Introduction Autism spectrum disorder (ASD) is a neurodevelopmental disorder commonly associated with behavioral challenges. There are few evidence based pharmacological interventions available for the treatment of behavioral symptoms associated with ASD. Cannabidiol (CBD), the non-psychoactive component of cannabis, has potential neuroprotective, antiepileptic, anxiolytic, and antipsychotic effects and may be useful in treating the behavioral symptoms of ASD.

Methods We describe the research methods of a 27-week double-blind placebo-controlled cross-over trial of cannabidiol for the treatment of irritability and aggression associated with ASD, utilizing the irritability subscale of the Aberrant Behavior Checklist-2nd edition (ABC-2) as the primary outcome measure. Adverse effects and safety monitoring protocols are included. Several secondary and exploratory outcomes measures also include anxiety, communication, repetitive behaviors, attention, hyperactivity, autism family experience, and telehealth functional behavior assessment.

Conclusion There is a significant need for clinical research exploring alternative medications for the treatment of behavioral symptoms of ASD. Cannabidiol (CBD) is being studied for the management of irritability, aggression, and other problem behaviors associated with ASD.

The authors have declared no competing interest.

NCT04520685

This study was funded by the Colorado Department of Public Health and Environment (CDPHE) with the investigational product (Epidiolex) provided by Jazz Pharmaceuticals. CDPHE and Jazz Pharmaceuticals are not involved in the study design, collection, analysis, and interpretation of data, writing of the report, or in the decision to submit the article for publication. Research infrastructure support at Childrens Hospital Colorado and University of Colorado School of Medicine were supported by NIH/NCATS Colorado CTSA Grant Number UM1 TR004399. Contents are the authors sole responsibility and do not necessarily represent official NIH views.

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The details of the IRB/oversight body that provided approval or exemption for the research described are given below:

Colorado Multiple Institution Review Board (COMIRB) gave ethical approval for this work. COMIRB 19-2168.

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(elise.sannarchildrenscolorado.org), (joan.winterchildrenscolorado.org), (ronda.frankechildrenscolorado.org), (emily.wernerchildrenscolorado.org), (rebecca.rochowiakchildrenscolorado.org), (patrick.romanichildrenscolorado.org), (owen.s.millercuanschutz.edu), (jacci.bainbridgecuanschutz.edu), (Obehi.enabulelechildrenscolorado.org), (talia.thompsonchildrenscolorado.org), (crystal.natvigcuanschutz.edu), (susan.mikulichcuanschutz.edu), (nicole.tartagliachildrenscolorado.org)

All data produced in the present study are available upon reasonable request to the authors following peer-reviewed publication