Conditions: Phenylketonuria
Keywords: PKU
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 111
Healthy volunteers: no

Brief summary:
The objective of this study is to evaluate the safety of long-term treatment with Phenoptin in subjects with phenylketonuria (PKU) who participated in Phase 3 clinical studies with Phenoptin.

Detailed description:

Arms:

Interventions: intervention 1: sapropterin dihydrochloride
Intervention descriptions: intervention 1: 5-20mg/kg/day orally, dose may be adjusted up or down as needed at the discretion of the investigator in increments of 5mg/kg/day.

Protocol document: