Ataluren (ptc124®) (DrugBank: Ataluren)
6 diseases| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 0 |
| 113 | 筋ジストロフィー | 8 |
| 140 | ドラベ症候群 | 0 |
| 246 | メチルマロン酸血症 | 2 |
| 299 | 嚢胞性線維症 | 24 |
| 329 | 無虹彩症 | 0 |
| 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 | NCT02819557 (ClinicalTrials.gov) | June 9, 2016 | 16/6/2016 | Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy | A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged =2 to <5 Years Old With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 2 Years | 5 Years | Male | 14 | Phase 2 | United States |
| 2 | EUCTR2011-004853-18-GB (EUCTR) | 21/08/2012 | 29/03/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | |||
| 3 | EUCTR2011-004853-18-ES (EUCTR) | 13/06/2012 | 17/04/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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: no Male: yes | 96 | Phase 3 | Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 4 | NCT01557400 (ClinicalTrials.gov) | May 20, 2012 | 15/3/2012 | Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy;Dystrophinopathy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | N/A | N/A | Male | 94 | Phase 3 | Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 5 | EUCTR2011-004853-18-FR (EUCTR) | 02/05/2012 | 13/08/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 14.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;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 |
| 6 | EUCTR2011-004853-18-DE (EUCTR) | 02/05/2012 | 12/03/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 7 | EUCTR2011-004853-18-BE (EUCTR) | 27/04/2012 | 03/01/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 16.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 8 | EUCTR2011-004853-18-SE (EUCTR) | 26/04/2012 | 27/02/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2009-016654-41-BE (EUCTR) | 04/11/2010 | 22/12/2010 | Study of ataluren in patients with Methylmalonic Acidemia | A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia | Nonsense Mutation Methylmalonic Acidemia (MMA) MedDRA version: 14.0;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased;System Organ Class: 10022891 - Investigations;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | 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 | 18 | Phase 2 | France;Belgium;Italy;United Kingdom;Switzerland | ||
| 2 | NCT01141075 (ClinicalTrials.gov) | July 19, 2010 | 7/6/2010 | Ataluren for Nonsense Mutation Methylmalonic Acidemia | A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia | Amino Acid Metabolism, Inborn Errors | Drug: Ataluren | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | 2 Years | N/A | All | 11 | Phase 2 | Belgium;France;Germany;Italy;Switzerland;United Kingdom |
| 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 |