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NCT Number: NCT04821856

Evaluation of the Effectiveness of Cannabidiol in Treating Severe Behavioural Problems in Children and Adolescents With Intellectual Disability

This is a multi-site, double-blind, parallel group, randomized, placebo-controlled study of 140 participants comparing oral purified cannabidiol isolate (CBD) with placebo in reducing Severe Behavioral Problems (SBP) at 8 weeks in children aged 6 - 18 years with Intellectual Disability (ID). Eligible participants will be randomized 1:1 to receive either CBD or placebo.

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Key information

Conditions

Intellectual Disability Child Behavior Problem Mental Disorders Nervous System Diseases Neurobehavioral Manifestations Neurodevelopmental Disorders Neurologic Manifestations Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

6 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The Children's Hospital at Westmead, Westmead, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Males and females aged 6 - 18 years of age;
  • DSM-5 diagnosis of intellectual disability (ID):
  • Full scale IQ < 70 on standardized cognitive assessment. Testing results must be sighted by the investigators and performed within two years of enrollment. In the event that records of prior testing are unavailable or the assessment was more than 2 years prior, IQ will be estimated using the Wechsler Abbreviated Scale of Intelligence-II.
  • Deficit in adaptive function (basis for severity rating of ID in DSM-5) in at least one activity of life on the Vineland Adaptive Behavior Scales (derives scores in Communication, Daily Living Skills and Socialization domains, and a Global Adaptive score). If records of prior testing are unavailable or the assessment was more than 2 years prior, this will be completed by the parent or guardian.
  • SBP: Defined as scores of:
  • 18 or higher on the Aberrant Behavior Checklist-Irritability subscale (ABC-I), and
  • moderate or higher on the Clinical Global Impressions-Severity scale;
  • No changes in either medication or other interventions in the 4 weeks prior to randomization, and intention to remain on same dose for the duration of the study;
  • Written informed consent from parent or legal guardian;
  • Participant and family have the ability to comply with the protocol requirements, in the opinion of the investigator.

Exclusion criteria

  • Non-English speaking parents;
  • Psychosis;
  • Taking clobazam, mTOR inhibitors (e.g sirolimus, tacrolimus), anti-cancer agents, citalopram >20mg/day, escitalopram >10mg/day.;
  • Abnormal liver function tests: defined as ALT > twice ULN;
  • Abnormal renal function tests: defined as creatinine > ULN
  • Current use of medicinal cannabis, or use in the 4 weeks prior to screening;
  • Pregnant or intending to become pregnant during the study, or breastfeeding;
  • Known allergy to cannabidiol or cannabis products

Treatment and study plan

Cannabidiol Oil

Drug

Cannabidiol (CBD) isolate 100mg/ml in MCT oil oral solution, manufactured by THC Pharma

Placebo

Drug

MCT oil and flavoring solution, also manufactured by THC Pharma

Primary outcomes

  1. Mean difference between the cannabidiol 100mg/ml and placebo arms on the Aberrant Behavior Checklist-Irritability (ABC-I) subscale total score at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 15 item parent-rated questionnaire, used to measure Severe Behavioral Problems in children

Secondary outcomes

  1. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total scores of the four remaining subscales of the Aberrant Behavior Checklist (ABC) at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    43 parent-rated items assessing: Social withdrawal, Stereotypic behavior, Hyperactivity/Noncompliance and Inappropriate Speech

  2. Comparison between the cannabidiol 100mg/ml and placebo arms of clinician ratings on the Clinical Global Impressions-Improvement at day 64. Data will be presented as the proportion of participants in each arm with a rating suggesting improvement

    Time frame: At day 64 (end of maintenance treatment period)

    This is a single item clinician-rated summary measure of improvement

  3. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Parent Rated Anxiety Scale-ASD at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 25 item parent-rated measure of anxiety in youth with ASD

  4. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Child & Adolescent Scale of Participation at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 20 item parent-rated questionnaire that includes sub-scales for participation in home, school, and community activities

  5. Mean difference between the cannabidiol 100mg/ml and placebo arms on the weighted total score of the Child Health Utility 9D (CHU-9D) at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 9 item parent-rated, preference-weighted measure used to calculate quality adjusted life years for children

  6. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Sleep Disturbance Scale for Children at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 26 item parent-rated questionnaire assessing sleep difficulties in children

  7. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Assessment of Quality of Life 4D (AQOL-4D) at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 12 item self-report health-related instrument used to calculate quality adjusted life years for parents

  8. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Beach Center Family Quality of Life at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 25 item parent report measure that includes subscales assessing family interaction, parenting, emotional and material wellbeing, and disability-related support.

