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.
This study was a planned extension study of PTC124-GD-028 ANI (NCT02647359) and was never initiated.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Oral
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: 104 weeks
Change From Baseline in Visual Acuity at Week 104
Time frame: 104 weeks
Change From Baseline in Severity of Corneal Keratopathy at Week 104
Time frame: 104 weeks
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