299. Cystic fibrosis Clinical trials / Disease details
Clinical trials : 1,695 / Drugs : 1,527 - (DrugBank : 268) / Drug target genes : 111 - Drug target pathways : 174
| 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 | NCT03256799 (ClinicalTrials.gov) | March 17, 2017 | 18/8/2017 | Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations | An Open Label Study to Investigate the Role of Ivacaftor for the Treatment of Cystic Fibrosis in Combination With Ataluren (PTC124) in Cystic Fibrosis Patients Using Ataluren for Nonsense Mutations | Cystic Fibrosis | Drug: Ivacaftor/Ataluren | University of Alabama at Birmingham | NULL | Completed | 19 Years | N/A | All | 1 | Phase 4 | United States |
| 2 | NCT03256968 (ClinicalTrials.gov) | January 27, 2017 | 18/8/2017 | PTC Study to Evaluate Ataluren in Combination With Ivacaftor | An Open Label N of 1 Study to Evaluate the Study and Efficacy of Long-Term Treatment With Ivacaftor in Combination With Ataluren (PTC124) in Subjects With Nonsense Mutation Cystic Fibrosis | Cystic Fibrosis | Drug: Ataluren | University of Alabama at Birmingham | NULL | Completed | 6 Years | N/A | All | 1 | Phase 4 | United States |
| 3 | 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 | ||
| 4 | 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] | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | ||
| 11 | 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 | ||
| 12 | NCT02456103 (ClinicalTrials.gov) | August 31, 2015 | 26/5/2015 | Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis | Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis | Cystic Fibrosis | Drug: Ataluren | PTC Therapeutics | NULL | Terminated | 6 Years | N/A | All | 246 | Phase 3 | United States;Argentina;Australia;Belgium;Brazil;Bulgaria;Canada;France;Germany;Greece;Israel;Italy;Netherlands;Poland;Spain;United Kingdom |
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | 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 | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;United Kingdom;Italy;France;Canada;Argentina;Belgium;Brazil;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany | ||
| 16 | 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 | ||
| 17 | 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 | ||
| 18 | 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 | ||
| 19 | EUCTR2013-005449-35-IT (EUCTR) | 16/09/2014 | 25/06/2014 | An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) | 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 | United States;France;Spain;Belgium;Israel;Germany;Italy;Sweden | ||
| 20 | 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 | ||
| 21 | 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 | ||
| 22 | 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 | ||
| 23 | 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 | ||
| 24 | 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 | ||
| 25 | 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 | ||
| 26 | 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 |
| 27 | NCT02107859 (ClinicalTrials.gov) | May 23, 2014 | 4/4/2014 | Study of Ataluren (PTC124) in Cystic Fibrosis | An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124) | Cystic Fibrosis | Drug: Ataluren | PTC Therapeutics | NULL | Terminated | 6 Years | N/A | All | 61 | Phase 3 | United States;Belgium;France;Israel;Italy;Spain;Sweden;Germany |
| 28 | EUCTR2010-019692-30-ES (EUCTR) | 07/09/2011 | 29/07/2011 | Extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 14.0;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 | France;United States;Canada;Belgium;Spain;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 29 | EUCTR2010-019692-30-GB (EUCTR) | 22/08/2011 | 23/08/2011 | Extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 14.0;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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;Italy;United Kingdom;Sweden | ||
| 30 | EUCTR2010-019692-30-DE (EUCTR) | 11/05/2011 | 13/04/2011 | Extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 14.1;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 | France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 31 | EUCTR2010-019692-30-SE (EUCTR) | 16/12/2010 | 11/10/2010 | Extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 14.0;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 | France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 32 | EUCTR2010-019692-30-NL (EUCTR) | 04/11/2010 | 24/06/2010 | Extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 14.0;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 | France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 33 | EUCTR2010-019692-30-IT (EUCTR) | 06/10/2010 | 02/11/2010 | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis - PTC124-GD-009e-CF | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis - PTC124-GD-009e-CF | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 9.1;Level: LLT;Classification code 10011762 | 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 | France;Spain;Belgium;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 34 | EUCTR2010-019692-30-FR (EUCTR) | 05/10/2010 | 17/06/2010 | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 12.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | France;Spain;Belgium;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 35 | 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 |
| 36 | EUCTR2010-019692-30-BE (EUCTR) | 10/08/2010 | 25/05/2010 | Extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 14.0;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 | France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 37 | NCT00803205 (ClinicalTrials.gov) | September 8, 2009 | 4/12/2008 | Study of Ataluren (PTC124™) in Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | Cystic Fibrosis | Drug: Ataluren;Drug: Placebo | PTC Therapeutics | Cystic Fibrosis Foundation | Completed | 6 Years | N/A | All | 238 | Phase 3 | United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Spain;Sweden;United Kingdom |
| 38 | EUCTR2008-003924-52-GB (EUCTR) | 26/08/2009 | 09/04/2009 | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 9.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: yes Male: yes | 208 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Spain;Belgium;Germany;Netherlands;Italy;United Kingdom;Sweden | ||
| 39 | EUCTR2008-003924-52-DE (EUCTR) | 17/08/2009 | 10/07/2009 | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 9.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;Italy;United Kingdom;Sweden | ||
| 40 | EUCTR2008-003924-52-BE (EUCTR) | 08/04/2009 | 20/02/2009 | Study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 14.0;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 | France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;Italy;United Kingdom;Sweden | ||
| 41 | EUCTR2008-003924-52-SE (EUCTR) | 08/04/2009 | 17/12/2008 | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | Nonsense-Mutation-Mediated Cystic Fibrosis MedDRA version: 9.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | France;Spain;Belgium;Germany;Netherlands;Italy;United Kingdom;Sweden | ||
| 42 | NCT00458341 (ClinicalTrials.gov) | March 23, 2007 | 6/4/2007 | A Study of Ataluren in Pediatric Participants With Cystic Fibrosis | A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | Cystic Fibrosis | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 6 Years | 18 Years | All | 30 | Phase 2 | Belgium;France;Israel |
| 43 | NCT00237380 (ClinicalTrials.gov) | November 30, 2005 | 7/10/2005 | Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis | A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis | Cystic Fibrosis | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 18 Years | N/A | All | 24 | Phase 2 | Israel |