:Ataluren    (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2
113筋ジストロフィー51
140ドラベ症候群1
246メチルマロン酸血症5
299嚢胞性線維症42
329無虹彩症2

19. ライソゾーム病 [臨床試験数:784,薬物数:673(DrugBank:101),標的遺伝子数:68,標的パスウェイ数:184
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 784 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2015-003105-41-GB13/10/201508/09/2015Treatment for Nonsense Mutation Mucopolysaccharidosis Type ICNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS) - COMPASS Nonsense mutation Mucopolysaccharidosis Type I
MedDRA version: 18.1 Level: PT Classification code 10056886 Term: Mucopolysaccharidosis I System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
PTC Therapeutics Inc.NULLNot Recruiting Female: yes
Male: yes
5 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noUnited Kingdom
2EUCTR2014-002596-28-GB28/01/201512/06/2015Treatment for Nonsense Mutation Mucopolysaccharidosis Type IA Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I Nonsense mutation Mucopolysaccharidosis Type I
MedDRA version: 20.1Level: PTClassification code 10056886Term: Mucopolysaccharidosis ISystem Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
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
18Phase 2Germany;United Kingdom

113. 筋ジストロフィー [臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151
Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
51 / 567 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04336826June 1, 20213/4/2020A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular DystrophyNonsene Mutation Duchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLNot yet recruiting6 Months2 YearsMale6Phase 2NULL
2NCT03796637April 11, 20194/1/2019A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenPhase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for =9 MonthsDuchenne Muscular DystrophyOther: Dystrophin levels;Drug: AtalurenPTC TherapeuticsNULLCompletedN/AN/AMale6Phase 2United States
3NCT03648827December 21, 201824/8/2018A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With AtalurenDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLActive, not recruiting2 Years7 YearsMale20Phase 2United States
4EUCTR2017-001223-49-BG16/01/201811/10/2017A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular DystrophyA Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension Nonsense Mutation Duchenne Muscular Dystrophy
MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Translarna 125 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
340Phase 3United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of
5NCT03179631July 6, 20171/6/2017Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyA Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label ExtensionMuscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Disease;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, InbornDrug: Ataluren;Drug: PLACEBOPTC TherapeuticsNULLActive, not recruiting5 YearsN/AMale250Phase 3United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Puerto Rico;Russian Federation;Taiwan;Thailand;Turkey;Argentina;Chile;Jordan;Sri Lanka
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2013-005489-20-FR18/10/201618/06/2015Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.0 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0 Level: PT 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.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
7NCT02819557June 9, 201616/6/2016Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyA Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged =2 to <5 Years Old With Nonsense Mutation DystrophinopathyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLCompleted2 Years5 YearsMale14Phase 2United States
8EUCTR2013-005489-20-BG25/08/201526/06/2015Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem 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.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
9EUCTR2013-005489-20-CZ08/01/201524/10/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
10EUCTR2013-005489-20-DE29/09/201428/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in 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 Level: PT 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.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2013-005489-20-IT10/09/201430/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
12EUCTR2013-005489-20-ES01/09/201406/08/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
13EUCTR2013-005489-20-BE28/08/201411/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
14EUCTR2013-005489-20-GB22/08/201411/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in 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 Level: PT 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.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
15EUCTR2013-005489-20-SE22/08/201411/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in 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 Level: PT 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.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Sweden;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT02090959March 20, 201417/3/2014An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyA Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation DystrophinopathyMuscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, InbornDrug: AtalurenPTC TherapeuticsNULLTerminated7 Years15 YearsMale219Phase 3United States;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic
17EUCTR2012-004527-20-PL12/02/201427/11/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
18EUCTR2012-004527-20-SE30/08/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Sweden;Korea, Republic of
19EUCTR2012-004527-20-CZ30/08/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
20EUCTR2012-004527-20-DE20/08/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2012-004527-20-GB08/08/201324/06/2013 Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
PTC Therapeutics, IncNULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
