Status:
COMPLETED
Sapropterin as a Treatment for Autistic Disorder
Lead Sponsor:
The Children's Health Council
Collaborating Sponsors:
BioMarin Pharmaceutical
Conditions:
Autistic Disorder
Eligibility:
All Genders
3-6 years
Phase:
PHASE2
Brief 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...
Detailed Description
Over the past 20 years, several studies have suggested that sapropterin (tetrahydrobiopterin) might ameliorate core symptoms of autism at least in young (under age 6) subjects. However, those studies ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
March 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2011
Estimated Enrollment :
46 Patients enrolled
Trial Details
Trial ID
NCT00850070
Start Date
March 1 2009
End Date
October 1 2011
Last Update
May 1 2018
Active Locations (1)
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1
The Children's Health Council
Palo Alto, California, United States, 94304