299. 嚢胞性線維症 Cystic fibrosis Clinical trials / Disease details


臨床試験数 : 1,695 薬物数 : 1,527 - (DrugBank : 268) / 標的遺伝子数 : 111 - 標的パスウェイ数 : 174

  
50 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03256799
(ClinicalTrials.gov)
March 17, 201718/8/2017Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense MutationsAn 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 MutationsCystic FibrosisDrug: Ivacaftor/AtalurenUniversity of Alabama at BirminghamNULLCompleted19 YearsN/AAll1Phase 4United States
2NCT03256968
(ClinicalTrials.gov)
January 27, 201718/8/2017PTC Study to Evaluate Ataluren in Combination With IvacaftorAn 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 FibrosisCystic FibrosisDrug: AtalurenUniversity of Alabama at BirminghamNULLCompleted6 YearsN/AAll1Phase 4United States
3EUCTR2014-005355-83-GR
(EUCTR)
05/04/201608/02/2016Confirmatory extension study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
4EUCTR2014-005355-83-GB
(EUCTR)
22/03/201615/02/2016Confirmatory 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.NULLNot 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): noUnited States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
5EUCTR2014-005355-83-BG
(EUCTR)
19/02/201616/12/2015Confirmatory extension study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
6EUCTR2014-005355-83-NL
(EUCTR)
22/01/201625/11/2015Confirmatory extension study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
7EUCTR2014-005355-83-FR
(EUCTR)
08/12/201504/12/2015Confirmatory extension study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
8EUCTR2014-005355-83-IT
(EUCTR)
16/10/201526/08/2015Confirmatory extension study of ataluren (PTC124) in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
9EUCTR2014-005355-83-ES
(EUCTR)
08/10/201502/09/2015Confirmatory extension study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
10EUCTR2014-005355-83-BE
(EUCTR)
01/10/201524/08/2015Confirmatory extension study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
200Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
11EUCTR2013-004581-34-BE
(EUCTR)
01/10/201531/07/2014Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany
12NCT02456103
(ClinicalTrials.gov)
August 31, 201526/5/2015Extension Study of Ataluren in Participants With Nonsense Mutation Cystic FibrosisPhase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic FibrosisCystic FibrosisDrug: AtalurenPTC TherapeuticsNULLTerminated6 YearsN/AAll246Phase 3United States;Argentina;Australia;Belgium;Brazil;Bulgaria;Canada;France;Germany;Greece;Israel;Italy;Netherlands;Poland;Spain;United Kingdom
13EUCTR2013-005449-35-FR
(EUCTR)
25/08/201518/06/2015Extension study of ataluren in patients with Cystic FibrosisAn 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.NULLNot RecruitingFemale: yes
Male: yes
76Phase 3United States;France;Spain;Belgium;Israel;Germany;Italy;Sweden
14EUCTR2013-004581-34-GR
(EUCTR)
29/07/201523/07/2015Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany
15EUCTR2013-004581-34-NL
(EUCTR)
26/02/201506/08/2014Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3United States;Greece;Spain;Israel;United Kingdom;Italy;France;Canada;Argentina;Belgium;Brazil;Poland;Australia;Denmark;Bulgaria;Netherlands;Germany
16EUCTR2013-004581-34-BG
(EUCTR)
05/02/201506/01/2015Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany
17EUCTR2013-004581-34-DE
(EUCTR)
25/11/201405/08/2014Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany
18EUCTR2013-005449-35-DE
(EUCTR)
01/10/201404/08/2014Extension study of ataluren in patients with Cystic FibrosisAn 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.NULLNot RecruitingFemale: yes
Male: yes
76Phase 3France;United States;Belgium;Spain;Israel;Germany;Italy;Sweden
19EUCTR2013-005449-35-IT
(EUCTR)
16/09/201425/06/2014An 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.NULLNot RecruitingFemale: yes
Male: yes
76Phase 3United States;France;Spain;Belgium;Israel;Germany;Italy;Sweden
20EUCTR2013-004581-34-IT
(EUCTR)
15/09/201410/06/2014Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
210Phase 3United States;Greece;Spain;Turkey;Israel;Russian Federation;United Kingdom;Italy;Canada;Argentina;Brazil;Belgium;Australia;Bulgaria;Germany;Netherlands
21EUCTR2013-004581-34-GB
(EUCTR)
12/09/201425/09/2014Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany
22EUCTR2013-005449-35-ES
(EUCTR)
01/09/201411/08/2014Extension study of ataluren in patients with Cystic FibrosisAn 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.NULLNot RecruitingFemale: yes
Male: yes
76Phase 3France;United States;Belgium;Spain;Israel;Germany;Italy;Sweden