22EUCTR2012-004527-20-BE30/07/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Belgium;Brazil;Australia;Germany;Korea, Republic of;Sweden
23EUCTR2012-004527-20-ES17/07/201312/07/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 16.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
24EUCTR2012-004527-20-IT01/07/201327/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 16.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
25NCT01826487March 26, 201326/3/2013Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation DystrophinopathyMuscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, InbornDrug: Ataluren;Drug: PlaceboPTC TherapeuticsNULLCompleted7 Years16 YearsMale230Phase 3United States;Australia;Belgium;Brazil;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Bulgaria;Czech Republic
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2011-004853-18-IT18/09/201227/09/2012An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation DystrophinopathyAn Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 15.0Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 15.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem 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: na
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: na
PTC THERAPEUTICS, INC.NULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;United Kingdom;Italy;Sweden
27EUCTR2011-004853-18-GB21/08/201229/03/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn 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 Level: PT 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.NULLNot Recruiting Female: no
Male: yes
96 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noFrance;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
28EUCTR2011-004853-18-ES13/06/201217/04/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden
29NCT01557400May 20, 201215/3/2012Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation DystrophinopathyDuchenne Muscular Dystrophy;Becker Muscular Dystrophy;DystrophinopathyDrug: AtalurenPTC TherapeuticsNULLCompletedN/AN/AMale94Phase 3Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
30EUCTR2011-004853-18-FR02/05/201213/08/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn 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 Level: PT 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, IncNULLNot Recruiting Female: no
Male: yes
96 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noFrance;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2011-004853-18-DE02/05/201212/03/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.1Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem 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.NULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
32EUCTR2011-004853-18-BE27/04/201203/01/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 16.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.1Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
33EUCTR2011-004853-18-SE26/04/201227/02/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1Level: PTClassification code 10059117Term: Becker's muscular dystrophySystem 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, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
34NCT01247207November 30, 201022/11/2010Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation DystrophinopathyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLEnrolling by invitationN/AN/AMale160Phase 3United States;Canada
35EUCTR2009-013169-24-GB19/01/201017/03/2010A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMDA Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
MedDRA version: 12.0Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 12.0Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: Ataluren 125 mg
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: Ataluren 250 mg
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: Ataluren 1000 mg
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics IncNULLNot RecruitingFemale: no
Male: yes
30Phase 2United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT01009294January 13, 20105/11/2009Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: Ataluren;Drug: Chronic Corticosteroid TherapyPTC TherapeuticsGenzyme, a Sanofi CompanyTerminated7 YearsN/AMale6Phase 2United States;United Kingdom
37EUCTR2008-007648-32-ES09/07/200904/05/2009Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora.Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora. Distrofia muscular de Duchenne, Distrofia muscular de Becker
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
174United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
38EUCTR2008-007648-32-DE22/06/200908/04/2009A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
174Phase 2France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden
39EUCTR2008-007648-32-GB22/06/200929/04/2009A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
174Phase 2Germany;United Kingdom;Belgium;France;Spain;Italy;Sweden
40EUCTR2008-007648-32-SE02/06/200917/04/2009A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
174Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41EUCTR2008-007648-32-FR28/05/200903/04/2009A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
174Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
42EUCTR2008-007648-32-IT27/05/200922/05/2009A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - NDA Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801
MedDRA version: 9.1Level: LLTClassification code 10059117
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: no
Male: yes
174Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
43EUCTR2008-007648-32-BE14/04/200925/02/2009A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
174Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
44NCT00847379January 31, 200916/2/2009Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: AtalurenPTC TherapeuticsGenzyme, a Sanofi CompanyTerminated5 YearsN/AMale173Phase 2United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
