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, and reported results 2007-000724-40 A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis bad-data
Reported results 2007-005478-29 A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy 2009-12-31 due-trials
Reported results 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 due-trials
Reported results 2008-007648-32 A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy 2010-03-18 due-trials
Reported results Terminated 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
Reported results 2009-016654-41 A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia 2014-04-22 due-trials
Reported results 2010-019692-30 A Phase 3 Extension Study of Ataluren (PTC124) in Subjects with Nonsense-Mutation-Mediated Cystic Fibrosis 2013-12-02 due-trials
Reported results 2010-020224-22 A Phase 2a Study of Ataluren (PTC124) as an Oral Treatment for Nonsense-Mutation-Mediated Hemophilia A and B 2011-08-30 due-trials
Reported results 2011-004853-18 An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy STUDIO IN APERTO SU PAZIENTI TRATTATI IN PRECEDENZA CON ATALUREN (PTC124) AFFETTI DA D... 2018-01-19 due-trials
Reported results 2012-004527-20 A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy 2015-10-03 due-trials
Listed as ongoing, but also has a completion date and reported results 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
Reported results 2013-005449-35 An Open-Label Safety and Efficacy Study for Patients with Nonsense Mutation Cystic Fibrosis Previously Treated with Ataluren (PTC124®) 2018-06-05 due-trials
Reported results 2013-005489-20 A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy 2018-06-21 due-trials
Reported results 2014-002596-28 A Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I 2017-07-20 due-trials
Reported results 2014-005355-83 A Phase 3 extension Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis 2017-06-02 due-trials
Reported results Terminated 2015-003105-41 CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS) 2017-03-09 due-trials
Trial is outside EEC, and reported results 2016-001764-11 A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy bad-data
Not reported 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 2023-07-21 due-trials
Reported results 2018-004740-36 A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I 2021-01-15 due-trials
Trial is outside EEC, and reported results 2019-001691-11 Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for ≥9 Months bad-data
Trial is outside EEC, and reported results 2019-001767-67 Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment with Ataluren bad-data
Trial is outside EEC, and reported results 2020-000980-21 An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy bad-data
Reported results 2020-001872-13 Evaluation of the efficacy and safety of PTC299 in hospitalized subjects with COVID-19 (FITE19) 2022-07-21 due-trials
Listed as ongoing, but also has a completion date and reported results 2020-002100-39 Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy (MIT-E) 2023-12-27 bad-data
Listed as ongoing, but also has a completion date and reported results 2020-002812-36 A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich ataxia (MOVE-FA) 2023-10-02 bad-data
Listed as ongoing, but also has a completion date and reported results 2021-000474-29 A Phase 3 Study of PTC923 in Subjects with Phenylketonuria 2023-04-03 bad-data
Other 2021-000497-28 Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria not-yet-due
Other 2021-003852-18 A PHASE 2A, RANDOMIZED, PLACEBO-CONTROLLED, DOSE-RANGING STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PTC518 IN SUBJECTS WITH HUNTINGTON'S DISEASE not-yet-due
Other 2021-006511-29 A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL STUDY TO ASSESS THE EFFICACY, SAFETY, TOLERABILITY, PK, AND BIOMARKER EFFECTS OF PTC857 IN ADULT SUBJECTS WITH AMYOTROPHIC LATERAL SCL... not-yet-due
Completed, report not yet due 2022-000073-12 A PHASE 2/3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF UNESBULIN IN UNRESECTABLE OR METASTATIC, RELAPSED OR REFRACTORY LEIOMYOSARCOMA 2024-10-03 not-yet-due
Listed as ongoing, but also has a completion date 2022-000375-39 AN OPEN-LABEL, SAFETY STUDY FOR PREVIOUSLY TREATED VATIQUINONE (PTC743) SUBJECTS WITH INHERITED MITOCHONDRIAL DISEASE 2024-08-29 bad-data
Trial is outside EEC, and reported results 2022-001013-39 A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia bad-data
Other 2022-002668-65 Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia not-yet-due
Trial is outside EEC 2022-003265-38 An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age bad-data