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Table 2 Demographic and baseline characteristics (ITT population)

From: Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial

Characteristic Sapropterin + Phe- restricted diet (n = 27) Phe-restricted diet only (n = 29)
Age, months
 mean±SD 21.1±12.3 21.2±12.0
 min; max 2; 47 2; 44
Age group, n (%)
 <12 months 7 (25.9) 8 (27.6)
 12 − <24 months 9 (33.3) 9 (31.0)
 24 − <48 months 11 (40.7) 12 (41.4)
 Males, n (%) 16 (59.3) 14 (48.3)
Height, cm
 mean±SD 82.0±11.3 82.3±11.6
 min; max 59; 108 57; 105
Weight, kg
 mean±SD 11.3±3.1 11.3±2.8
 min; max 5; 20 6; 16
BMI, kg/m2
 mean±SD 16.5±1.0 16.5±1.4
 min; max 14; 18 14; 20
Age at PKU diagnosis, days
 mean±SD 27.2±79.8 32.6±72.2
 min; max 1; 425 4; 382
Blood Phe concentration at diagnosis, μmol/L
 mean±SD 780.3±480.7 879.9 ± 596.5
 min; max 191; 2062 221; 2600
PKU severitya n (%)
 Classical PKU 5 (18.5) 7 (24.1)
 Mild PKU 10 (37.0) 8 (27.6)
 Mild HPA 12 (44.4) 14 (48.3)
  1. BMI body mass index, HPA hyperphenylalaninemia, ITT intention-to-treat, Phe phenylalanine, PKU phenylketonuria, SD standard deviation
  2. aDisease severity according to blood Phe concentrations: Classical PKU, >1200 μmol/L; Mild PKU, 600–1200 μmol/L; Mild HPA, 120–600 μmol/L2, 5, 6