CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 46 enrolled
Drug / intervention
sapropterin +1 moredrug
Likely dose
sapropterin 20 mgfrom 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/NCT00850070
NCT00850070Phase 2Completed
CompareCT.gov

Sapropterin as a Treatment for Autistic Disorder: A Phase II Randomized, Double-Blind, Placebo-Controlled Trial

The Children's Health Council·interventional·Posted Feb 24, 2009·Updated May 1, 2018

In Brief

A Phase 2 clinical trial evaluating sapropterin and Placebo for Autistic Disorder. Completed, enrolled 46 participants across 1 site.

Detailed Summary

This study is intended to provide a definitive test of the hypothesis that elevating sapropterin (tetrahydrobiopterin, a cofactor for several key brain enzymes)concentrations in the CNS will result in measurable improvements in core symptoms of autism in young individuals, under age 6 years. The study will entail a double-blind, placebo-controlled 16-week intervention.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsAutistic Disorder
CountriesUnited States
CollaboratorsBioMarin Pharmaceutical

Timeline

Phase 2CompletedFinished
200920102011201220132014201520162017201820192020202120222023202420252026
First PostedFeb 24, 2009
Enrollment StartMar 1, 2009
Primary CompletionAug 1, 2011
Study CompletionOct 1, 2011
TodayJul 2, 2026
Enrollment to primary: 2.4 yearsPosted 17.4 years ago

Interventions

sapropterindrug

Patients will receive sapropterin 20 mg per kilogram per day for 16 weeks

Placebodrug

Patients will receive a placebo identical in form and dosage to the active drug daily for 16 weeks.