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) ScaleClinical 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
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