A Phase 2/3, Multicenter, Randomized, Double-Masked, 2-Year Trial of Pegaptanib Sodium for the Treatment of Diabetic Macular Edema

Purpose To confirm the safety and compare the efficacy of intravitreal pegaptanib sodium 0.3 mg versus sham injections in subjects with diabetic macular edema (DME) involving the center of the macula associated with vision loss not due to ischemia. Design Randomized (1:1), sham-controlled, multicenter, parallel-group trial. Participants Subjects with DME. Intervention Subjects received pegaptanib 0.3 mg or sham injections every 6 weeks in year 1 (total = 9 injections) and could receive focal/grid photocoagulation beginning at week 18. During year 2, subjects received injections as often as every 6 weeks per prespecified criteria. Main Outcome Measures The primary efficacy endpoint was the proportion gaining ≥10 letters of visual acuity (VA) from baseline to year 1. Safety was monitored throughout. Results In all, 260 (pegaptanib, n=133; sham, n=127) and 207 (pegaptanib, n=107; sham, n=100) subjects were included in years 1 and 2 intent-to-treat analyses, respectively. A total of 49 of the 133 (36.8%) subjects from the pegaptanib group and 25 of the 127 (19.7%) from the sham group experienced a VA improvement of ≥10 letters at week 54 compared with baseline (odds ratio [OR], 2.38; 95% confidence interval, 1.32–4.30; P = 0.0047). For pegaptanib-treated subjects, change in mean VA from baseline by visit was superior ( P P P = 0.002; week 102, 27/107 [25.2%] vs 45/100 [45.0%], respectively, P = 0.003). The pegaptanib treatment group showed significantly better results on the National Eye Institute–Visual Functioning Questionnaire than sham for subscales important in this population. Pegaptanib was well tolerated; the frequencies of discontinuations, adverse events, treatment-related adverse events, and serious adverse events were comparable in the pegaptanib and sham groups. Conclusions Patients with DME derive clinical benefit from treatment with the selective vascular endothelial growth factor antagonist pegaptanib 0.3 mg. These findings indicate that intravitreal pegaptanib is effective in the treatment of DME and, taken together with prior study data, support a positive safety profile in this population. Financial Disclosure(s) Proprietary or commercial disclosure may be found after the references.