Table 2.
First-affected and second-affected/not-yet-affected eyes: change from baseline to 1.5 years in LogMAR BCVA—observed data and ANCOVA analysis (ITT population)
| TARM1 (Bilateral treatment) | TARM2 (Unilateral treatment) | TARM1 (Bilateral treatment) | TARM2 (Unilateral treatment) | ||
|---|---|---|---|---|---|
| First-affected eye | Second/not-yet-affected eye | ||||
| Statistic | Lenadogene nolparvovec (N = 48) |
Lenadogene nolparvovec (N = 50) |
Lenadogene nolparvovec (N = 48) |
Placebo (N = 50) |
|
| Baseline | n | 48 | 50 | 48 | 50 |
| Mean (SD) | 1.59 (0.470) | 1.68 (0.432) | 1.44 (0.512) | 1.50 (0.455) | |
| Year 1.5 (Visit 12) | n a | 48 | 50 | 48 | 50 |
| Observed | Mean (SD) | 1.34 (0.527) | 1.46 (0.587) | 1.35 (0.585) | 1.45 (0.604) |
| Change from baseline (observed) | Mean (SD) | −0.25 (0.468) | −0.22 (0.412) | −0.08 (0.568) | −0.05 (0.467) |
| Estimates from ANCOVAb | LS mean (SE) [95% CI] |
−0.26 (0.063) [−0.38, −0.13] |
−0.21 (0.061) [−0.33, −0.09] |
−0.09 (0.072) [−0.24, 0.05] |
−0.04 (0.071) [−0.18, 0.10] |
|
P-value H0: LS mean = 0 |
<0.0001 | 0.0008 | 0.1955 | 0.5536 | |
| Lenadogene nolparvovec versus placebo | LS mean difference (SE) [95% CI] | − |
−0.05 (0.101) [−0.25, 0.15] |
||
| P-value | − | 0.6080 | |||
TARM1: First-affected eye and second-affected/not-yet-affected eye administered lenadogene nolparvovec. TARM2: First-affected eye administered lenadogene nolparvovec, second-affected/not-yet-affected eye administered placebo. For patients whose LogMAR evaluation date was out of the visit 12 window (518–578 days post-treatment), the first LogMAR value after visit 12 (578 days post-treatment) was used. CI = confidence interval; N = number of patients in the ITT population; n = number of patients; SE = standard error.
Missing data were imputed using the last observation carried Fforward method.
An ANCOVA model was used to model change from baseline to 1.5 years (visit 12) for LogMAR BCVA, using baseline LogMAR as covariate and treatment as fixed effect.