12 completed, 5 terminated, 1 recruiting, 1 withdrawn. 19 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 19 | Completed 12, Terminated 5, Recruiting 1, Withdrawn 1 |
| Phase 1 | 1 | Recruiting 1 |
Follow ataluren: new trials by email Literature Review of ataluren trials Research Report on ataluren
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| 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) Ataluren - Nonsene Mutation Duchenne Muscular Dystrophy | Phase 2 | Completed | PTC Therapeutics | 6 | 2021-12-29 | 2024-04-01 |
| Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study Ataluren + Pembrolizumab - Colorectal Cancer, Endometrium Cancer | Phase 1 / Phase 2 | Recruiting | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | 47 | 2019-08-01 | 2023-02-01 |
| A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren Ataluren - Duchenne Muscular Dystrophy | Phase 2 | Completed | PTC Therapeutics | 6 | 2019-04-11 | 2022-04-05 |
| A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia Ataluren - Aniridia | Phase 2 | Withdrawn | PTC Therapeutics | 2018-12-31 | 2019-10-07 | |
| A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Ataluren - Duchenne Muscular Dystrophy | Phase 2 | Completed | PTC Therapeutics | 20 | 2018-12-21 | 2022-04-05 |
| Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome ataluren, Placebo - Epilepsy | Phase 2 | Completed | NYU Langone Health | 15 | 2016-11-01 | 2023-02-16 |
| Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy Ataluren - Duchenne Muscular Dystrophy | Phase 2 | Completed | PTC Therapeutics | 14 | 2016-06-09 | 2020-08-28 |
| Study of Ataluren in Participants With Nonsense Mutation Aniridia Ataluren, Placebo - Aniridia | Phase 2 | Completed | PTC Therapeutics | 39 | 2016-01-31 | 2022-05-27 |
| Ataluren for Nonsense Mutation Methylmalonic Acidemia Ataluren - Amino Acid Metabolism, Inborn Errors | Phase 2 | Terminated | PTC Therapeutics | 11 | 2010-07-19 | 2020-07-07 |
| Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) Ataluren, Chronic Corticosteroid Therapy - Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Phase 2 | Terminated | PTC Therapeutics | 6 | 2010-01-13 | 2020-07-29 |
| Study of Ataluren (PTC124) in Hemophilia A and B Ataluren - Hemophilia A, Hemophilia B | Phase 2 | Terminated | PTC Therapeutics | 13 | 2009-10-14 | 2020-06-16 |
| Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) Ataluren - Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Phase 2 | Terminated | PTC Therapeutics | 173 | 2009-01-31 | 2020-07-15 |
| Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) Ataluren - Duchenne Muscular Dystrophy | Phase 2 | Terminated | PTC Therapeutics | 36 | 2008-08-13 | 2020-10-29 |
| Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) Ataluren, Placebo - Duchenne Muscular Dystrophy, Becker Muscular Dystrophy | Phase 2 | Completed | PTC Therapeutics | 174 | 2008-02-29 | 2020-04-07 |
| A Study of Ataluren in Pediatric Participants With Cystic Fibrosis Ataluren - Cystic Fibrosis | Phase 2 | Completed | PTC Therapeutics | 30 | 2007-03-23 | 2020-03-06 |
| PTC124 for the Treatment of Cystic Fibrosis PTC124 - Cystic Fibrosis | Phase 2 | Completed | PTC Therapeutics | 19 | 2006-12-01 | 2017-12-13 |
| Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy PTC124 - Duchenne Muscular Dystrophy | Phase 2 | Completed | PTC Therapeutics | 38 | 2005-12-01 | 2009-01-14 |
| Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis Ataluren - Cystic Fibrosis | Phase 2 | Completed | PTC Therapeutics | 24 | 2005-11-30 | 2020-06-11 |
| PTC124 for Cystic Fibrosis PTC124 - Cystic Fibrosis | Phase 2 | Completed | PTC Therapeutics | 24 | 2005-09-01 | 2009-01-13 |
Muscular Dystrophy, Duchenne (8) · Cystic Fibrosis (4) · Aniridia (2) · Amino Acid Metabolism, Inborn Errors (1) · Colorectal Neoplasms (1) · Endometrial Neoplasms (1) · Epilepsy (1) · Hemophilia A (1) · Hemophilia B (1) · Neoplasm Metastasis (1)
PTC Therapeutics (17) · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) (1) · NYU Langone Health (1)
United States (13) · France (5) · Belgium (4) · Canada (4) · Israel (4) · Italy (4) · United Kingdom (4) · Germany (3) · Australia (2) · Spain (2) · Sweden (2) · Netherlands (1) · Switzerland (1)
Francesco Bibbiani, MD (3) · Jay Barth, MD (2) · Leone Atkinson, MD, PhD (2) · Adriaan D Bins, MD PhD (1) · Eitan Kerem, MD (1) · Eiten Kerem, MD (1) · Isabelle Sermet-Gaudelus, MD (1) · John P Clancy, MD (1) · Leone Atkinson (1) · Leone Atkinson, M.D., Ph.D. (1) · Orrin Devinsky, MD (1) · Quintus Ngumah, OD, PhD (1) · Richard Finkel, MD (1) · Vinay Penematsa (1)
PMID 18722008, PMID 20622033, PMID 21233271, PMID 24349052, PMID 32851872, PMID 33538404, PMID 34693725, PMID 37236246, PMID 40983668
2019 (2), 2009 (1), 2017 (1)