An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
Status:
Terminated
Trial end date:
2018-06-12
Target enrollment:
Participant gender:
Summary
The primary objective of this study is to obtain long term safety data of ataluren in male
participants with nonsense mutation dystrophinopathy (who participated and completed a
previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall
safety database. Screening and baseline procedures are structured to avoid a gap in treatment
between the double-blind study (PTC124-GD-020-DMD) and this extension study.
This study may be further extended by amendment until either ataluren becomes commercially
available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is
discontinued.