PTC Therapeutics: Muscular Dystrophy, Duchenne trials

The registry lists 19 Muscular Dystrophy, Duchenne studies with PTC Therapeutics as lead sponsor. 0 are open to participants now and 13 have results posted.

Phase 1 2Phase 2 9Phase 3 6No phase 2(a study in two phases counts in both)

Studies

Open to participants first, 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
• 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
• 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
• Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)TerminatedNCT01009294Phase 2Results posted
• Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)TerminatedNCT00847379Phase 2Results 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 Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)WithdrawnNCT03642145Phase 3
• Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular DystrophyApproved for marketingNCT02592941
• Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)CompletedNCT02369731
• An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability DeflazacortCompletedNCT02295748Phase 1
• A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyCompletedNCT02251600Phase 1
• Safety and Efficacy Study of PTC124 in Duchenne Muscular DystrophyCompletedNCT00264888Phase 2