45NCT00759876August 13, 200823/9/2008Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)A Phase 2a Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedN/AN/AMale36Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46EUCTR2007-005478-29-SE16/04/200822/02/2008A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
174Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
47EUCTR2007-005478-29-DE14/04/200828/01/2008A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
174Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
48EUCTR2007-005478-29-BE11/03/200827/02/2008A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
174Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
49NCT00592553February 29, 20081/1/2008Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: Ataluren;Drug: PlaceboPTC TherapeuticsNULLCompleted5 YearsN/AMale174Phase 2United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
50EUCTR2012-004527-20-FR25/09/2015 Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 18.0 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0 Level: PT 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, IncNULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51EUCTR2017-001223-49-PL05/06/2018A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular DystrophyA Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension Nonsense Mutation Duchenne Muscular Dystrophy
MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Translarna 125 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
PTC Therapeutics, Inc.NULLNAFemale: no
Male: yes
340Phase 3United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Poland;Brazil;Malaysia;Australia;Bulgaria;China;Japan;Korea, Republic of

140. ドラベ症候群 [臨床試験数:83,薬物数:54(DrugBank:13),標的遺伝子数:48,標的パスウェイ数:62
Searched query = "Dorabe syndrome", "Dravet syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 83 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02758626November 20164/4/2016Ataluren for Nonsense Mutation in CDKL5 and Dravet SyndromeA Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of Ataluren for Drug Resistant Epilepsy in Patients With Nonsense Mutation CDKL5 or Dravet SyndromeEpilepsyDrug: ataluren;Drug: PlaceboNYU Langone HealthPTC TherapeuticsActive, not recruiting2 Years12 YearsAll16Phase 2United States

246. メチルマロン酸血症 [臨床試験数:14,薬物数:22(DrugBank:2),標的遺伝子数:1,標的パスウェイ数:6
Searched query = "Methylmalonic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
5 / 14 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2009-016654-41-IT11/11/201005/07/2010A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia - NDA Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia - ND Nonsense Mutation Methylmalonic Acidemia
MedDRA version: 12.1Level: LLTClassification code 10064180Term: Blood methylmalonic acid increased
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
18Phase 2France;United Kingdom;Italy
2EUCTR2009-016654-41-BE04/11/201022/12/2010Study of ataluren in patients with Methylmalonic AcidemiaA Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia Nonsense Mutation Methylmalonic Acidemia (MMA)
MedDRA version: 14.0Level: LLTClassification code 10064180Term: Blood methylmalonic acid increasedSystem 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.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2France;Belgium;Italy;United Kingdom;Switzerland
3NCT01141075July 19, 20107/6/2010Ataluren for Nonsense Mutation Methylmalonic AcidemiaA Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic AcidemiaAmino Acid Metabolism, Inborn ErrorsDrug: AtalurenPTC TherapeuticsGenzyme, a Sanofi CompanyTerminated2 YearsN/AAll11Phase 2Belgium;France;Germany;Italy;Switzerland;United Kingdom
4EUCTR2009-016654-41-GB01/06/201017/03/2010A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic AcidemiaA Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia Nonsense Mutation Methylmalonic Acidemia
MedDRA version: 12.1 Level: LLT Classification code 10064180 Term: Blood methylmalonic acid increased
PTC Therapeutics, Inc.NULLNot Recruiting Female: yes
Male: yes
18 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance;Belgium;Italy;United Kingdom
5EUCTR2009-016654-41-FR02/03/201004/01/2010A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic AcidemiaA Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia Nonsense Mutation Methylmalonic Acidemia
MedDRA version: 12.1Level: LLTClassification code 10064180Term: Blood methylmalonic acid increased
Product Name: Ataluren 125mg
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 250mg
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 1000mg
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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Phase 2United Kingdom;France;Italy

299. 嚢胞性線維症 [臨床試験数:1,592,薬物数:1,539(DrugBank:255),標的遺伝子数:81,標的パスウェイ数:162
Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
42 / 1,592 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03256799March 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
2NCT03256968January 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-GR05/04/201608/02/2016Confirmatory 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: 18.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-GB22/03/201615/02/2016Confirmatory 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]
PTC Therapeutics, Inc.NULLNot Recruiting Female: yes
Male: yes
200 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noUnited States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany
5EUCTR2014-005355-83-BG19/02/201616/12/2015Confirmatory 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: 18.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2014-005355-83-NL22/01/201625/11/2015Confirmatory 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: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-FR08/12/201504/12/2015Confirmatory 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: 18.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-IT16/10/201526/08/2015Confirmatory 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: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-ES08/10/201502/09/2015Confirmatory 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: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-BE01/10/201524/08/2015Confirmatory 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: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2013-004581-34-BE01/10/201531/07/2014Confirmatory 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: 17.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
