Conditions: Phenylketonuria
Keywords: EMR 700733_510, Genetic disorder, Phenylketonuria, phenylalanine, Kuvan, sapropterin dihydrochloride
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 90
Healthy volunteers: no

Brief summary:
This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® (sapropterin dihydrochloride) in subjects aged 4 to 18 years who have phenylketonuria and with elevated blood phenylalanine level of greater than or equal to 450 micromole per liter.

Detailed description:

Arms:
arm 1: None

Interventions: intervention 1: Kuvan®
Intervention descriptions: intervention 1: Kuvan® (sapropterin dihydrochloride) will be administered orally at a dose of 20 milligram per kilogram per day (mg/kg/day) once daily for 8 days. If there is 30 percent (%) decrease in blood phenylalanine levels from baseline at the end of Day 8, then treatment will be continued at the same dose for further 6 weeks.

Protocol document: