Sapropterin as a Treatment for Autistic Disorder
| Status: | Completed |
|---|---|
| Conditions: | Neurology, Psychiatric |
| Therapuetic Areas: | Neurology, Psychiatry / Psychology |
| Healthy: | No |
| Age Range: | 3 - 6 |
| Updated: | 5/3/2018 |
| Start Date: | March 2009 |
| End Date: | October 2011 |
Sapropterin as a Treatment for Autistic Disorder: A Phase II Randomized, Double-Blind, Placebo-Controlled Trial
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.
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.
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.
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.
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
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