113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details


臨床試験数 : 622 薬物数 : 485 - (DrugBank : 99) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 168

  
53 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1JPRN-jRCT2041200087
07/01/202125/12/2020An Open-label, Long-Term study of Ataluren in Nonsense Mutation Duchenne Muscular DystrophyAn Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy - PTC124-GD-049-DMD Nonsense Mutation Duchenne Muscular DystrophySubjects will receive ataluren tid as an oral suspension at respective morning, mid-day, and evening doses of
10 mg/kg, 10 mg/kg, and 20 mg/kg.
Penematsa VinayNULLRecruiting>= 5age oldNot applicableMale25Phase 3China;Japan
2EUCTR2017-001223-49-BG
(EUCTR)
16/01/201811/10/2017A 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: 20.0;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]
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 3Korea, Republic of;Japan;China;Bulgaria;Australia;Poland;Malaysia;Brazil;Argentina;Jordan;Canada;Mexico;India;Russian Federation;Turkey;Thailand;Hong Kong;Taiwan;United States
3EUCTR2013-005489-20-FR
(EUCTR)
18/10/201618/06/2015Extension 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;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic 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
4NCT02819557
(ClinicalTrials.gov)
June 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
5EUCTR2013-005489-20-BG
(EUCTR)
25/08/201526/06/2015Extension 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: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.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
6EUCTR2013-005489-20-CZ
(EUCTR)
08/01/201524/10/2014Extension 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: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.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
7EUCTR2013-005489-20-DE
(EUCTR)
29/09/201428/07/2014Extension 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;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic 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
8EUCTR2013-005489-20-IT
(EUCTR)
10/09/201430/07/2014Extension 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: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.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-ES
(EUCTR)
01/09/201406/08/2014Extension 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: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.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
10EUCTR2013-005489-20-BE
(EUCTR)
28/08/201411/07/2014Extension 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: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.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
11EUCTR2013-005489-20-SE
(EUCTR)
22/08/201411/07/2014Extension 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;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic 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
12EUCTR2013-005489-20-GB
(EUCTR)
22/08/201411/07/2014Extension 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;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic 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
13NCT02090959
(ClinicalTrials.gov)
March 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
14EUCTR2012-004527-20-PL
(EUCTR)
12/02/201427/11/2013Study 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: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, 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
15EUCTR2012-004527-20-SE
(EUCTR)
30/08/201324/06/2013Study 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: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, 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
16EUCTR2012-004527-20-CZ
(EUCTR)
30/08/201324/06/2013Study 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: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, 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
17EUCTR2012-004527-20-DE
(EUCTR)
20/08/201324/06/2013Study 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: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, 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-GB
(EUCTR)
08/08/201324/06/2013 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;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic 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
19EUCTR2012-004527-20-BE
(EUCTR)
30/07/201324/06/2013Study 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: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, 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
20EUCTR2012-004527-20-ES
(EUCTR)
17/07/201312/07/2013Study 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: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Sweden;Korea, Republic of;Germany;Australia;Belgium;Brazil;Poland;Canada;Czech Republic;Switzerland
21EUCTR2012-004527-20-IT
(EUCTR)
01/07/201327/06/2013Study 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: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, 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
22EUCTR2011-004853-18-IT
(EUCTR)
18/09/201227/09/2012An 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: 15.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 15.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: 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
23EUCTR2011-004853-18-GB
(EUCTR)
21/08/201229/03/2012Study 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;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
96 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
24EUCTR2011-004853-18-ES
(EUCTR)
13/06/201217/04/2012Study 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: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden
25NCT01557400
(ClinicalTrials.gov)
May 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
26EUCTR2011-004853-18-FR
(EUCTR)
02/05/201213/08/2012Study 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;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot Recruiting Female: no
Male: yes
96 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
27EUCTR2011-004853-18-DE
(EUCTR)
02/05/201212/03/2012Study 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: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
28EUCTR2011-004853-18-BE
(EUCTR)
27/04/201203/01/2012Study 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: 16.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
29EUCTR2011-004853-18-SE
(EUCTR)
26/04/201227/02/2012Study 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: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
30NCT01247207
(ClinicalTrials.gov)
November 30, 201022/11/2010Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation DystrophinopathyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLEnrolling by invitationN/AN/AMale270Phase 3United States;Canada
31EUCTR2009-013169-24-GB
(EUCTR)
19/01/201017/03/2010A 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: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 12.0;Classification code 10059117;Term: 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
32NCT01009294
(ClinicalTrials.gov)
January 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
33EUCTR2008-007648-32-ES
(EUCTR)
09/07/200904/05/2009Un 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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;Belgium;Spain;Germany;Italy;United Kingdom;Sweden
34EUCTR2008-007648-32-GB
(EUCTR)
22/06/200929/04/2009A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
35EUCTR2008-007648-32-DE
(EUCTR)
22/06/200908/04/2009A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
36EUCTR2008-007648-32-SE
(EUCTR)
02/06/200917/04/2009A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
37EUCTR2008-007648-32-FR
(EUCTR)
28/05/200903/04/2009A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
38EUCTR2008-007648-32-IT
(EUCTR)
27/05/200922/05/2009A 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: 9.1;Level: LLT;Classification code 10013801
MedDRA version: 9.1;Classification 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
39EUCTR2007-005478-29-GB
(EUCTR)
27/04/200922/02/2008A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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 Recruiting Female: no
Male: yes
174 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Spain;Belgium;Germany;Italy;United Kingdom;Sweden
40EUCTR2008-007648-32-BE
(EUCTR)
14/04/200925/02/2009A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
41NCT00847379
(ClinicalTrials.gov)
January 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
42NCT00759876
(ClinicalTrials.gov)
August 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
43EUCTR2007-005478-29-IT
(EUCTR)
03/06/200814/07/2008A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - NDA Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - ND Duchenne Muscular Dystrophy, Becker Muscular Dystrophy.
MedDRA version: 9.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy
Product Code: PTC124PTC THERAPEUTICS, INC.NULLNot RecruitingFemale: no
Male: yes
165Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
44EUCTR2007-005478-29-ES
(EUCTR)
20/05/200810/03/2008Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadoraEstudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadora Distrofia muscular de Duchenne, Distrofia muscular de Becker
MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
165Phase 2France;Belgium;Spain;Germany;Italy;United Kingdom;Sweden
45EUCTR2007-005478-29-SE
(EUCTR)
16/04/200822/02/2008A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
46EUCTR2007-005478-29-DE
(EUCTR)
14/04/200828/01/2008A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
47EUCTR2007-005478-29-FR
(EUCTR)
17/03/200824/01/2008A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy
Product Name: PTC124
Product Code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
165Phase 2France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden
48EUCTR2007-005478-29-BE
(EUCTR)
11/03/200827/02/2008A 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: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: 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
49NCT00592553
(ClinicalTrials.gov)
February 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
50NCT00264888
(ClinicalTrials.gov)
December 20059/12/2005Safety and Efficacy Study of PTC124 in Duchenne Muscular DystrophyA Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PTC124PTC TherapeuticsMuscular Dystrophy AssociationCompleted5 YearsN/AMale38Phase 2United States
51EUCTR2012-004527-20-FR
(EUCTR)
25/09/2015 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;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic 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
52EUCTR2020-000980-21-Outside-EU/EEA
(EUCTR)
24/06/2021A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Subjects 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 Dystrophy Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD);Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Trade Name: Translarna
Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: Ataluren
PTC Therapeutics, Inc.NULLNAFemale: no
Male: yes
Phase 2United States
53EUCTR2017-001223-49-PL
(EUCTR)
05/06/2018A 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: 20.0;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]
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