These trials completed more than 12 months ago and should have reported results. Some have, some have not.

These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.

These trials have problematic data on the registry. Details why »

Status Trial ID Title Completion date Category
Completed, but no date 2007-000724-40 A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis bad-data
Listed as ongoing, but also has a completion date 2007-005478-29 A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy 2009-12-17 bad-data
Listed as ongoing, but also has a completion date 2008-003924-52 A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis 2011-11-12 bad-data
Listed as ongoing, but also has a completion date 2008-007648-32 A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy 2010-03-18 bad-data
Not reported 2009-013169-24 A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy 2010-03-03 due-trials
Listed as ongoing, but also has a completion date 2009-016654-41 A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia 2014-04-22 bad-data
Listed as ongoing, but also has a completion date 2010-019692-30 A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis 2013-12-02 bad-data
Ongoing 2010-020224-22 A Phase 2a Study of Ataluren (PTC124) as an Oral Treatment for Nonsense-Mutation-Mediated Hemophilia A and B not-yet-due
Listed as ongoing, but also has a completion date 2011-004853-18 An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy 2018-01-19 bad-data
Listed as ongoing, but also has a completion date 2012-004527-20 A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy 2015-08-20 bad-data
Listed as ongoing, but also has a completion date 2013-004581-34 A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis 2016-11-02 bad-data
Listed as ongoing, but also has a completion date 2013-005449-35 An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) 2017-06-05 bad-data
Listed as ongoing, but also has a completion date 2013-005489-20 A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy 2018-06-21 bad-data
Listed as ongoing, but also has a completion date and reported results 2014-002596-28 A Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I 2017-07-20 bad-data
Listed as ongoing, but also has a completion date 2014-005355-83 A Phase 3 extension Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis 2017-06-02 bad-data
Ongoing 2015-003105-41 CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS) not-yet-due
Other 2017-001223-49 A 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 not-yet-due