Jagjit Singh Gilhotra1, Brendon Vote2, Philip Hykin3, Timothy Lai4, Paolo Lanzetta5,6, Sabine Desset-Brethes7, Harry Staines7, Shaio Hui Melissa Liew8
Purpose: To evaluate the efficacy and safety of ranibizumab 0.5 mg (RBZ) in adult patients with visual impairment due to choroidal neovascularization (CNV) associated with rare diseases. We report the 12-month results of the MINERVA study.
Methods: MINERVA was a 12month, doublemasked, shamcontrolled, randomized study. 178 adult patients were randomized (2:1) to receive RBZ 0.5 mg (n=119) or sham (n=59) at baseline, and if needed at Month 1. From Month 2 (M2), patients in both arms could receive open-label RBZ as needed. The primary objective was to demonstrate superior efficacy of RBZ versus sham (change in best-corrected visual acuity [BCVA] from baseline to M2).
Results: 167 patients (93.8%) completed the study. Baseline characteristics were balanced between groups. The primary endpoint was met: RBZ showed superior efficacy versus sham at M2 (mean change in BCVA from baseline: +9.5 vs -0.4 letters? p<0.001). Secondary outcomes included the mean changes in BCVA letters/injections at M12 (+11.0/5.8 for RBZ, versus +9.3/5.4 for sham), the mean change in CSFT from baseline to M2 (-77.0 µm for RBZ vs +9.8 µm for sham? p<0.001) and the proportions of patients gaining ?10/?15 letters at M2 and M12 (respectively 42.4%/31.4% and 56.8%/48.7% for RBZ, vs 14.0%/12.3% and 50.9%/41.8% for sham). No ocular or drug-related SAEs were reported up to M12.
Conclusions: RBZ treatment resulted in a treatment effect of 9.9 letters gained at M2 (p<0.001). The BCVA gain was maintained up to M12. There were no new safety findings in comparison to the well-established safety profile of RBZ.