23EUCTR2013-004581-34-ES
(EUCTR)
01/09/201414/08/2014Confirmatory study of ataluren in patients with Cystic FibrosisA 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.NULLNot RecruitingFemale: yes
Male: yes
210Phase 3United States;Greece;Spain;Turkey;Israel;Russian Federation;United Kingdom;Italy;Canada;Argentina;Brazil;Belgium;Australia;Bulgaria;Germany;Netherlands
24EUCTR2013-005449-35-BE
(EUCTR)
26/08/201404/06/2014Extension study of ataluren in patients with Cystic FibrosisAn 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.NULLNot RecruitingFemale: yes
Male: yes
70Phase 3France;United States;Spain;Belgium;Israel;Germany;Italy;Sweden
25EUCTR2013-005449-35-SE
(EUCTR)
06/08/201405/06/2014Extension study of ataluren in patients with Cystic FibrosisAn 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.NULLNot RecruitingFemale: yes
Male: yes
70Phase 3France;United States;Spain;Belgium;Israel;Germany;Italy;Sweden
26NCT02139306
(ClinicalTrials.gov)
August 201413/5/2014Study 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 FibrosisCystic FibrosisDrug: Ataluren (PTC124®);Drug: PlaceboPTC TherapeuticsCystic Fibrosis Foundation;ECFS-Clinical Trial Network (ECFS-CTN)Completed6 YearsN/AAll279Phase 3United States;Argentina;Australia;Belgium;Brazil;Bulgaria;Canada;France;Germany;Greece;Israel;Italy;Netherlands;Poland;Spain;United Kingdom
27NCT02107859
(ClinicalTrials.gov)
May 23, 20144/4/2014Study of Ataluren (PTC124) in Cystic FibrosisAn Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)Cystic FibrosisDrug: AtalurenPTC TherapeuticsNULLTerminated6 YearsN/AAll61Phase 3United States;Belgium;France;Israel;Italy;Spain;Sweden;Germany
28EUCTR2010-019692-30-ES
(EUCTR)
07/09/201129/07/2011Extension study of ataluren in patients with Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
200Phase 3France;United States;Canada;Belgium;Spain;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden
29EUCTR2010-019692-30-GB
(EUCTR)
22/08/201123/08/2011Extension study of ataluren in patients with Cystic FibrosisA 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, IncNULLNot 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): noFrance;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;Italy;United Kingdom;Sweden
30EUCTR2010-019692-30-DE
(EUCTR)
11/05/201113/04/2011Extension study of ataluren in patients with Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
200Phase 3France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy;Sweden
31EUCTR2010-019692-30-SE
(EUCTR)
16/12/201011/10/2010Extension study of ataluren in patients with Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
200Phase 3France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden
32EUCTR2010-019692-30-NL
(EUCTR)
04/11/201024/06/2010Extension study of ataluren in patients with Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
200Phase 3France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden
33EUCTR2010-019692-30-IT
(EUCTR)
06/10/201002/11/2010A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis - PTC124-GD-009e-CFA 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.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3France;Spain;Belgium;Germany;Netherlands;United Kingdom;Italy;Sweden
34EUCTR2010-019692-30-FR
(EUCTR)
05/10/201017/06/2010A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
208Phase 3France;Spain;Belgium;Germany;Netherlands;United Kingdom;Italy;Sweden
35NCT01140451
(ClinicalTrials.gov)
August 12, 20107/6/2010Extension Study of Ataluren (PTC124) in Cystic FibrosisA Phase 3 Extension Study of Ataluren (PTC124®) in Subjects With Nonsense-Mutation-Mediated Cystic FibrosisCystic FibrosisDrug: AtalurenPTC TherapeuticsCystic Fibrosis FoundationCompleted6 YearsN/AAll191Phase 3United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Spain;Sweden;United Kingdom
36EUCTR2010-019692-30-BE
(EUCTR)
10/08/201025/05/2010Extension study of ataluren in patients with Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
200Phase 3France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy;Sweden
37EUCTR2008-003924-52-NL
(EUCTR)
06/10/200905/02/2009A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisA 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: not assigned
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: not assigned
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: not assigned
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
PTC Therapeutics, IncNULLNot RecruitingFemale: yes
Male: yes
208Phase 3Germany;United Kingdom;Netherlands;Belgium;France;Spain;Italy;Sweden
38NCT00803205
(ClinicalTrials.gov)
