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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
51 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