Translational Medicine
Open Access

Abstract

A Pilot Open-Label Trial of Use of the Glycine Transporter I Inhibitor, Sarcosine, in High-Functioning Children with Autistic Disorder

Pinchen Yang, Hsien-Yuan Lane, Cheng-Fang Yen and Chen-Lin Chang

This open-label trial examined the efficacy and safety of a glycine transporter I inhibitor, sarcosine, in the 24-week treatment of high-functioning children with autistic disorder. Four children (three boys, one girl, 9-11 years of age; average intelligence quotient 80.5) completed the 24 weeks of study. Sarcosine administration was at 30 mg/kg/day in the form of a capsule in two divided doses. The outcome measures were. Autism Diagnostic Observation Schedule (Module 3), parent and teacher-reported Adaptive Behavior Assessment System-II, parenting stress index, Conners’ Continuous Performance Test, Wisconsin Card Sorting Test, child behavior checklist and Swanson, Nolan and Pelham IV hyperactivity attention scales. Safety assessments included monthly recorded vital signs, body weight, body height and adverse events. Statistical analysis found no significant treatment effect on all the outcome measures using the Wilcoxon Signed Rank test and generalized estimating equations analysis. However, an activation effect was reported by caregivers, and was corroborated by clinician’s observation. Details were reported as a case-series in the text. We concluded that sarcosine was well tolerated. Though the data are too preliminary to draw any definite conclusions about efficacy, they do suggest this therapy to be safe, and worthy of a double-blind placebo-controlled study with a focus on a certain subgroup of children with autism spectrum disorders.