Intellectual Disability, Child Behavior Problem
Conditions
Keywords
pediatric, intellectual disability, severe behavior problems
Brief summary
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.
Interventions
DRUGCannabidiol Oil
Cannabidiol (CBD) isolate 100mg/ml in MCT oil oral solution, manufactured by THC Pharma
DRUGPlacebo
MCT oil and flavoring solution, also manufactured by THC Pharma
Sponsors
Monash University
University of Sydney
Deakin University
Murdoch Childrens Research Institute
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Males and females aged 6 - 18 years of age; 2. DSM-5 diagnosis of intellectual disability (ID): 1. 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. 2. 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. 3. SBP: Defined as scores of: 1. 18 or higher on the Aberrant Behavior Checklist-Irritability subscale (ABC-I), and 2. moderate or higher on the Clinical Global Impressions-Severity scale; 4. 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; 5. Written informed consent from parent or legal guardian; 6. Participant and family have the ability to comply with the protocol requirements, in the opinion of the investigator.
Exclusion criteria
1. Non-English speaking parents; 2. Psychosis; 3. Taking clobazam, mTOR inhibitors (e.g sirolimus, tacrolimus), anti-cancer agents, citalopram \>20mg/day, escitalopram \>10mg/day.; 4. Abnormal liver function tests: defined as ALT \> twice ULN; 5. Abnormal renal function tests: defined as creatinine \> ULN 6. Current use of medicinal cannabis, or use in the 4 weeks prior to screening; 7. Pregnant or intending to become pregnant during the study, or breastfeeding; 8. Known allergy to cannabidiol or cannabis products
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean difference between the cannabidiol 100mg/ml and placebo arms on the Aberrant Behavior Checklist-Irritability (ABC-I) subscale total score at day 64 | 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
| Measure | Time frame | Description |
|---|---|---|
| Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Child & Adolescent Scale of Participation at day 64 | 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 |
| Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Sleep Disturbance Scale for Children at day 64 | At day 64 (end of maintenance treatment period) | This is a 26 item parent-rated questionnaire assessing sleep difficulties in children |
| 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 | 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 |
| 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 | 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. |
| Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Depression Anxiety Stress Scale-21 at day 64 | 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 |
| Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Autism Parenting Stress Index at day 64 | 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 |
| 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 | 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. |
| Mean difference between the cannabidiol 100mg/ml and placebo arms on the total score of the Parent Rated Anxiety Scale-ASD at day 64 | At day 64 (end of maintenance treatment period) | This is a 25 item parent-rated measure of anxiety in youth with ASD |
| 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 | At day 64 (end of maintenance treatment period) | 43 parent-rated items assessing: Social withdrawal, Stereotypic behavior, Hyperactivity/Noncompliance and Inappropriate Speech |
| 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 | At day 64 (end of maintenance treatment period) | This is a single item clinician-rated summary measure of improvement |
| 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 | 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 |
Other
| Measure | Time frame | Description |
|---|---|---|
| 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 | 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. |
| Comparing the societal cost-effectiveness of cannabidiol vs placebo in terms of Quality Adjusted Life Years calculated from the CHU-9D at day 64 | 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. |
| 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 | 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. |
| Comparing the societal cost-effectiveness of cannabidiol vs placebo in terms of Quality Adjusted Life Years calculated from the AQOL-4D at day 64 | 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. |
| Comparing the societal cost-effectiveness of cannabidiol vs placebo in terms of Aberrant Behavior Checklist - Irritability (ABC-I) scores at day 64 | 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. |
| Comparing the health sector cost-effectiveness of cannabidiol vs placebo in terms of Aberrant Behavior Checklist - Irritability (ABC-I) scores at day 64 | 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. |
Countries
Australia
Outcome results
None listed