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Sapropterin as a Treatment for Autistic Disorder

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Trial Status Completed

Trial Identifier

NCT00850070

Condition

Autistic Disorder

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

3 Years to 6 Years

Healthy Volunteers

No

Drug/Treatment:

sapropterin, Placebo

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

46

Study Started:

2009-03

Study Updated:

2018-05-01

Trial Locations

  • The Children's Health Council

    Palo Alto, California, United States

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 pals

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