Sapropterin as a Treatment for Autistic Disorder: A Phase II Randomized, Double-Blind, Placebo-Controlled Trial
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.
Stanford is not currently accepting new patients for this trial. You may want to check clinicaltrials.gov to see if other locations are recruiting.
- drug : Placebo
- drug : sapropterin
Phase: Phase 2
Ages Eligible For Study:
- Parents sign informed consent - Child meets criteria for autistic disorder (based on score on the Autism Diagnostic Interview?Revised (ADI-R) and the Autism Diagnostic Observation Schedule (ADOS), given by a certified administrator, research reliable) - Child has a Developmental Quotient (DQ) ? 50 (Vineland Adaptive Scales, Interview Edition) - Parents agree to delay initiation of other treatments during double-blind trial