Conditions: Phenylketonuria
Keywords: Phenylketonuria (PKU) Hyperphenylalaninemia (HPA) Kuvan® Sapropterin dihydrochloride Kuvan responder Tetrahydrobiopterin (BH4)
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 59
Healthy volunteers: no

Brief summary:
The primary objective of the study is to evaluate the proportion of responders (that is, greater than or equal to \[\>=\] 30 percent reduction from Baseline in blood phenylalanine \[Phe\] level) to treatment with Kuvan® (sapropterin dihydrochloride) 20 milligram per kilogram per day (mg/kg/day) for 28 days.

Detailed description:

Arms:
arm 1: None

Interventions: intervention 1: Kuvan®
Intervention descriptions: intervention 1: Kuvan® (sapropterin dihydrochloride) oral solution 20 milligram per kilogram (mg/kg) will be given once daily for 28 +/- 1 days.

Protocol document: