Table 5.
IB1001-301 EP Demographics and Baseline Characteristics
| Parameter | Total (n = 53) |
| Age, n (%) | |
| Pediatric (<18 y) | 22 (41.5) |
| Adult (≥18 y) | 31 (58.5) |
| Sex, n (%) | |
| Female | 24 (45.3) |
| Male | 29 (54.7) |
| Race, n (%) | |
| American Indian or Alaska Native | 0 (0.0) |
| Asian | 2 (3.8) |
| Black or African American | 0 (0.0) |
| Native Hawaiian or other Pacific Islander | 0 (0.0) |
| White | 47 (88.7) |
| Other | 4 (7.5) |
| Age at diagnosis group, n (%) | |
| Early-infantile (<2 y) | 9 (17.0) |
| Late-infantile (2–<6 y) | 13 (24.5) |
| Juvenile (6–<15 y) | 21 (39.6) |
| Adolescent/adult (≥15 y) | 10 (18.9) |
| Dose group, n (%) | |
| Age 4–12 y: 15–<25 kg: 2 g/d | 5 (9.4) |
| Age 4–12 y: 25–<35 kg: 3 g/d | 3 (5.7) |
| Age 4–12 y: ≥35 kg or age ≥13 y: 4 g/d | 45 (84.9) |
| Miglustat at baseline, n (%)a | |
| Yes | 45 (84.9) |
| No | 8 (15.1) |
Abbreviation: EP = extension phase.
a
Indicates concurrent miglustat use throughout the duration of the trial.