Table 4Summary of Baseline Demographic and Disease Characteristics (Intention-to-Treat Population)

PKU-016SPARK
SAP + Diet
N = 98		Placebo + Diet
N = 108		SAP + Diet
N = 27		Diet Alone
N = 29
Gender, n (%)
Female
Male		41 (41.8)
57 (58.2)		54 (50.0)
54 (50.0)		11 (40.7)
16 (59.3)		15 (51.7)
14 (48.3)
Race, n (%)
White
Black
Asian		96 (98.0)
0
1 (1.0)		102 (94.4)
2 (1.9)
1 (0.9)		▬▬▬▬
▬▬
▬		▬▬▬
▬
▬▬
Age (Years or Months)a
Mean (SD)
Median (min, max)
< 18 years, n (%)
≥ 18 years, n (%)
< 12 months, n (%)
12 to < 24 months, n (%)
24 to < 48 months, n (%)		23.6 years (12.7)
▬▬▬▬▬
▬▬▬
▬▬▬▬
▬▬▬▬
▬▬
▬		22.5 years (10.4)
▬▬▬▬
▬▬▬
▬▬▬
▬
▬
▬		21.1 months (12.3)
▬▬▬
▬
▬
▬
▬▬
▬▬▬▬		21.2 months (12.0)
▬▬▬
▬
▬
▬
▬▬
▬▬▬▬
Standing Height (cm)
Mean (SD)
Median (min, max)		▬▬
▬▬▬▬		▬▬
▬▬▬▬		82.0 (11.3)
▬▬		82.3 (11.6)
▬▬
Weight (kg)
Mean (SD)
Median (min, max)		▬▬
▬▬▬
▬		▬▬
▬▬▬▬		11.3 (3.1)
▬▬		11.3 (2.8)
▬▬
ADHD Symptoms, n (%)
Yes
No		▬▬
▬▬		▬▬
▬▬		NR
NR		NR
NR
ADHD Medication, n (%)b
Yes
No		▬▬
▬▬▬		▬▬
▬▬▬		NR
NR		NR
NR
Phe Blood Level (μmol/L)c
Mean (SD)
Median (min, max)		▬▬▬▬
▬▬▬
▬		▬▬▬▬
▬▬▬▬
▬		780.3 (480.7)
▬▬▬)		879.9 (596.5)
▬▬▬
PKU Severity, n (%)
Classic
Mild
Moderate		NR
NR
NR		NR
NR
NR		▬▬
▬▬▬
▬▬▬		▬▬
▬▬▬
▬▬

ADHD = attention-deficit/hyperactivity disorder; ITT = intention-to-treat; NR = not reported; Phe = phenylalanine; PKU = phenylketonuria; SAP = sapropterin; SD = standard deviation.

a

In PKU-016, age is reported in years and in the SPARK study, age is reported in months.

b

In PKU-016, Phe blood level is at baseline and in the SPARK study it is at diagnosis (i.e., because patients had to have Phe levels within the target range of 120 to 360 μmol/L to enter the SPARK study).

c

ln PKU-016, 5 patients did not have a baseline Phe blood level and were included as non-Phe responders for the primary efficacy population.

Source: Clinical Study Report (CSR) PKU-016,10 CSR SPARK study.11

From: 4, Results

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