|
Timepoint
|
Statistics
|
‘sapropterin continuous’ (n=25)
|
‘sapropterin extension’ (n = 26)
|
|---|
|
Age
|
|---|
|
< 12 Months
|
12–< 24 Months
|
24–< 48 Months
|
< 12 Months
|
12–< 24 Months
|
24–< 48 Months
|
|---|
|
Full scale IQ (Wechsler scale of intelligence)
| | | | | | | |
|
Baseline
|
n (missing)
|
0 (7)
|
0 (9)
|
2 (7)
|
0 (8)
|
1 (9)
|
0 (8)
|
| |
Mean (SD)
|
–
|
–
|
115.50 (17.68)
|
–
|
124.00
|
–
|
|
Month 12
|
n (missing)
|
0 (7)
|
0 (9)
|
9 (0)
|
0 (8)
|
7 (3)
|
0 (8)
|
| |
Mean (SD)
|
–
|
–
|
110.44 (17.58)
|
–
|
111.43 (26.22)
|
–
|
|
Month 24
|
n (missing)
|
0 (7)
|
3 (6)
|
4 (5)
|
0 (8)
|
4 (6)
|
5 (3)
|
| |
Mean (SD)
|
–
|
98.00 (20.07)
|
105.50 (12.12)
|
–
|
115.25 (7.27)
|
94.40 (19.69)
|
|
EOS
|
n (missing)
|
6 (1)
|
1 (8)
|
4 (5)
|
5 (3)
|
5 (5)
|
4 (4)
|
| |
Mean (SD)
|
120.67 (14.67)
|
94.00
|
95.25 (8.26)
|
108.60 (14.81)
|
108.20 (14.75)
|
88.25 (14.17)
|
|
Performance IQ (Wechsler scale of intelligence)
| | | | | | | |
|
Baseline
|
n (missing)
|
0 (7)
|
0 (9)
|
2 (7)
|
0 (8)
|
1 (9)
|
0 (8)
|
| |
Mean (SD)
|
–
|
–
| |
–
|
114.00
|
–
|
|
Month 12
|
n (missing)
|
0 (7)
|
0 (9)
|
9 (0)
|
0 (8)
|
7 (3)
|
0 (8)
|
| |
Mean (SD)
|
–
|
–
| |
–
|
96.43 (20.26)
|
–
|
|
Month 24
|
n (missing)
|
0 (7)
|
3 (6)
|
4 (5)
|
0 (8)
|
4 (6)
|
5 (3)
|
| |
Mean (SD)
|
–
|
92.33 (22.01)
|
105.25 (4.99)
|
–
|
111.00 (12.19)
|
95.40 (22.79)
|
|
EOS
|
n (missing)
|
6 (1)
|
1 (8)
|
4 (5)
|
5 (3)
|
5 (5)
|
4 (4)
|
| |
Mean (SD)
|
116.33 (15.28)
|
90.00
|
93.75 (11.24)
|
96.80 (13.41)
|
103.40 (18.17)
|
89.75 (15.41)
|
- Treatment Groups from the Study Period. Both groups received sapropterin + Phe-restricted diet in the extension period. Age group at study entry used. Missing could be either have been due to missing analysis values or age out of range for assessment
- EOS end of study, IQ intelligence quotient, ITTE Intention-to-treat extension (population), SD standard deviation
