Sapropterin as a Treatment for Autistic Disorder

• Status: Completed
• Trial end date: 2011-10-01
• Target enrollment: 0
• Participant gender: All

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 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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The Children's Health Council
• Collaborator: BioMarin Pharmaceutical
• Treatments: Verapamil

Criteria

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