At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 206 enrolled
Drug / intervention
Sapropterin dihydrochloride +1 moredrug
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 Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria
In Brief
A Phase 3 clinical trial evaluating Sapropterin dihydrochloride and Placebo for Phenylketonuria. Completed, enrolled 206 participants across 36 sites in 2 countries.
Detailed Summary
This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPhenylketonuria
CountriesCanada, United States
Collaborators--
Timeline
Phase 3CompletedFinished
20102011201220132014201520162017201820192020202120222023202420252026
First PostedMay 2010
Enrollment StartAug 2010
Primary CompletionMar 2013
TodayJul 2026
First PostedMay 3, 2010
Enrollment StartAug 1, 2010
Primary CompletionMar 1, 2013
TodayJul 2, 2026
Enrollment to primary: 2.6 yearsPosted 16.2 years ago
Interventions
Sapropterin dihydrochloridedrug
A dose of 20 mg/kg/day will be administered. Route of administration is oral (intact).
Placebodrug
Placebo (tablet without active ingredient) is dosed once/day for the first 13 weeks of the study.