CFTR-G542X nonsense allele inhib 
Welcome,         Profile    Billing    Logout  
 1 Company  1 Product   1 Product   36 Diseases   5 Trials   696 News 
7 Trials

   

 
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Translarna (ataluren) / PTC Therap
2017-001223-49: A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy

Ongoing
3
250
Europe
ataluren, PTC124, Granules in sachet, Translarna 125 mg granules for oral suspension, Translarna 250 mg granules for oral suspension, Translarna 1000 mg granules for oral suspension
PTC Therapeutics, Inc., PTC Therapeutics, Inc.
Nonsense Mutation Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy, Diseases [C] - Musculoskeletal Diseases [C05]
 
 
NCT03179631: Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Completed
3
360
Europe, Canada, Japan, US, RoW
Ataluren, PTC124, PLACEBO, Matching Placebo
PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
03/22
07/23
NCT01247207: Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

Enrolling by invitation
3
270
Canada, US
Ataluren, PTC124, Translarna
PTC Therapeutics
Duchenne Muscular Dystrophy
04/25
04/25
2015-003105-41: Treatment for Nonsense Mutation Mucopolysaccharidosis Type I

Ongoing
2
5
Europe
ataluren, PTC124, Powder for oral suspension
PTC Therapeutics Inc., PTC Therapeutics Inc.
Nonsense mutation Mucopolysaccharidosis Type I, Mucopolysaccharidosis I, Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT04336826 / 2020-000980-21: A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Completed
2
6
US
Ataluren, PTC124
PTC Therapeutics
Nonsene Mutation Duchenne Muscular Dystrophy
08/23
08/23
ATAPEMBRO, NCT04014530 / 2017-004752-34: Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the Study

Recruiting
1/2
47
Europe
Ataluren + Pembrolizumab
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA), Merck Sharp & Dohme LLC, PTC Therapeutics
Colorectal Cancer, Endometrium Cancer
06/23
08/23
NCT02369731: Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Checkmark Presentation of data from STRIDE trial at WMS 2019
Oct 2019 - Oct 2019: Presentation of data from STRIDE trial at WMS 2019
Active, not recruiting
N/A
316
Europe, RoW
PTC Therapeutics, The John Walton Muscular Dystrophy Research Centre (TREAT-NMD), Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Duchenne
05/25
05/25
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Translarna (ataluren) / PTC Therap
2017-001223-49: A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy

Ongoing
3
250
Europe
ataluren, PTC124, Granules in sachet, Translarna 125 mg granules for oral suspension, Translarna 250 mg granules for oral suspension, Translarna 1000 mg granules for oral suspension
PTC Therapeutics, Inc., PTC Therapeutics, Inc.
Nonsense Mutation Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy, Diseases [C] - Musculoskeletal Diseases [C05]
 
 
NCT03179631: Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Completed
3
360
Europe, Canada, Japan, US, RoW
Ataluren, PTC124, PLACEBO, Matching Placebo
PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
03/22
07/23
NCT01247207: Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

Enrolling by invitation
3
270
Canada, US
Ataluren, PTC124, Translarna
PTC Therapeutics
Duchenne Muscular Dystrophy
04/25
04/25
2015-003105-41: Treatment for Nonsense Mutation Mucopolysaccharidosis Type I

Ongoing
2
5
Europe
ataluren, PTC124, Powder for oral suspension
PTC Therapeutics Inc., PTC Therapeutics Inc.
Nonsense mutation Mucopolysaccharidosis Type I, Mucopolysaccharidosis I, Diseases [C] - Nutritional and Metabolic Diseases [C18]
 
 
NCT04336826 / 2020-000980-21: A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Completed
2
6
US
Ataluren, PTC124
PTC Therapeutics
Nonsene Mutation Duchenne Muscular Dystrophy
08/23
08/23
ATAPEMBRO, NCT04014530 / 2017-004752-34: Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the Study

Recruiting
1/2
47
Europe
Ataluren + Pembrolizumab
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA), Merck Sharp & Dohme LLC, PTC Therapeutics
Colorectal Cancer, Endometrium Cancer
06/23
08/23
NCT02369731: Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Checkmark Presentation of data from STRIDE trial at WMS 2019
Oct 2019 - Oct 2019: Presentation of data from STRIDE trial at WMS 2019
Active, not recruiting
N/A
316
Europe, RoW
PTC Therapeutics, The John Walton Muscular Dystrophy Research Centre (TREAT-NMD), Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Duchenne
05/25
05/25

Download Options