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Clinical trial to test possible new therapy for autism deficits

Apr. 23, 2020, 10:00 AM


by Kelsey Herbers

Vanderbilt University Medical Center is participating in a multisite clinical trial to test a potential new medication for treating core deficits of autism spectrum disorders in children ages 2-4.

The oral medication works to block a receptor for the hormone vasopressin in the brain that has been linked to the control of socialization, stress, anxiety and aggression.

The drug has been effective in children over the age of 5 and adolescents with autism spectrum disorders (ASD).

While there are treatments available to help relieve behavioral symptoms of autism in younger populations, there are currently no approved medications that target the social and communication deficits at the core of ASD. These deficits usually emerge early in development and can be observed by age 3.

Evidence links early identification and treatment of ASD to better outcomes, meaning extending pharmacological treatments to those under the age of 4 could have significant long-term impacts.

“Because there currently are no approved medications to treat the core symptoms of ASD, our team is dedicated to conducting research supporting novel therapies and medications for this population. We believe this work by Roche Pharmaceuticals is very promising and are happy to continue our portfolio of research with this particular compound,” said Allyson Witters Cundiff, MD, assistant professor of Clinical Psychiatry and Behavioral Sciences at VUMC and primary investigator for Vanderbilt’s portion of the study.

According to the Centers for Disease Control and Prevention (CDC), one in 54 U.S. children have a diagnosis of autism by the age of 8. While increasing evidence shows children with ASD can be accurately identified during their second year of life, early identification is lacking, with the average age of diagnosis in both Tennessee and across the country remaining well above 4 years of age.

For those who are identified early, interventions targeting behavior and development can help improve daily functioning, but treatments for core symptoms fall short.

The medication study aims to assess the pharmacokinetics, safety, tolerability and preliminary efficacy of the drug, which will be given to participants once a day for six weeks.

Participants will also receive in-person assessments of their general health, social communication skills and social interaction skills three times during the same period to identify any changes.

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