  9. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Depression Anxiety Stress Scale-21 at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 21 item self-rated assessment of parent mental health, including symptoms of depression, anxiety and stress

  10. Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Autism Parenting Stress Index at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This is a 13 item self-rated measure of 3 categories of stress drivers in parents: core social disability, difficult behavior, physical issues

  11. The frequency of adverse events as reported on the modified version of the Liverpool Adverse Event Profile (LAEP) at day 64 will be summarised across the cannabidiol 100mg/ml and placebo arms

    Time frame: At day 64 (end of maintenance treatment period)

    Completed by the parent or guardian, the LAEP was designed to capture known side-effects of anti-epileptic medication. The modified version includes additional items to ascertain other known side-effects of CBD. This measure includes 34 items.

Other outcomes

  1. Comparing the health sector cost-effectiveness of cannabidiol vs placebo in terms of Aberrant Behavior Checklist - Irritability (ABC-I) scores at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This involves calculation of an incremental cost effectiveness ratio (ICER): health sector costs (CBD-placebo) divided by incremental ABC-I scores. Health sector costs will be calculated through: (1) Estimation of intervention costs from the study's financial records; (2) A brief Resource Use Questionnaire, completed by parents at day 64, which will capture medication and health sector resource use associated with their child's behaviour across the 8 week intervention period.

  2. Comparing the societal cost-effectiveness of cannabidiol vs placebo in terms of Aberrant Behavior Checklist - Irritability (ABC-I) scores at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This involves calculation of an incremental cost effectiveness ratio (ICER): societal costs (CBD-placebo) divided by incremental ABC-I scores. Societal costs will be calculated through a brief Resource Use Questionnaire, completed by parents at day 64, which will capture societal resource use and lost productivity associated with their child's behaviour across the 8 week intervention period.

  3. Comparing the health sector cost-effectiveness of cannabidiol vs placebo in terms of Quality Adjusted Life Years calculated from the CHU-9D at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This involves calculation of an incremental cost effectiveness ratio (ICER): health sector costs (CBD-placebo) divided by incremental Quality adjusted life years (QALYs) from CHU-9D . Health sector costs will be calculated through: (1) Estimation of intervention costs from the study's financial records; (2) A brief Resource Use Questionnaire, completed by parents at day 64, which will capture medication and health sector resource use associated with their child's behaviour across the 8 week intervention period.

  4. Comparing the societal cost-effectiveness of cannabidiol vs placebo in terms of Quality Adjusted Life Years calculated from the CHU-9D at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This involves calculation of an incremental cost effectiveness ratio (ICER): societal costs (CBD-placebo) divided by incremental Quality adjusted life years (QALYs) from CHU-9D . Societal costs will be calculated through a brief Resource Use Questionnaire, completed by parents at day 64, which will capture societal resource use and lost productivity associated with their child's behaviour across the 8 week intervention period.

  5. Comparing the health sector cost-effectiveness of cannabidiol vs placebo in terms of Quality Adjusted Life Years calculated from the AQOL-4D at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This involves calculation of an incremental cost effectiveness ratio (ICER): health sector costs (CBD-placebo) divided by incremental Quality adjusted life years (QALYs) from AQOL-4D . Health sector costs will be calculated through: (1) Estimation of intervention costs from the study's financial records; (2) A brief Resource Use Questionnaire, completed by parents at day 64, which will capture medication and health sector resource use associated with their child's behaviour across the 8 week intervention period.

  6. Comparing the societal cost-effectiveness of cannabidiol vs placebo in terms of Quality Adjusted Life Years calculated from the AQOL-4D at day 64

    Time frame: At day 64 (end of maintenance treatment period)

    This involves calculation of an incremental cost effectiveness ratio (ICER): societal costs (CBD-placebo) divided by incremental Quality adjusted life years (QALYs) from AQOL-4D . Societal costs will be calculated through a brief Resource Use Questionnaire, completed by parents at day 64, which will capture societal resource use and lost productivity associated with their child's behaviour across the 8 week intervention period.

Sponsors and collaborators

Lead sponsor

Murdoch Childrens Research Institute

Other

Collaborators

  • Deakin University
  • Monash University
  • University of Sydney

Registry information

Official study title

A Randomized Placebo-controlled Trial of Cannabidiol to Treat Severe Behavioral Problems in Children and Adolescents With Intellectual Disability

Important dates

Study start
2021
Primary completion
2025
Study completion
2025
First posted
Mar 30, 2021
Registry last updated
Sep 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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