Analysis of outcomes
SECONDARY
All analyses and summaries were produced using SAS version 9.4 or higher. Primary and key secondary efficacy endpoints were tested under a hierarchical procedure to maintain the type I error for the comparison between the high-dose group and the control group. Nominal Analysis of the primary endpoint was based on the intent-to-treat population, defined as all participants who were randomized, completed visit 2 (that is, received the study treatment or received a phone call (if in control group)) and had at least one post-treatment BCVA measurement. Change from baseline BCVA score was compared between the study groups (that is, high dose versus control and low dose versus control) using Fisher’s exact test supported by a Fisher’s exact Boschloo test with a Berger–Boos correction of beta = 0.001, in which the reported Key secondary efficacy endpoints were also based on the intent-to-treat population. Change from baseline in BCVA at 12 months, a key secondary endpoint, was summarized over time using descriptive statistics (that is, mean, s.d. and 95% CI) and evaluated by analysis of covariance (ANCOVA) with missing data handled by the last observation carried forward approach. Fisher’s exact test was used to analyze the proportion of participants with a ≥10-letter improvement from baseline in BCVA at month 12 measured by the ETDRS chart and the proportion of participants with no decrease from baseline in BCVA or a decrease from baseline in BCVA of <5 ETDRS letters at 12 months, respectively.