Cannabidiol for ASD Open Trial

Summary

This is a 6-week open trial to identify the optimal dosing of cannabidiol (CBD) in youth with autism spectrum disorder (ASD) and to identify primary and secondary outcomes for future controlled studies. This study evaluates change in symptoms commonly associated with ASD, as evidence suggests that CBD may be effective in addressing difficulties such as irritability and anxiety, while maintaining a benign adverse effect (AE) profile in children and adolescents. 30 male and female participants with ASD between the ages of 7 and 17 years old are being recruited. Participants have fluent speech and an estimated IQ greater than or equal to 80. Study intervention is 98% pure CBD. The CBD is Greenwich Biosciences, Inc.'s 100mg/mL oral solution, brand name EPIDIOLEX. First, a Bayesian optimal interval (BOIN) design was used, such that participants were assigned to cohorts of size 3 receiving doses of 3, 6, or 9 mg/kg/day, depending on the treatment response of participants in prior cohorts. The BOIN design ended after the fifth cohort of participants, and the two lower doses, 3 and 6 mg/kg/day, were eliminated. The highest dose, 9 mg/kg/day, was not tested. Therefore, in subsequent cohorts, we will be examining 9 mg/kg/day exclusively in up to 15 additional participants with co-occurring ASD and attention-deficit/hyperactivity disorder (ADHD) diagnoses, as this clinical profile appears to most closely resemble youth classified as responders within the BOIN design.

Timeline

Start

2019-03-12

Primary completion

2023-05-04

Completion

2023-05-04

Publications

• Lawson J, Robinson L, Conlon GR, Shalev RA, Cervantes PE, Yoncheva Y, Hirsch GS, Troxel AB, Friedman D, Devinsky O, Castellanos FX. A Phase-2 Open-Label Trial of Cannabidiol to Treat Core and Associated Symptoms of Autism in Children and Adolescents Without Intellectual Disability. J Child Adolesc Psychopharmacol. 2026 Aug;36(6):350-358. doi: 10.1177/10445463261452514. Epub 2026 May 28.

Drugs

Indications