Clinical Trial: Study of Ataluren in Patients With Aniridia
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: STAR: A Phase 2, Multicenter, Randomized, Double Masked, Placebo Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia<
Brief Summary: This is a Phase 2, randomized, double-masked, placebo-controlled study of ataluren in patients with aniridia caused by a nonsense mutation. Patients will receive masked study drug for 48 weeks followed by open-label ataluren for another 48 weeks. Safety and efficacy will be assessed.
Detailed Summary:
Sponsor: PTC Therapeutics
Current Primary Outcome: Safety as assessed by systemic and ocular adverse events [ Time Frame: 96 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Visual acuity as assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart [ Time Frame: 48 weeks ]
- Severity of corneal keratopathy as assessed by slit lamp examination and macro photography [ Time Frame: 48 weeks ]
- Iris area [ Time Frame: 48 weeks ]
Original Secondary Outcome: Same as current
Information By: PTC Therapeutics
Dates:
Date Received: December 29, 2015
Date Started: January 2016
Date Completion: June 2018
Last Updated: December 13, 2016
Last Verified: December 2016
