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.
Phase:
Phase 2
Details
Lead Sponsor:
New York University School of Medicine NYU Langone Health