Randomized Trial to Evaluate Tandosporine in Geographic Atrophy Secondary to Age-Related Macular Degeneration: the GATE Study.

PURPOSE To determine the safety and efficacy of AL-8309B (tandospirone) in the management of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) and obtain standardized data on GA lesion growth progression. DESIGN Prospective, controlled, double-masked, randomized, multicenter phase 3 clinical trial. METHODS Setting: 48 clinical sites. PATIENTS Patients with GA associated with AMD were enrolled. All patients were followed for a minimum of 30 months, and up to 36 months. Intervention Procedures: Patients were randomized (1:1:1) to receive AL-8309B ophthalmic solution 1.0%, 1.75%, or vehicle, administered as a twice-daily topical ocular drop. MAIN OUTCOME MEASURES The primary efficacy endpoint was mean annualized lesion enlargement from baseline as assessed with fundus autofluorescence (FAF) imaging. RESULTS A total of 768 eyes of 768 patients were enrolled and treated with AL-8309B 1.0% (N=250), AL-8309B 1.75% (N=258), or vehicle (N= 260). An increase in mean lesion size was observed in both the AL-8309B and vehicle treatment groups, and growth rates were similar in all treatment groups. Annualized lesion growth rates were 1.73, 1.76 and 1.71 mm(2) for AL-8309B 1.0%, AL-8309B 1.75%, and vehicle, respectively. CONCLUSIONS AL-8309B 1.0% and 1.75% did not affect lesion growth in eyes with GA secondary to AMD. There were no clinically relevant safety issues identified for AL-8309B. The large natural history dataset from this study is a valuable repository for future comparisons.