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E.S. Gragoudas; VEGF Inhibition Study in Ocular Neovascularization–1 (VISION–1): Efficacy Results From Phase II/III MacugenTM (Pegaptanib Sodium) Clinical Trials . Invest. Ophthalmol. Vis. Sci. 2004;45(13):2364.
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© ARVO (1962-2015); The Authors (2016-present)
Purpose: To evaluate the efficacy of Macugen, a selective vascular endothelial growth factor–165 (VEGF165) antagonist in the treatment of neovascular age–related macular degeneration (AMD). Methods: Two pivotal phase II/III multicenter, randomized, double–masked trials in patients with all subtypes of neovascular AMD were performed. Wide vision and entry criteria were established. Intravitreal Macugen (0.3 mg, 1 mg, or 3 mg) or sham injection was given every 6 weeks for 54 weeks. The proportion of patients losing <15 letters of visual acuity by week 54 was the primary efficacy endpoint; secondary efficacy endpoints also were evaluated. Results: 1186 patients were randomized evenly among groups and included in analyses. Baseline demographic and ocular characteristics were similar across groups; 92% of patients completed the study. In combined analyses, the primary endpoint was met with statistical significance for all Macugen doses (P<.05 for each dose). The 0.3 mg dose was the lowest effective dose and was more effective than sham in both individual trials (P<.05 for each trial). A larger proportion of patients treated with 0.3 mg Macugen compared with sham lost less than 3 lines of vision (70% versus 55%; P<.05). Macugen was beneficial for all lesion subtypes in analyses of the 0.3 mg and sham groups. For secondary endpoints, combined analyses of both trials of the 0.3 mg Macugen versus sham groups found 1) at week 54, larger proportions of Macugen patients gained >15 letters of visual acuity (6% versus 2%; P<.05) and either maintained baseline vision or gained vision (33% versus 23%; P<.05), and 2) at weeks 6, 12, and 54, mean visual acuity loss from baseline was lower in the Macugen group (P<.05 for each time period). Severe vision loss (>6 lines) occurred in 55% fewer of 0.3 mg Macugen– than sham–treated patients at week 54. No evidence that photodynamic therapy usage modulated the treatment effect of Macugen was found. Conclusion: With wide entry criteria for vision and lesion size, Macugen appears to be an effective treatment for all subtypes of AMD.
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