Ataluren: all 32 studies that test it

Page 1 of 1. Open to participants first (0), then studies with results posted, then the rest; newest registration first within each.

• 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)CompletedNCT04336826Phase 2Results posted
• A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenCompletedNCT03796637Phase 2Results posted
• A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT03648827Phase 2Results posted
• PTC Study to Evaluate Ataluren in Combination With IvacaftorCompletedNCT03256968Phase 4Results posted
• Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense MutationsCompletedNCT03256799Phase 4Results posted
• Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyCompletedNCT03179631Phase 3Results posted
• Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyCompletedNCT02819557Phase 2Results posted
• Ataluren for Nonsense Mutation in CDKL5 and Dravet SyndromeCompletedNCT02758626Phase 2Results posted
• Study of Ataluren in Participants With Nonsense Mutation AniridiaCompletedNCT02647359Phase 2Results posted
• Extension Study of Ataluren in Participants With Nonsense Mutation Cystic FibrosisTerminatedNCT02456103Phase 3Results posted
• Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)CompletedNCT02139306Phase 3Results posted
• Study of Ataluren (PTC124) in Cystic FibrosisTerminatedNCT02107859Phase 3Results posted
• An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyTerminatedNCT02090959Phase 3Results posted
• Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT01826487Phase 3Results posted
• Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaCompletedNCT01557400Phase 3Results posted
• Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)TerminatedNCT01247207Phase 3Results posted
• Ataluren for Nonsense Mutation Methylmalonic AcidemiaTerminatedNCT01141075Phase 2Results posted
• Extension Study of Ataluren (PTC124) in Cystic FibrosisCompletedNCT01140451Phase 3Results posted
• Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)TerminatedNCT01009294Phase 2Results posted
• Study of Ataluren (PTC124) in Hemophilia A and BTerminatedNCT00947193Phase 2Results posted
• Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)TerminatedNCT00847379Phase 2Results posted
• Study of Ataluren (PTC124™) in Cystic FibrosisCompletedNCT00803205Phase 3Results posted
• Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)TerminatedNCT00759876Phase 2Results posted
• Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)CompletedNCT00592553Phase 2Results posted
• A Study of Ataluren in Pediatric Participants With Cystic FibrosisCompletedNCT00458341Phase 2Results posted
• A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation AniridiaWithdrawnNCT04117880Phase 2
• Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO StudyStatus unknownNCT04014530Phase 1/2
• Effects of Rifampin on the Pharmacokinetics of AtalurenCompletedNCT02409004Phase 1
• PTC124 for the Treatment of Cystic FibrosisCompletedNCT00351078Phase 2
• Safety and Efficacy Study of PTC124 in Duchenne Muscular DystrophyCompletedNCT00264888Phase 2
• Safety and Efficacy of Ataluren (PTC124) for Cystic FibrosisCompletedNCT00237380Phase 2
• PTC124 for Cystic FibrosisCompletedNCT00234663Phase 2

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