Conditions: Phenylketonuria
Keywords: Phenylketonuria (PKU) Kuvan® (Sapropterin) Phe-restricted diet Paediatric <4 years old responders to BH4 Phe tolerance
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 56
Healthy volunteers: no

Brief summary:
This is a Phase 3b, multicenter, open-label, randomized, controlled study to evaluate efficacy, safety and population pharmacokinetics of sapropterin dihydrochloride (Kuvan®) in less than 4 year-old infants and children with phenylketonuria (PKU).

Detailed description:

Arms:
arm 1: Subjects will be treated with Kuvan® tablets once daily along with Phe-restricted diet therapy. arm 2: Subjects will follow a Phe-restricted diet alone.

Interventions: intervention 1: Kuvan® intervention 2: Phenylalanine (Phe)-restricted diet
Intervention descriptions: intervention 1: Kuvan® (sapropterin dihydrochloride) tablets will be administered orally at the dose of 10 mg/kg/day and will be escalated to 20 mg/kg/day if after 4 weeks a subject's Phe tolerance is not increased by at least 20% versus baseline. intervention 2: Phe intake will be adjusted every 2 weeks, based on the mean Phe levels of the previous 2 weeks using pre-defined Phe adjustment criteria.

Protocol document: