Trial of Cannabidiol to Treat Severe Behavior Problems in Children With Autism

NCT04517799 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 181455
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autism; Autism Spectrum Disorder

Keywords

cannabidiol; CBD; autism; autism spectrum disorder

Outcome Measures

Primary Outcomes (3)

• Total Score on Repetitive Behavior Scale-Revised (RBS-R)

  This scale aims to capture the frequency and severity of the repetitive and restricted behaviors that are a common feature in individuals with autism spectrum disorders. The RBS-R has been validated and has been shown to have high internal consistency and inter-rater reliability when used in outpatient settings. There are 5 subscales: Stereotypic Behavior, Self-Injurious Behavior, Compulsive Behavior, Ritualistic/Sameness Behavior, and Restricted Interests. This scale will be administered to the parent or caregiver, who will report on the child's recent behaviors. Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

• Total Score on Child Behavior Checklist (CBCL)

  The CBCL is a widely used test that assesses problem behaviors in children by posing questions to the parent or caregiver. It poses questions about a wide range of potential negative behaviors including aggression, rule-breaking, and social problems. The school-aged scale, which will be used in this study, consists of 118 questions. The CBCL has 8 syndrome scales, including Aggressive Behavior, Anxiety/Depressed, Attention Problems, Rule-breaking, Somatic Complaints, Social Problems, Thought Problems, and Withdrawn/Depressed. Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

• Autism Diagnostic Observation Scale- 2 (ADOS-2)

  This a widely used scale that diagnoses autism spectrum disorders across individuals with a broad range of ages, developmental levels, and language abilities. It is a semi-structured assessment and consists of behavioral observation and coding of relevant behaviors. The ADOS-2 evaluates communication, social interaction, and imaginative play. The child is asked to complete various activities and then rated on their performance by the tester. Time of administration is 40-60 minutes. Range of scores is 1-10. Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

Secondary Outcomes (5)

• Total Score on Aberrant Behavior Checklist (ABC)

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

• Total Score on Social Responsiveness Scale

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

• Total Score on Peabody Picture Vocabulary test (PPVT-4)

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

• Total Score on Expressive One Word Picture Vocabulary test, Fourth Edition (EOWPVT-4)

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

• Total Score on Children's Sleep Habits Questionnaire (CSHQ)

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

Other Outcomes (1)

• Magnetic Resonance Spectroscopy

  Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)

Study Arms (2)

cannabidiol

EXPERIMENTAL

cannabidiol arm

Drug: Epidiolex

placebo

PLACEBO COMPARATOR

placebo arm

Drug: placebo oral solution

Interventions

Epidiolex DRUG

While in the intervention arm subjects will receive EPIDIOLEX, an oral purified CBD solution developed by GW Pharmaceuticals. The formulation is a 100 mg/mL solution, which allows for easy dose titration and administration even to young children. EPIDIOLEX is naturally-derived CBD, and has been found to have a purity of over 98%. The solution includes a sweetener to make it palatable. Dosing is dependent upon each subject's weight. The dosage will be titrated over time as follows: Week 1: 5 mg/kg/day, divided into 2 doses Week 2: 10 mg/kg/day, divided into 2 doses Weeks 3-8: 20 mg/kg/day, divided into 2 doses If a child's weight changes by greater than 2 kg during the study, the dose will be adjusted to reflect the weight change.

Also known as: cannabidiol

cannabidiol

placebo oral solution DRUG

GW has developed a placebo that is identical in color and taste, and will be administered in the same volume as the CBD, so that parents and investigators should not be able to detect differences between the 2 treatments. Dosing is dependent upon each subject's weight. The dosage will be titrated as follows: Week 1: 5 mg/kg/day, divided into 2 doses Week 2: 10 mg/kg/day, divided into 2 doses Weeks 3-8: 20 mg/kg/day, divided into 2 doses If a child's weight changes by greater than 2 kg during the study, the dose will be adjusted to reflect the weight change.

placebo

Eligibility Criteria

• Age: 7 Years - 14 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Boys ages 7-14 years
• Confirmed diagnosis of autism based on ADOS testing
• Autism severity assessed as severe with substantial behavioral problems
• Severity of symptoms will be based on a number of criteria:
• Aggressive and/or self-injurious (SIBs) behaviors occur almost daily (more than 6 times per week) in any situation (home, school, clinic, etc.).
• Frequent (daily), persistent (lasting at least 5-10 minutes and repeated through the day) stereotypies (repetitive behaviors such as hand flapping, running in circles, jumping repeatedly, waving fingers in front of eyes)
• Pervasive hyperactivity (child is so physically active that he cannot sit for meals or school work, is moving all the time, jumping off furniture, climbing onto furniture, etc.)
• One of more of the above activities is deemed to contribute significantly to child's inability to function by parental report and with clinician agreement based on history and/or direct observation

You may not qualify if:

• the presence of epilepsy
• a known genetic condition such as tuberous sclerosis
• other significant health issues such as cardiac disease, presence of known congenital brain malformation, or a history of central nervous system infection.
• children who are on anticonvulsant medications such as clobazam or valproic acid will also be excluded because of potential drug-drug interactions. At the time of screening, each child's medication list will be checked for drugs that are known to cause interactions with cannabidiol.
• children with an allergy to any components of the study drug
• children who are taking CBD from another source, unless parents are willing to stop the treatment for at least 4 weeks prior to entering the study. CBD and other cannabinoid blood levels will be performed at baseline and if CBD is detected in the blood, the child will be not be included in the study.
• children who might travel out of the area for a significant time during the study
• children who recently participated in another investigational drug trial may be excluded

Sponsors & Collaborators

• University of California, San Diego: lead
• Wholistic Research and Education Foundation: collaborator
• Center for Medicinal Cannabis Research: collaborator

Study Sites (1)

University of California San Diego

La Jolla, California, 92093, United States

Location

Related Publications (1)

• Trauner D, Umlauf A, Grelotti DJ, Fitzgerald R, Hannawi A, Marcotte TD, Knight C, Smith L, Paez G, Crowhurst J, Brown A, Suhandynata RT, Lund K, Menlyadiev M, Grant I. Cannabidiol (CBD) Treatment for Severe Problem Behaviors in Autistic Boys: A Randomized Clinical Trial. J Autism Dev Disord. 2025 May 24. doi: 10.1007/s10803-025-06884-y. Online ahead of print.

  PMID: 40410546 DERIVED

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Doris Trauner, MD

  University of California, San Diego

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 3, 2020
• First Posted: August 18, 2020
• Study Start: June 1, 2020
• Primary Completion: August 30, 2025
• Study Completion: December 31, 2025
• Last Updated: September 30, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)