Table 1.
Baseline demographics and disease characteristics (ITT population)
| Statistic | TARM1 Bilateral treatment |
TARM2 Unilateral treatment |
Total | |
|---|---|---|---|---|
| (N = 48) | (N = 50) | (N = 98) | ||
| Age, years | Mean (SD) | 32.4 (14.4) | 31.9 (13.4) | 32.1 (13.8) |
| Median | 27.0 | 29.5 | 28.5 | |
| Min, Max | 15, 74 | 15, 65 | 15, 74 | |
| Gender | ||||
| Male | n (%) | 37 (77.1) | 41 (82.0) | 78 (79.6) |
| Female | n (%) | 11 (22.9) | 9 (18.0) | 20 (20.4) |
| Region | ||||
| Asia | n (%) | 8 (16.7) | 7 (14.0) | 15 (15.3) |
| Europe | n (%) | 11 (22.9) | 16 (32.0) | 27 (27.6) |
| USA | n (%) | 29 (60.4) | 27 (54.0) | 56 (57.1) |
| Affected eye status a | ||||
| Bilateral | n (%) | 47 (97.9) | 50 (100.0) | 97 (99.0) |
| Unilateral | n (%) | 1 (2.1) | 0 | 1 (1.0) |
| Duration of disease, months | n | 48 | 50 | 98 |
| Mean (SD) | 8.33 (3.36) | 8.27 (3.09) | 8.30 (3.20) | |
| Median | 8.85 | 8.85 | 8.85 | |
| Min, Max | 1.7, 11.9 | 2.4, 11.9 | 1.7, 11.9 | |
| Time interval of vision loss between first and second-affected eyes, days | n | 48 | 50 | 98 |
| Mean (SD) | 56.85 (66.34) | 61.88 (54.08) | 59.42 (60.14) | |
| Median | 33.50 | 59.50 | 46.00 | |
| Min, Max | 0.0; 266 | 0.0; 197 | 0.0; 266 | |
| Baseline LogMAR BCVA of first-affected eye | n | 48 | 50 | 98 |
| Mean (SD) | 1.59 (0.47) | 1.68 (0.43) | 1.64 (0.45) | |
| Median | 1.50 | 1.60 | 1.60 | |
| Min, Max | 0.6; 2.3 | 0.8; 2.3 | 0.6; 2.3 | |
| Baseline LogMAR BCVA of second/not-yet-affected eye | n | 48 | 50 | 98 |
| Mean (SD) | 1.44 (0.51) | 1.50 (0.46) | 1.47 (0.48) | |
| Median | 1.40 | 1.50 | 1.40 | |
| Min, Max | 0.0; 2.3 | 0.7; 2.3 | 0.0; 2.3 |
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. N = number of patients in the ITT population; n = number of patients; % = percentage of patients calculated relative to N.
Bilateral status is defined as LogMAR > 0 for both eyes on the day of injection. Unilateral status is defined as LogMAR = 0 for one of the two eyes on the day of injection.