Ranibizumab for Choroidal Neovascularization Secondary to Causes Other Than Age-Related Macular Degeneration: A Phase I Clinical Trial

Purpose: To investigate the safety and efficacy of ranibizumab for the treatment of choroidal neovascularization (CNV) secondary to causes other than age-related macular degeneration (AMD). Design: Multicenter, randomized, 12-month clinical trial. Participants: Thirty patients with CNV due to causes other than AMD, including pathologic myopia, ocular histoplasmosis, and angioid streaks. Methods: Participants were randomly assigned 1: 1 to monthly intravitreal injections of 0.5 mg ranibizumab or 3 monthly injections followed by dosing as needed (pro re nata [PRN]) at monthly follow-up visits. Main Outcome Measures: Outcome measures included the incidence of ocular and nonocular adverse events, the percentage of patients gaining >= 15 letters of visual acuity (VA) at 6 and 12 months from baseline, the percentage of patients losing >= 15 letters of VA at 6 and 12 months from baseline, and mean change in VA and central retinal thickness (CRT) at 6 and 12 months from baseline. Results: No serious ocular or systemic adverse events related to ranibizumab or the injection procedure were reported. Among patients who received monthly ranibizumab injections, 8 of 12 (66.7%) gained >= 15 letters of VA at both 6 and 12 months, and among patients who received PRN injections, 9 of 14 (64.3%) and 8 of 14 (57.1%) gained >= 15 letters of VA at 6 and 12 months, respectively. No patient in the study lost >= 15 letters of VA. Mean VA improved significantly from baseline by 23.9 +/- 5.4 (P = 0.001) and 21.1 +/- 4.3 letters (P = 0.0003) at 6 months and by 26.9 +/- 5.3 (P = 0.0003) and 19.2 +/- 3.8 letters (P = 0.0002) at 12 months in the monthly and PRN treatment arms, respectively. Mean CRT decreased significantly from baseline by 103.4 +/- 18.9 (P = 0.0005) and 161.1 +/- 43.7 mu m (P = 0.0034) at 6 months and by 109.3 +/- 19.5 (P = 0.0004) and 166.6 +/- 38.4 mu m (P = 0.001) at 12 months in the monthly and PRN treatment groups, respectively. No statistically significant difference was found between treatment groups in change in VA or CRT at any time point. Conclusions: Intravitreal ranibizumab, given as monthly injections or as 3 monthly injections followed by PRN dosing, showed a promising efficacy and safety profile in the treatment of CNV due to causes other than AMD.