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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
  1. Daryl Efron1,2,3,
  2. Kaitlyn Taylor1,
  3. Jonathan M Payne3,4,
  4. Jeremy L Freeman2,4,
  5. Noel Cranswick2,
  6. Melissa Mulraney1,3,
  7. Chidambaram Prakash2,
  8. Katherine J Lee5,
  9. Katrina Williams1,3,6,7
  1. 1Health Services, Murdoch Childrens Research Institute, Parkville, Victoria, Australia
  2. 2The Royal Children's Hospital Melbourne, Parkville, Victoria, Australia
  3. 3Department of Paediatrics, Faculty of Medicine, Dentistry and Health Science, The University of Melbourne, Melbourne, Victoria, Australia
  4. 4Neuroscience, Murdoch Childrens Research Institute, Parkville, Victoria, Australia
  5. 5Clinical Epidemiology & Biostatistics, Murdoch Childrens Research Institute, Parkville, Victoria, Australia
  6. 6Department of Paediatrics, Monash University, Clayton, Victoria, Australia
  7. 7Monash Children's Hospital, Clayton, Victoria, Australia
  1. Correspondence to Dr Daryl Efron; daryl.efron{at}rch.org.au

Abstract

Introduction Severe behavioural problems (SBPs) are a common contributor to morbidity and reduced quality of life in children with intellectual disability (ID). Current medication treatment for SBP is associated with a high risk of side effects. Innovative and safe interventions are urgently needed. Anecdotal reports and preliminary research suggest that medicinal cannabis may be effective in managing SBP in children with developmental disabilities. In particular, cannabidiol (CBD) may be a plausible and safe alternative to current medications. Families who are in urgent need of solutions are seeking cannabis for their ID children with SBP. However there is no evidence from randomised controlled trials to support the use of CBD for SBP. This pilot study aims to investigate the feasibility of conducting a randomised placebo-controlled trial of CBD to improve SBP in children with ID.

Methods and analysis This is a single-site, double-blind, parallel-group, randomised, placebo-controlled pilot study of 10 participants comparing 98% CBD oil with placebo in reducing SBP in children aged 8–16 years with ID. Eligible participants will be randomised 1:1 to receive either CBD 20 mg/kg/day or placebo for 8 weeks. Data will be collected regarding the feasibility and acceptability of all study components, including recruitment, drop-out rate, study visit attendance, protocol adherence and the time burden of parent questionnaires. Safety outcomes and adverse events will be recorded. All data will be reported using descriptive statistics. These data will inform the design of a full scale randomised controlled trial to evaluate the efficacy of CBD in this patient group.

Ethics and dissemination This protocol has received ethics approval from the Royal Children’s Hospital ethics committee (Human Research Ethics Committee no. 38236). Results will be disseminated through peer-reviewed journals, professional networks, conferences and social media.

Trial registration number ACTRN12618001852246

  • cannabidiol
  • severe behavioural problems
  • children
  • intellectual disability
http://creativecommons.org/licenses/by-nc/4.0/

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Footnotes

  • Contributors All authors (DE, KT, JMP, JLF, NC, MM, CP, KJL and KW) made substantial contributions to the design of this study and the writing of the protocol. All authors (DE, KT, JMP, JLF, NC, MM, CP, KJL and KW) made substantial contributions to drafting the work and revising it critically for intellectual content. All authors (DE, KT, JMP, JLF, NC, MM, CP, KJL and KW) approved the final version submitted. All authors (DE, KT, JMP, JLF, NC, MM, CP, KJL and KW) agreed to be accountable for the accuracy or integrity of the work

  • Funding This work was supported by an internal grant scheme available to employees of MCRI. This research received no specific grant from any external funding agency in the public, commercial or not-for-profit sectors.

  • Disclaimer Investigational product was supplied in kind from Tilray, who had no role in the conception or design of the study and will have no role in data collection, management, analysis, or interpretation, preparation, review, or approval of the manuscript; nor in thedecision to submit the manuscript for publication.

  • Competing interests None declared.

  • Patient consent for publication Not required.

  • Ethics approval This project has ethics approval from the Human Research Ethics Committee of the Royal Children’s Hospital, Melbourne (38236)

  • Provenance and peer review Not commissioned; externally peer reviewed.

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