UK-based GW Pharmaceuticals has started a Phase II/III clinical trial of Epidiolex (cannabidiol or CBD) to treat Dravet syndrome, a rare and catastrophic treatment-resistant form of childhood epilepsy.
The Phase II/III trial is a two-part randomised double-blind, placebo-controlled parallel group safety, tolerability, pharmacokinetic and efficacy trial designed to evaluate single and multiple doses of Epidiolex in children with Dravet syndrome who are being treated with other anti-epileptic drugs.
Part one of the trial includes the pharmacokinetic and dose-finding elements in a total of 30 patients over a three-week treatment period, while the second part is a placebo-controlled safety and efficacy evaluation of Epidiolex over a three-month treatment period in a total of 80 patients.
The company said that all patients who complete the Phase II/III trial will be eligible to receive Epidiolex under a long-term open label extension trial.
NYU School of Medicine professor of Neurology, Neurosurgery, and Psychiatry and principal Investigator of the trial Orrin Devinsky said: "The results of our open-label trial of Epidiolex in children with Dravet syndrome have been very encouraging and we are excited to begin this important placebo-controlled clinical trial."
GW chief executive officer Justin Gover said: "Epidiolex is the first plant-based CBD medicine to be studied in a FDA-authorised, placebo-controlled trial and we look forward to working with leading paediatric epilepsy centres across the US to advance this clinical program as rapidly as possible."
An additional Phase III trial in Dravet syndrome is expected to be started in the first quarter of 2015 in parallel with part two of the first Phase II/III trial.
In the first quarter of 2015, the company intends to begin two Phase III clinical trials in Lennox-Gastaut syndrome.