundation;ECFS-Clinical Trial Network (ECFS-CTN) | Completed | 6 Years | N/A | All | 279 | Phase 3 | United States;Argentina;Australia;Belgium;Brazil;Bulgaria;Canada;France;Germany;Greece;Israel;Italy;Netherlands;Poland;Spain;United Kingdom |
| 24 | NCT01140451 (ClinicalTrials.gov) | August 12, 2010 | 7/6/2010 | Extension Study of Ataluren (PTC124) in Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124®) in Subjects With Nonsense-Mutation-Mediated Cystic Fibrosis | Cystic Fibrosis | Drug: Ataluren | PTC Therapeutics | Cystic Fibrosis Foundation | Completed | 6 Years | N/A | All | 191 | Phase 3 | United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Spain;Sweden;United Kingdom |