Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study
Trial Snapshot
- Phase
- Phase 2
- Status
- Withdrawn
- Sponsor
- PTC Therapeutics
- Primary Endpoint
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Study Overview
Brief 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.
Detailed Description
This study was a planned extension study of PTC124-GD-028 ANI (NCT02647359) and was never initiated.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 2 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Ataluren
Ataluren Oral suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20 mg/kg in the evening).
Intervention: Ataluren (Drug)
Outcomes
Primary Outcomes
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time Frame: 104 weeks
Secondary Outcomes
- Change From Baseline in Severity of Corneal Keratopathy at Week 104(104 weeks)
- Change From Baseline in Visual Acuity at Week 104(104 weeks)