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"
38 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-001223-49-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
2EUCTR2013-005489-20-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
3EUCTR2013-005489-20-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
4EUCTR2013-005489-20-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
5EUCTR2013-005489-20-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
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-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
7EUCTR2012-004527-20-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
8EUCTR2012-004527-20-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
9EUCTR2012-004527-20-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
10EUCTR2012-004527-20-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
11EUCTR2012-004527-20-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
12EUCTR2012-004527-20-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
13EUCTR2012-004527-20-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
14EUCTR2011-004853-18-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
15EUCTR2011-004853-18-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2011-004853-18-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
17EUCTR2011-004853-18-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
18EUCTR2011-004853-18-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
19EUCTR2009-013169-24-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
20EUCTR2008-007648-32-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2008-007648-32-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
22EUCTR2008-007648-32-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
23EUCTR2008-007648-32-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
24EUCTR2008-007648-32-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
25EUCTR2008-007648-32-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2007-005478-29-GB27/04/200922/02/2008A 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 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
174 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance;Spain;Belgium;Germany;Italy;United Kingdom;Sweden
27EUCTR2008-007648-32-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
28NCT00847379January 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
29NCT00759876August 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
30EUCTR2007-005478-29-IT03/06/200814/07/2008A 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: 9.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophy
Product Code: PTC124PTC THERAPEUTICS, INC.NULLNot RecruitingFemale: no
Male: yes
165Phase 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
31EUCTR2007-005478-29-ES20/05/200810/03/2008Estudio 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: 9.1Level: 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: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
165United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
32EUCTR2007-005478-29-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
33EUCTR2007-005478-29-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
34EUCTR2007-005478-29-FR17/03/200824/01/2008A 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: 9.1Level: LLTClassification code 10013801Term: Duchenne muscular dystrophy
MedDRA version: 9.1Level: LLTClassification code 10059117Term: Becker's muscular dystrophy
Product Name: PTC124
Product Code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
165Phase 2United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
35EUCTR2007-005478-29-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT00592553February 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
37NCT00264888December 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
38EUCTR2017-001223-49-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