UIC to begin STX209 clinical trial for treatment of autism spectrum disorders

An experimental drug to treat social withdrawal in children and young adults with autism is being studied in a clinical trial at the University of Illinois at Chicago's Institute for Juvenile Research.

Children with autism -- or autism spectrum disorders -- often have difficulty communicating and interacting with others. Although behavioral and psychological interventions are often beneficial, currently there is no medication to address social communication difficulties, a core symptom of ASD.

A drug treatment is needed that would address symptoms that are "often disabling for patients and families," says Dr. Edwin Cook, professor of psychiatry and director of autism and genetics at UIC.

UIC is the only study site in Illinois and one of 25 sites nationwide. The study is sponsored by Seaside Therapeutics, Inc.

The clinical trial will enroll approximately 150 patients diagnosed with autism spectrum disorders between ages 5 and 21 to evaluate the efficacy, safety, and tolerability of STX209 (arbaclofen).

Participants in the 22-week study will be randomized to receive either the study drug, STX209, or a placebo. The clinical trial will include screening, treatment, withdrawal of medication, and a follow-up period. Subjects who complete the study may be eligible to enroll in a subsequent open-label study in which all subjects are treated with STX209.

STX209 has been studied in children with Fragile X syndrome, a genetic disorder that is the most common identified cause of autism. Previous research has found that from one-quarter to one-half of people with Fragile X have autism spectrum disorders, said Cook, principal investigator of the UIC study.

"This trial is exciting, because it represents the culmination of 20 years work in Fragile X research," said Cook, who describes the preliminary data leading to the STX209 study in autism as a "scientific and rational approach" to medication development.

"We're not expecting this to cure Fragile X or autism, but it's a very important step in the development of new treatments," Cook said.

"Finding genes is great, but it's all about treatment."