Sapropterin as a Treatment for Autistic Disorder

Completed · Phase 2 · Has a placebo group

Conditions studied: Autistic Disorder

In brief

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.

Key facts

Study ID

NCT00850070

Run by

The Children's Health Council

People needed

46

Starts

2009-03-01

Expected to finish

2011-10-01

Last updated by the study team

2018-05-01

Who can join

Age: 3 and older, up to 6. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• The Children's Health Council — Palo Alto, California, United States

Full record on ClinicalTrials.gov

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