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Sapropterin as a Treatment for Autistic Disorder

Information source: The Children's Health Council
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Autistic Disorder

Intervention: sapropterin (Drug); Placebo (Drug)

Phase: Phase 2

Status: Completed

Sponsored by: The Children's Health Council

Official(s) and/or principal investigator(s):
Glen R Elliott, Ph.D., M.D., Principal Investigator, Affiliation: The Children's Health Council

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.

Clinical Details

Official title: Sapropterin as a Treatment for Autistic Disorder: A Phase II Randomized, Double-Blind, Placebo-Controlled Trial

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Primary outcome:

Clinical Global Impression -- Improvement (CGI-I) Scale

Clinical Global Impression -- Severity (CGI-S) Scale

Secondary outcome:

Preschool Language Scale-Fourth Edition (PLS-4). Assesses Expressive and Receptive Language Skills in Ages Birth Through 6 Years, 11 Months.

Vineland Adaptive Behavior Scale-II.

Children's Yale Brown Obsessive Compulsive Scale (C-YBOCS)

Connor's Preschool ADHD Questionnaire

Adverse Events Scale

Aberrant Behavior Checklist (ABC) - Inappropriate Speech

Social Responsiveness Scale (SRS)

Parent Global Assessment (PGA) Scale

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 had somewhat questionable methodologies, a major one being that the doses of sapropterin used were roughly one tenth that thought to be needed to provide physiologically meaningful increases of sapropterin in the central nervous system (CNS). This study will look at the impact of a sustained exposure to this higher dose in well-diagnosed young children with autism.

Eligibility

Minimum age: 3 Years. Maximum age: 6 Years. Gender(s): Both.

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

Locations and Contacts

The Children's Health Council, Palo Alto, California 94304, United States
Additional Information

Starting date: March 2009
Last updated: January 27, 2014

Page last updated: August 23, 2015

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