An open-label, one-year, noncomparative study to evaluate the safety and tolerability of intravitreal pegaptanib sodium in patients with diabetic macular edema

BACKGROUND: The purpose of this study was to evaluate the safety and tolerability of pegaptanib in patients with diabetic macular edema. METHODS: An open-label, multicenter, noncomparative, one-year study of approximately 500 patients was planned. Recruitment was terminated after enrollment of 46 patients. Enrolled patients were fully informed and reconsented; 12 patients elected to complete the study. Patients received intravitreal injections of pegaptanib 0.3 mg once every 6 weeks or less frequently, as determined by the investigator. Clinical benefit was evaluated after the patient received two or more injections. Ocular and nonocular adverse events were closely monitored throughout the study. RESULTS: Compared with baseline, mean best-corrected visual acuity increased by week 6. Ten patients reported ocular-related adverse events, none of which were severe, and eight patients reported nonocular adverse events, two of which were severe but unrelated to study treatment. Three serious adverse events, unrelated to study treatment, were reported. CONCLUSION: In this limited set of patients with diabetic macular edema, pegaptanib appeared to be well tolerated with evidence of efficacy.