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.