A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

Summary

This is a Phase 2, multicenter, open-label study evaluating the overall systemic and ocular safety profile of ataluren in nonsense mutation aniridia as determined by the incidences of treatment-emergent adverse events (TEAEs) as well as abnormal findings on laboratory assessments, vital signs, physical examinations, ophthalmoscopy, and slit-lamp examination. Participants who complete PTC124-GD-028 ANI (NCT02647359) meet all inclusion and none of the exclusion criteria will be enrolled into this study.

Timeline

Start

2018-12-31

Primary completion

2021-01-31

Completion

2021-01-31

Drugs

Indications