Table 14Study PKU-016: Hamilton Depression Rating Scale Total Score in Phe Responders

PKU-016
SAP + Diet
(n = 61)		Placebo + Diet
(n = 57)
Change From Baseline to Week 13
Baseline
LS mean (SE)a
95% CI of LS mean		8.7 (1.1)
(6.5 to 10.9)		8.2 (1.0)
(6.1 to 10.3)
LS mean difference from placebo (SE)a
95% CI of LS mean difference		0.5 (1.1)
(–1.7 to 2.7)
Week 13
LS mean (SE)b
95% CI of LS mean		6.3 (0.9)
(4.5 to 8.0)		5.6 (0.8)
(4.0 to 7.3)
LS mean difference from placebo (SE)b
95% CI of LS mean difference		0.6 (0.9)
(–1.2 to 2.4)
Week 13 change from baseline
LS mean (SE)c
95% CI of LS mean; P valued		–2.1 (0.7)
(–3.6 to –0.6); < 0.001		–2.5 (0.7)
(–3.9 to –1.1); < 0.001
LS mean difference from placebo (SE)c
95% CI of LS mean difference; P valuee		0.4 (0.8)
(–1.1 to 1.9); 0.588
Change From Week 13 to Week 26
Week 13
LS mean (SE)a
95% CI of LS mean		6.3 (0.9)
(4.5 to 8.0)		5.6 (0.8)
(4.0 to 7.3)
LS mean difference from placebo (SE)a
95% CI of LS mean difference		0.6 (0.9)
(–1.2 to 2.4)
Week 26
LS mean (SE)b
95% CI of LS mean		6.4 (1.0)
(4.5 to 8.4)		5.5 (0.9)
(3.7 to 7.4)
LS mean difference from placebo (SE)b
95% CI of LS mean difference		0.9 (1.0)
(–1.1 to 2.9)
Week 26 change from week 13
LS mean (SE)c
95% CI of LS mean; P valued		0.5 (0.8)
(–1.1 to 2.0); 0.873		0.1 (0.7)
(–1.3 to 1.5); 0.734
LS mean difference from placebo (SE)c
95% CI of LS mean difference; P valuee		0.4 (0.8)
(–1.2 to 1.9); 0.636

CI = confidence interval; HAM-D = Hamilton Depression Rating Scale; LS = least squares; SAP = sapropterin; SE = standard error. Note: Decline in mean score in the Hamilton Depression rating scale represents an improvement in symptoms.

a

Based on ANCOVA of baseline HAM-D response with model terms treatment, age group, ADHD symptoms, and ADHD medication.

b

Based on ANCOVA of week 13/week 26 HAM-D response with model terms treatment, age group, ADHD symptoms, and ADHD medication.

c

ANCOVA of change from baseline/week 13 with terms treatment, baseline HAM-D response, age group, ADHD symptoms, and ADHD medication.

d

P value determined by t test testing mean change from baseline/week 13 at week 13/week 26 equals 0.

e

P value determined by t test versus placebo from ANCOVA model.

Source: Clinical Source Report PKU-016.10

From: Appendix 4, Detailed Outcome Data

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