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Study register · detail Pilot-RCT (doppelblind, placebokontrolliert) · Safety & Adverse Effects · 2021

A pilot randomised placebo-controlled trial of cannabidiol to reduce severe behavioural problems in children and adolescents with intellectual disability.

Efron et al. ·British Journal of Clinical Pharmacology Impact 2.1

Mixed GRADE Moderate 37 citations

Samplen = 8 Pat.

Duration8 weeks

ControlPlacebo, oral, same regimen

EndpointFeasibility/acceptability of…

Blindingdoppelblind

DesignPilot-RCT (doppelblind, placebokontrolliert)

Cannabinoidcbd

Max. dose1000.0 mg

Routeoral

Key finding

The study protocol was fully feasible and acceptable; a preliminary efficacy signal favoring cannabidiol was observed, without significant adverse events.

Summary

Pilot RCT (n=8 children with intellectual disability and severe behavioral problems, 8–16 years); CBD 20 mg/kg/day (max. 500 mg 2×/day) vs. placebo over 8 weeks; 0 serious adverse events, 0 study dropouts; medication adherence 100%; blood tests 92%; parental acceptance high; efficacy signal favoring CBD.

PopulationChildren and adolescents (8–16 years) with intellectual disability and severe behavioral problems, n=8

InterventionCannabidiol 98% in oil (Tilray, Canada), oral, titrated up to 20 mg/kg/day in two doses (max. 500 mg twice daily), 8 weeks

ControlPlacebo (oil), oral, same regimen

OutcomeFeasibility/acceptability evidence: study completion 100%, medication adherence 100%, blood tests 92%, questionnaire completion 88%; efficacy signal favoring active treatment (no statistics reported)

Confidence in the evidence

Moderate

The third of four GRADE levels, the effect estimate is probably reliable.

Downgraded for

Imprecision

Quality profile

Sample size ★★★★★

Blinding Double-blind

Effect size Mixed

Citations / year ★★★★★

Authors

Efron D, Freeman JL, Cranswick N, Payne JM, Mulraney M, Prakash C, Lee KJ, Taylor K, Williams K.

DOI 10.1111/bcp.14399↗

Design: Pilot-RCT (doppelblind, placebokontrolliert)

Abstract

<h4>Aims</h4>Severe behavioural problems (SBP) are a major contributor to morbidity in children with intellectual disability (ID). Medications used to treat SBP in ID are associated with a high risk of side effects. Cannabidiol has potential therapeutic effects in SBP. This pilot study aimed to investigate the feasibility of conducting a randomised placebo-controlled trial of cannabidiol to reduce SBP in children with ID.<h4>Methods</h4>This is a double-blind, placebo-controlled, two-armed, parallel-design, randomised controlled trial of cannabidiol in children aged 8-16 years with ID and SBP. Participants were randomised 1:1 to receive either 98% cannabidiol in oil (Tilray, Canada) or placebo orally for 8 weeks. The dose was up-titrated over 9 days to 20 mg/kg/day in two divided doses, with a maximum dose of 500 mg twice/day. The feasibility and acceptability of all study components were assessed.<h4>Results</h4>Eight children were randomised, and all completed the full study protocol. There were no serious adverse events or drop-outs. Protocol adherence for key study components was excellent: study visits 100%, medication adherence 100%, blood tests 92% and questionnaire completion 88%. Parents reported a high degree of acceptability with the study design. All parents reported they would recommend the study to other families with children with similar problems. There was an efficacy signal in favour of active drug.<h4>Conclusions</h4>The findings suggest that the study protocol is feasible and acceptable to patients with ID and SBP and their families.</h4>

The impediment to action advances action. — Marcus Aurelius