Pediatric Clinical Trials
Sapropterin as a Treatment for Autistic Disorder
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 currently not accepting patients for this trial.
Intervention(s):
- drug: sapropterin
- drug: Placebo
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
Ages Eligible for Study
3 Years - 6 Years
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Lynne Huffman, MD
(650) 725-8314
Not Recruiting