Stanford drug trial seeks participants with autism spectrum disorder

Researchers at the Stanford University School of Medicine are seeking participants for a study examining the effectiveness of vasopressin, a neuropeptide, in treating children with autism spectrum disorder.

Difficulty with social interactions is characteristic of people with autism, who often have problems interpreting facial expressions or maintaining eye contact while talking with someone. There are currently no effective medicines available to treat social problems in people with autism.

Neuropeptides, such as vasopressin, are molecules used by neurons in the brain to communicate with one another. Vasopressin has been shown to facilitate social interactions in animals.

Animal studies have also shown that when the proper functioning of vasopressin is interfered with, animals develop a variety of social deficits, including impaired memory for peers and a reduced interest in social interaction.

Researchers found that when vasopressin was administered to mice with a genetically induced form of autism, their social behavior became more normal.

Vasopressin is already approved by the U.S. Food and Drug Administration for use in humans, and has proved to be a successful treatment for some common pediatric conditions, including bedwetting. It also has been shown to improve social cognition and memory in people who do not have autism.

The researchers will test the effects of vasopressin on social impairments in 50 high-functioning boys and girls with autism, ages 6 to 12. The study will last four weeks.

Participants will be randomly chosen to receive either vasopressin or a placebo. At the end of the study, those who received the placebo will have the option of participating in a further four weeks of treatment during which they will be given vasopressin.

Stanford is the only site for the study. Participants do not need to live locally but will need to come to Stanford’s Department of Psychiatry and Behavioral Sciences for at least three visits. They will be provided with a $20 payment for each completed study visit and will be given general results from autism diagnostic assessments and neuropsychological tests.

Karen Parker, PhD, assistant professor of psychiatry and behavioral sciences, and Antonio Hardan, MD, professor of psychiatry and behavioral sciences and a medical director of the outpatient program in child and adolescent psychiatry, are principal investigators of the study.

This investigation is funded in part by the National Institute of Mental Health and by the Child Health Research Institute at Stanford University.

More information about the trial is available at http://med.stanford.edu/clinicaltrials/trials/NCT01962870. To apply to participate in the trial, contact Robin Libove at (650) 736-1235 or rlibove@stanford.edu