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

NCT ID: NCT04821856

Last Updated: 2025-09-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 132 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-05-11
• Study Completion Date: 2025-08-19

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.

Conditions

Intellectual Disability; Child Behavior Problem

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Cannabidiol 100mg/ml

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).

Group Type: EXPERIMENTAL

Cannabidiol Oil

Intervention Type: DRUG

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

Placebo

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).

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

MCT oil and flavoring solution, also manufactured by THC Pharma

Interventions

Cannabidiol Oil

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

Intervention Type: DRUG

Placebo

MCT oil and flavoring solution, also manufactured by THC Pharma

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Monash University

OTHER

Sponsor Role: collaborator

University of Sydney

OTHER

Sponsor Role: collaborator

Deakin University

OTHER

Sponsor Role: collaborator

Murdoch Childrens Research Institute

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Daryl Efron

Role: STUDY_DIRECTOR

Murdoch Childrens Research Institute

Locations

The Children's Hospital at Westmead

Westmead, New South Wales, Australia

Monash Children's Hospital

Clayton, Victoria, Australia

Royal Children's Hospital / Murdoch Children's Research Institute

Parkville, Victoria, Australia

Countries

Australia

References

Efron D, Taylor K, Payne JM, Freeman JL, Cranswick N, Mulraney M, Prakash C, Lee KJ, Williams K. Does cannabidiol reduce severe behavioural problems in children with intellectual disability? Study protocol for a pilot single-site phase I/II randomised placebo controlled trial. BMJ Open. 2020 Mar 8;10(3):e034362. doi: 10.1136/bmjopen-2019-034362.

Reference Type: BACKGROUND

PMID: 32152170 (View on PubMed)

Efron D, Freeman JL, Cranswick N, Payne JM, Mulraney M, Prakash C, Lee KJ, Taylor K, Williams K. A pilot randomised placebo-controlled trial of cannabidiol to reduce severe behavioural problems in children and adolescents with intellectual disability. Br J Clin Pharmacol. 2021 Feb;87(2):436-446. doi: 10.1111/bcp.14399. Epub 2020 Jul 1.

Reference Type: BACKGROUND

PMID: 32478863 (View on PubMed)

Other Identifiers

RCH HREC 70053

Identifier Type: -

Identifier Source: org_study_id