Table 6Exposure to Study Drug (Intention-to-Treat Population)

PKU-016
Randomized Treatment
(Week 0 to Week 13)		Open-Label Treatment
(Week 13 to Week 26)
SAP + Diet
▬		Placebo + Diet
▬		SAP + Diet
▬		Placebo to SAP + Diet
▬
Total duration of exposure (days)
Mean (SD)
Median (min, max)		▬▬
▬▬▬▬		▬▬▬
▬▬▬▬		▬▬
▬▬▬▬		▬▬▬
▬▬▬▬
Total duration of exposure, n (%)
< 46 days
46 to 91 days
> 91 days		▬▬
▬▬▬
▬▬▬		▬▬
▬▬▬
▬▬▬		▬▬
▬▬▬▬
▬▬▬		▬
▬▬▬
▬▬▬
Amount of drug taken (mg)
Mean (SD)
Median (min, max)		▬▬▬▬
▬▬		▬▬▬▬
▬▬		▬▬▬▬
▬▬		▬▬▬
▬▬
Average daily dose (mg/day)
Mean (SD)
Median (min, max)		▬▬▬▬▬
▬▬▬▬		▬▬▬▬▬
▬▬▬▬		▬▬▬▬▬
▬▬▬		▬▬▬▬▬
▬▬▬

SAP = sapropterin; SD = standard deviation.

Note: Treatment exposure was not analyzed in the Spark study, but at the end of the study period, 25 (92.6%) of patients were on SAP 10 mg/kg/day + diet and 2 (7.4%) of patients were on SAP 20 mg/kg/day + diet.

Source: Clinical Study Report PKU-0016.10

From: 4, Results

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Clinical Review Report: Sapropterin dihydrochloride (Kuvan) [Internet].
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