NCT00850070

Sapropterin as a Treatment for Autistic Disorder

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: The Children's Health Council · Academic / Other
• Sex: All
• Age: 3 Years – 6 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Autistic Disorder

Interventions

Type	Name	Description
DRUG	sapropterin	Patients will receive sapropterin 20 mg per kilogram per day for 16 weeks
DRUG	Placebo	Patients will receive a placebo identical in form and dosage to the active drug daily for 16 weeks.

Timeline

• Start date: 2009-03-01
• Primary completion: 2011-08-01
• Completion: 2011-10-01
• First posted: 2009-02-24
• Last updated: 2018-05-01
• Results posted: 2014-02-28

Locations

1 site across 1 country: United States