Table 1.
Patient demographics and baseline characteristics (safety analysis set)
| Follow-up cohort (n = 4) | Expansion cohort (n = 15) | All patients (n = 19) | |
|---|---|---|---|
| Mean age ± SD, years | 34.3 ± 5.5 | 37.5 ± 9.0 | 36.8 ± 8.4 |
| Median (range) | 35 (29–39) | 36 (25–52) | 36 (25–52) |
| Female:male, n | 3:1 | 11:4 | 14:5 |
| Race, n (%) | |||
| Caucasian | 4 (100.0) | 11 (73.3) | 15 (78.9) |
| Other | 0 | 4 (26.7) | 4 (21.1) |
| Median time since diagnosis (range), months | 82.5 (57.6–100.3) | 63.4 (12.2–155.2) | 70.2 (12.2–155.2) |
| Previous surgery, n (%) | 4 (100.0) | 13 (86.7) | 17 (89.5) |
| Mean baseline UFC ± SD, nmol/24 ha | 398 ± 176b | 1630 ± 3043 | 1371 ± 2734 |
SD standard deviation
aNormal range: 11–138 nmol/24 h
b3/4 patients had UFC > 1.5 × ULN at enrollment