Overview

Sapropterin as a Treatment for Autistic Disorder

Status:
Completed
Trial end date:
2011-10-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
The Children's Health Council
Collaborator:
BioMarin Pharmaceutical
Treatments:
Verapamil