Table 1.
Baseline characteristics of subjects enrolled in Part 2.
| Age groups |
Total (n = 65) | ||||
|---|---|---|---|---|---|
| Characteristic | <1 year (n = 11) | 1 to < 2 years (n = 11) | 2 to < 4 years (n = 23) | 4 to < 7 years (n = 20) | |
| Age (years) at enrollment, mean (SD) | 0.44 (0.28) | 1.37 (0.33) | 3.03 (0.67) | 5.62 (0.95) | 3.11 (2.04) |
| Sex, N (%) | |||||
| Female | 6 (54.5%) | 5 (45.5%) | 15 (65.2%) | 14 (70.0%) | 40 (61.5%) |
| Male | 5 (45.5%) | 6 (54.5%) | 8 (34.8%) | 6 (30.0%) | 25 (38.5%) |
| Race, N (%) | |||||
| White | 9 (81.8%) | 9 (81.8%) | 18 (78.3%) | 18 (90.0%) | 54 (83.1%) |
| Asian | 1 (9.1%) | 1 (9.1%) | 0 | 1 (5.0%) | 3 (4.6%) |
| Black or African American | 0 | 0 | 1 (4.3%) | 0 | 1 (1.5%) |
| Other | 1 (9.1%) 1 | 1 (9.1%) | 4 (17.4%) | 1 (5.0%) | 7 (10.8%) |
| Ethnicity | |||||
| Not Hispanic or Latino | 11 (100.0%) | 11 (100.0%) | 20 (87.0%) | 20 (100.0%) | 62 (95.4%) |
| Hispanic or Latino | 0 | 0 | 3 (13.0%) | 0 | 3 (4.6%) |
| Weight (z-score), mean (SD) | 0.25 (0.94) | 0.00 (0.86) | 0.48 (0.75) | 0.69 (0.77) | 0.42 (0.83) |
| Height (z-score), mean (SD) | 0.21 (1.15) | 0.47 (0.72) | 0.41 (1.05) | 0.47 (0.91) | 0.41 (0.96) |
| Head circumference (z-score), mean (SD) | −0.01 (0.66) | 0.38 (1.01) | 0.48 (1.25) | NA (NA) | 0.28 (0.99) |
| Blood phenylalanine concentration (μmol/L), mean (SD) | 298.41 (119.67) | 366.50 (107.17) | 337.02 (135.47) | 332.02 (158.81) | 333.93 (135.03) |
N: number of patients with data available for each variable; NA: not applicable; SD: standard deviation;
z-score was calculated based on the World Health Organization (WHO) growth charts for children < 24 months and Centers for Disease Control and Prevention (CDC) clinical growth charts for children > 24 months. Values within 1 SD of the population mean are within the expected growth percentiles.