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Table 1 Participant demographic and baseline characteristics

From: Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study

 

Phase 2 participants (N = 80)

Age at enrollment

 Mean (SD), years

28.3 (8.8)

 Min, max, years

16, 56

  < 18, n (%)

3 (3.8%)

  ≥ 18, n (%)

77 (96.3%)

Sex

 Female, n (%)

46 (57.5%)

Race

 White, n (%)

78 (97.5%)

Weight

n = 79

 Mean (SD), kg

80.4 (24.5)

 Min, max, kg

42.0, 178.0

Height

n = 74

 Mean (SD), cm

167.2 (9.6)

 Min, max, cm

149.8, 187.5

Body mass index

n = 74

 Mean (SD), kg/m2

28.5 (7.7)

 Min, max, kg/m2

17.2, 56.2

  < 25 kg/m2, n (%)

28 (35.0%)

 25 to < 30 kg/m2, n (%)

21 (26.3%)

  ≥ 30 kg/m2, n (%)

25 (31.3%)

Plasma Phe

 Mean (SD), μmol/L

1302.4 (351.5)

 Min, max, μmol/L

249.0, 2214.0

Protein intakea

n = 36

 Mean (SD), g/day

69.4 (40.0)

 Min, max, g/day

10.7, 197.3

Phe intakeb

n = 36

 Mean (SD), mg/day

1975 (1583)

 Min, max, mg/day

461, 8419

  1. Baseline was defined as the last measurement before the first dose of pegvaliase (i.e., while patients were treatment naïve) in the parent study. All phase 2 data are included. Sample size is indicated if N < 80
  2. max maximum, min minimum, Phe phenylalanine, SD standard deviation
  3. aProtein intake includes medical food and natural protein dietary intake and was calculated as the daily average intake over 3 days prior to the assessment point
  4. bPhe intake was calculated as the daily average intake over 3 days prior to the assessment point
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172