12NCT02456103August 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-FR25/08/201518/06/2015Extension 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: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-GR29/07/201523/07/2015Confirmatory 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: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-NL26/02/201506/08/2014Confirmatory 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: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
208Phase 3United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2013-004581-34-BG05/02/201506/01/2015Confirmatory 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: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-DE25/11/201405/08/2014Confirmatory 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: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-DE01/10/201404/08/2014Extension 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: 17.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-IT16/09/201425/06/2014Extension 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: 17.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
76Phase 3France;United States;Belgium;Spain;Israel;Germany;Italy;Sweden
20EUCTR2013-004581-34-IT15/09/201410/06/2014Confirmatory 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: 17.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2013-004581-34-GB12/09/201425/09/2014Confirmatory 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: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
22EUCTR2013-004581-34-ES01/09/201414/08/2014Confirmatory 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: 17.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
23EUCTR2013-005449-35-ES01/09/201411/08/2014Extension 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: 17.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
24EUCTR2013-005449-35-BE26/08/201404/06/2014Extension 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: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-SE06/08/201405/06/2014Extension 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: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT02139306August 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
27NCT02107859May 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-ES07/09/201129/07/2011Extension 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: 14.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
200Phase 3Germany;Canada;Netherlands;Belgium;France;Spain;Italy;United States;Sweden;Israel
29EUCTR2010-019692-30-GB22/08/201123/08/2011Extension 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]
PTC Therapeutics, IncNULLNot Recruiting Female: yes
Male: yes
200 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noFrance;United States;Canada;Spain;Belgium;Israel;Germany;Netherlands;Italy;United Kingdom;Sweden
30EUCTR2010-019692-30-DE11/05/201113/04/2011Extension 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: 14.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2010-019692-30-SE16/12/201011/10/2010Extension 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: 14.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-NL04/11/201024/06/2010Extension 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: 14.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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-IT06/10/201002/11/2010A 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: 9.1Level: LLTClassification 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-FR05/10/201017/06/2010A 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: 12.1Level: LLTClassification code 10011762Term: 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
208Phase 3Germany;Netherlands;Belgium;France;Spain;Italy;Sweden
35NCT01140451August 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2010-019692-30-BE10/08/201025/05/2010Extension 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: 14.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
37NCT00803205September 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
38EUCTR2008-003924-52-DE17/08/200910/07/2009A 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: 9.1Level: LLTClassification code 10011762Term: 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
39EUCTR2008-003924-52-BE08/04/200920/02/2009Study 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: 14.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem 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
40EUCTR2008-003924-52-SE08/04/200917/12/2008A 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: 9.1Level: LLTClassification code 10011762Term: 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41NCT00458341March 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
42NCT00237380November 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

329. 無虹彩症 [臨床試験数:3,薬物数:2(DrugBank:2),標的遺伝子数:0,標的パスウェイ数:0]
Searched query = "Aniridia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 3 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04117880December 31, 201819/9/2019A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation AniridiaNonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension StudyAniridiaDrug: AtalurenPTC TherapeuticsNULLWithdrawn2 YearsN/AAll0Phase 2NULL
2NCT02647359January 31, 201629/12/2015Study of Ataluren in Participants With Nonsense Mutation AniridiaSTAR: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation AniridiaAniridiaDrug: Ataluren;Drug: PlaceboPTC TherapeuticsNULLActive, not recruiting2 YearsN/AAll39Phase 2United States;Canada