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

Aniridia Clinical Trial

Official title:
Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study

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

Clinical Trial Details
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Status Withdrawn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
View Details
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