Protocol for a single patient therapy plan: A randomised, double-blind, placebo-controlled N-of-1 trial to assess the efficacy of cannabidiol in patients with intractable epilepsy
Katherine S Ong, John B Carlin, Michael Fahey, Jeremy L Freeman, Ingrid E Scheffer, Lynn Gillam, Monique Anderson, Md Hamidul Huque, Donna Legge, Nicole Dirnbauer, Brian Lilley, Simon Slota-Kan, Noel Cranswick
Journal of Paediatrics and Child Health | WILEY | Published : 2020
AIM: This paper describes the use of the single patient therapy plan (SPTP). The SPTP has been designed to assess the efficacy at an individual level of a commercially available cannabinoid product, cannabidiol, in reducing seizure frequency in paediatric patients with intractable epilepsy. METHODS: The SPTP is a randomised, double-blind, placebo-controlled N-of-1 trial designed to assess the efficacy of treatment in a neurology outpatient setting. The primary objective of the SPTP is to assess the efficacy of cannabidiol in reducing seizure frequency in each patient with intractable epilepsy, with change in seizure frequency being the primary outcome of interest. The analysis adopts a Bayes..View full abstract
The authors would like to thank: the clinic staff, human research and clinical ethics committees and pharmacy departments at Royal Children's Hospital, Monash Children's Hospital and Austin Hospital; Neuroscience Trials Australia for project management; Murdoch Children's Research Institute for data management and statistical services; members of the Independent Medical Advisory Committee on Medicinal Cannabis, chaired by James Angus; Department of Health and Human Services staff who assisted with conceptualisation, Margot Johnson, Matthew McCrone, Dania Swiatek; Tyson Ware Tasmanian Health Service for conceptual support; Tilray for development of matching placebo, and custom labelling of products purchased. This work was funded by the Department of Health and Human Services Victoria, Australia.