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 μm (P = 0.0034) at 6 months and by 109.3 ± 19.5 (P = 0.0004) and 166.6 ± 38.4 μm (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.