NCT01376908

Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

A Phase IIIb, Multicentre, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan®) in Phenylketonuria (PKU) Patients <4 Years Old.

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	Kuvan®	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.
OTHER	Phenylalanine (Phe)-restricted diet	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.

Timeline

• Start date: 2011-06-01
• Primary completion: 2014-07-01
• Completion: 2017-02-17
• First posted: 2011-06-20
• Last updated: 2017-09-15
• Results posted: 2016-05-12

Locations

22 sites across 9 countries: Austria, Belgium, Czechia, Germany, Italy, Netherlands, Slovakia, Turkey (Türkiye), United Kingdom