Children with autism spectrum disorders (ASD) may have severe behavior problems, including aggression, self-injurious behaviors, severe and persistent stereotypic behaviors, and extreme hyperactivity, which limit their ability to function socially and academically and are often disruptive to family life as well. Cannabidiol (CBD), a compound originally derived from the cannabis plant but without the psychoactive effects found in cannabis, has been shown to be safe and effective in the treatment of children with severe epilepsy. Two non-controlled studies using CBD from local sources have demonstrated improvements in behavior in children with autism. Parents of autistic children have been using CBD products in an unregulated fashion with unknown dosing with anecdotal reports of improved behavior. This study will use EPIDIOLEX (EPX), a purified CBD oral solution that was FDA approved in June 2018 to treat severe forms of pediatric epilepsy. Study subjects will be 30 boys between 7 and 14 years of age with autism who have severe behavior problems. Every child will undergo baseline clinical evaluation, neuropsychological, behavioral, cognitive, and language testing, will have a test of brain wave activity (EEG) and a brain MRI scan. parents will complete questionnaires on various aspects of their child's behavior. Fifteen children will receive CBD for 8 weeks and 15 will receive a placebo that looks and tastes similar to the CBD (Period 1). All of the baseline tests and questionnaires will then be repeated. After a 4 week washout period, behavioral and cognitive tests and questionnaires will be repeated and then the treatments will be reversed (Period 2). At the end of 8 weeks, all of the baseline tests and questionnaires will be repeated. Study personnel and parents will be blinded to the treatment status of each child. Statistical analyses will be used to determine whether there are significant differences between baseline testing and results after placebo or CBD treatment. Types and severities of adverse events will be tracked to provide information about the safety and tolerability of CBD in this population. If CBD is found to be safe and effective in treating the behavioral problems associated with autism, this would be a major new tool in the treatment of those children that could lead to improved functioning and quality of life for the affected individuals and their families.
There are very few treatments that are effective in reducing severe behavioral problems associated with autism. These behaviors include aggressive and self-harm behaviors, frequent repetitive behaviors and severe hyperactivity. This study is being conducted to determine whether cannabidiol can reduce any or all of these problem behaviors.