At a glance
ClinicalIndex Comparison RecordN/ACompleted· 10 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.
Biomarkers of Central Nervous System Tetrahydrobiopterin Concentration and Response to Tetrahydrobiopterin Supplementation in Children With Idiopathic Cognitive Developmental Disorders
The University of Texas Health Science Center, Houston·interventional·Posted Jun 10, 2010·Updated Apr 1, 2014
In Brief
A clinical study evaluating sapropterin dihydrochloride for Autism Spectrum Disorder. Completed, enrolled 10 participants across 1 site.
Detailed Summary
The purpose of this study is to develop biomarkers for central nervous system BH4 concentration in children with autism so they can be easily screened to determine if they may benefit from BH4 supplementation. Kuvan is a synthetic form of BH4. BH4 is an enzyme cofactor that is essential for several critical metabolic functions.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsAutism Spectrum Disorder
CountriesUnited States
CollaboratorsBioMarin Pharmaceutical
Timeline
N/ACompletedFinished
20102011201220132014201520162017201820192020202120222023202420252026
First PostedJun 2010
Enrollment StartJul 2010
Primary CompletionSep 2011
TodayJul 2026
First PostedJun 10, 2010
Enrollment StartJul 1, 2010
Primary CompletionSep 1, 2011
TodayJul 2, 2026
Enrollment to primary: 1.2 yearsPosted 16.1 years ago
Interventions
sapropterin dihydrochloridedrug
Patients will be instructed to take 20 mg/kg/day of Kuvan® orally dissolved in 4 - 8oz. of water or apple juice with breakfast.