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Sapropterin as a Treatment for Autistic Disorder
Not Recruiting
Trial ID: NCT00850070
Purpose
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.
Official Title
Sapropterin as a Treatment for Autistic Disorder: A Phase II Randomized, Double-Blind, Placebo-Controlled Trial
Eligibility
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
Exclusion Criteria:
- Child has had seizures in past 6 months or a change in seizure medications in past 4
weeks.
- Child has > 18 points on subscale of (Autism Behavior Checklist) ABC-I
- Child is taking any psychoactive medication other than supplements, anticonvulsants,
or soporifics (melatonin, diphenhydramine)
- Child has had any change in standing medications in the past 4 weeks.
- Child has known genetic disorders
- Child has known severe neurological disorders, including cerebral palsy
Intervention(s):
drug: sapropterin
drug: Placebo
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Lynne Huffman, MD
(650) 725-8314