Phase 2 Proof-of-Concept Study for ataluren in Nonsense Mutation Aniridia
PTC Therapeutics’ STAR study is a Phase 2 proof-of-concept study of ataluren, an orally administered, first-in-class, protein restoration therapy for the treatment of nonsense mutation aniridia, a rare genetic disorder that results in disruption in the development of the eye.
The STAR study is a multicenter, randomized, double-masked, placebo-controlled study of the safety and efficacy of ataluren in patients with nonsense mutation aniridia over 96 weeks. The primary objective of this study is to characterize the systemic and ocular safety profile of ataluren in aniridia patients.
Ataluren will be studied in patients with a clinical diagnosis of aniridia who are 2 years and older with a body weight equal to or greater than 12 kg and who have a nonsense mutation in 1 allele of the PAX6 gene.
Participating trial sites include University of Virginia; Casey Eye Institute, Oregon Health & Science University and University of British Columbia.
For additional information on the study or if you are interested in being considered for the study, please consult with your doctor and visit this page
for trial site contact information.
for more background on this study.