Clinical Trials Directory

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 to see if other locations are recruiting.



  • drug : Placebo
  • drug : sapropterin

Phase: Phase 2


Ages Eligible For Study:

3 Years - 6 Years

Inclusion Criteria

- 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

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Contact information

Primary Contact:

Lynne Huffman, MD (650) 725-8314

Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305

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