Sapropterin as a Treatment for Autistic Disorder

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.

Timeline

Start

2009-03

Primary completion

2011-08

Completion

2011-10

Outcome

Missed primary endpoint

paper RESULTS: Overall, no differences were found on global improvement as measured with the CGI-I or CGI-S. PMID 23782126 ↗

Publications

• Results Klaiman C, Huffman L, Masaki L, Elliott GR. Tetrahydrobiopterin as a treatment for autism spectrum disorders: a double-blind, placebo-controlled trial. J Child Adolesc Psychopharmacol. 2013 Jun;23(5):320-8. doi: 10.1089/cap.2012.0127.

Drugs

Indications