CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 56 enrolled
Drug / intervention
Kuvan® +1 moredrug
Likely dose
Kuvan® 10 mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01376908
NCT01376908Phase 3Completed
CompareCT.gov

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.

BioMarin Pharmaceutical·interventional·Posted Jun 20, 2011·Updated Sep 15, 2017

In Brief

A Phase 3 clinical trial evaluating Kuvan® and Phenylalanine (Phe)-restricted diet for Phenylketonuria. Completed, enrolled 56 participants across 22 sites in 9 countries.

Detailed 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).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPhenylketonuria
CountriesAustria, Belgium, Czechia, Germany, Italy, Netherlands, Slovakia, Turkey (Türkiye), United Kingdom
Collaborators--

Timeline

Phase 3CompletedFinished
2011201220132014201520162017201820192020202120222023202420252026
First PostedJun 20, 2011
Enrollment StartJun 1, 2011
Primary CompletionJul 1, 2014
Study CompletionFeb 17, 2017
TodayJul 2, 2026
Enrollment to primary: 3.1 yearsPosted 15.0 years ago

Interventions

Kuvan®drug

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.

Phenylalanine (Phe)-restricted dietother

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.