Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
PTC Therapeutics
The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).
Age
6–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.
You may not be if
Known hypersensitivity to any of the ingredients or excipients of the study drug.
Ongoing participation in any other therapeutic clinical trial.
Clareo Health | Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
