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Clinical Trial 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.


Clinical Trial Description

This study was a planned extension study of PTC124-GD-028 ANI (NCT02647359) and was never initiated. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04117880
Study type Interventional
Source PTC Therapeutics
Contact
Status Withdrawn
Phase Phase 2
Start date December 31, 2018
Completion date January 31, 2021

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