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

Murdoch Childrens Research Institute

Status and phase: Completed; Phase 2

Conditions: Child Behavior Problem; Intellectual Disability

Treatments: Drug: Cannabidiol Oil; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT04821856

RCH HREC 70053

Details and patient eligibility

About

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.

Enrollment: 132 patients

Sex: All

Ages: 6 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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132 participants in 2 patient groups, including a placebo group

Cannabidiol 100mg/ml

Experimental group

Description:

The starting dose of cannabidiol (CBD) will be 5 mg/kg/day and will be administered orally twice daily in doses of 2.5 mg/kg (up titration phase from day 1 to 7). After one week, the dose of CBD will be increased to 10 mg/kg/day in two daily doses of 5mg/kg (8-week maintenance phase from day 8 to 63). On completion of the maintenance phase the dose of CBD will be decreased to 5mg/kg/day for one week (day 64 to 70), after which the CBD administration will cease. A ceiling dose of 1000mg/day will be administered to all participants weighing 100kg or greater. These participants will receive a dose of 500mg/day during up- and down-titration. Doses will be rounded to the nearest 10mg (0.1mL).

Treatment:

Drug: Cannabidiol Oil

Placebo

Placebo Comparator group

Description:

The control group will receive placebo medium-chain triglyceride (MCT) oil which is indistinguishable from the active medication in appearance, smell and taste. Dose will be matched for volume to the cannabidiol arm, and administered twice daily for 10 weeks (including up- and down-titration).

Treatment:

Drug: Placebo