September 8, 20094/12/2008Study of Ataluren (PTC124™) in Cystic FibrosisA Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisCystic FibrosisDrug: Ataluren;Drug: PlaceboPTC TherapeuticsCystic Fibrosis FoundationCompleted6 YearsN/AAll238Phase 3United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Spain;Sweden;United Kingdom
39EUCTR2008-003924-52-GB
(EUCTR)
26/08/200909/04/2009A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisA 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, IncNULLNot 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): noFrance;Spain;Belgium;Germany;Netherlands;Italy;United Kingdom;Sweden
40EUCTR2008-003924-52-DE
(EUCTR)
17/08/200910/07/2009A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
208Phase 3France;Spain;Belgium;Netherlands;Germany;Italy;United Kingdom;Sweden
41EUCTR2008-003924-52-IT
(EUCTR)
26/06/200922/06/2009A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis - PTC124-GD-009-CFA Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis - PTC124-GD-009-CF Nonsense-Mutation-Mediated Cystic Fibrosis
MedDRA version: 12.0;Level: LLT;Classification code 10011762;Term: Cystic fibrosis
Product Name: PTC124
Product Name: PTC124
Product Name: PTC124
PTC THERAPEUTICS, INC.NULLNot RecruitingFemale: yes
Male: yes
208Phase 3Germany;United Kingdom;Netherlands;Belgium;France;Spain;Italy;Sweden
42EUCTR2008-003924-52-FR
(EUCTR)
12/06/200917/03/2009A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisA 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: not assigned
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: not assigned
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: not assigned
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
PTC Therapeutics, IncNULLNot RecruitingFemale: yes
Male: yes
208Phase 3France;Spain;Belgium;Germany;Netherlands;Italy;United Kingdom;Sweden
43EUCTR2008-003924-52-ES
(EUCTR)
18/05/200909/03/2009Estudio de fase 3 sobre la eficacia y la seguridad de PTC124 como tratamiento oral para la fibrosis quística mediada por mutación terminadoraEstudio de fase 3 sobre la eficacia y la seguridad de PTC124 como tratamiento oral para la fibrosis quística mediada por mutación terminadora Fibrosis quística mediada por mutación terminadora
MedDRA version: 9.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: no asignado
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: no asignado
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: no asignado
Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844
PTC Therapeutics, IncNULLNot RecruitingFemale: yes
Male: yes
208Phase 3France;Belgium;Spain;Germany;Netherlands;Italy;United Kingdom;Sweden
44EUCTR2008-003924-52-BE
(EUCTR)
08/04/200920/02/2009Study of ataluren in patients with Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
208Phase 3France;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;Italy;United Kingdom;Sweden
45EUCTR2008-003924-52-SE
(EUCTR)
08/04/200917/12/2008A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisA 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, IncNULLNot RecruitingFemale: yes
Male: yes
208Phase 3France;Spain;Belgium;Germany;Netherlands;Italy;United Kingdom;Sweden
46EUCTR2007-000724-40-BE
(EUCTR)
27/09/200727/04/2007A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisA Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis Cystic Fibrosis
MedDRA version: 9.1;Level: LLT;Classification code 10011762;Term: Cystic fibrosis
Product Code: PTC124PTC Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
30Phase 2Belgium
47NCT00458341
(ClinicalTrials.gov)
March 23, 20076/4/2007A Study of Ataluren in Pediatric Participants With Cystic FibrosisA Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisCystic FibrosisDrug: AtalurenPTC TherapeuticsNULLCompleted6 Years18 YearsAll30Phase 2Belgium;France;Israel
48NCT00351078
(ClinicalTrials.gov)
December 200611/7/2006PTC124 for the Treatment of Cystic FibrosisA Phase 2b Extension Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisCystic FibrosisDrug: PTC124PTC TherapeuticsNULLCompleted18 YearsN/AAll19Phase 2Israel
49NCT00237380
(ClinicalTrials.gov)
November 30, 20057/10/2005Safety and Efficacy of Ataluren (PTC124) for Cystic FibrosisA Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisCystic FibrosisDrug: AtalurenPTC TherapeuticsNULLCompleted18 YearsN/AAll24Phase 2Israel
50NCT00234663
(ClinicalTrials.gov)
September 20055/10/2005PTC124 for Cystic FibrosisA Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic FibrosisCystic FibrosisDrug: PTC124PTC TherapeuticsCystic Fibrosis Foundation Therapeutics;FDA Office of Orphan Products DevelopmentCompleted18 YearsN/ABoth24Phase 2United States