At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 95 enrolled
Drug / intervention
sapropterin dihydrochloridedrug
Likely dose
sapropterin dihydrochloride 20 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.
A Phase 3b Open-Label Study to Evaluate the Effect of Kuvan® on Neurocognitive Function, Maintenance of Blood Phenylalanine Concentrations, Safety, and Population Pharmacokinetics in Young Children With Phenylketonuria
In Brief
A Phase 3 clinical trial evaluating sapropterin dihydrochloride for Phenylketonuria. Completed, enrolled 95 participants across 19 sites in 2 countries.
Detailed Summary
This multicenter, open label study is designed to evaluate the safety of Kuvan® and its effect on neurocognitive function, blood Phe concentration, and growth in children with PKU who are 0-6 years old.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPhenylketonuria
CountriesCanada, United States
Collaborators--
Timeline
Phase 3CompletedFinished
200920102011201220132014201520162017201820192020202120222023202420252026
Enrollment StartFeb 2009
First PostedFeb 2009
Primary CompletionSep 2018
TodayJul 2026
First PostedFeb 6, 2009
Enrollment StartFeb 1, 2009
Primary CompletionSep 14, 2018
TodayJul 2, 2026
Enrollment to primary: 9.6 yearsPosted 17.4 years ago
Interventions
sapropterin dihydrochloridedrug
A dose of 20 mg/kg will be administered dissolved in water or apple juice, based on subject's age and ability, and taken orally once daily with food.