Placebo-Controlled Trial Announced for Cannabidiol Candidate for Dravet Syndrome
Dravet syndrome, also known as severe myoclonic epilepsy of infancy, is a rare genetic epileptic encephalopathy that begins during the first year of life. The first seizure is often associated with a fever and may be a tonic clonic seizure or involve clonic movements on one side of the body. During the second year of life, other seizure types tend to manifest (eg, absence seizures, atonic seizures, partial seizures, nonconvulsive status epilepticus) and development often begins to decline. In addition, these patients have a higher risk of sudden unexplained death. Because the seizures are often refractory to treatment with antiepileptic agents, cannabidiol (CBD) has been explored as a management option based on animal studies showing anticonvulsant efficacy in multiple animal models and high rates of perceived benefits noted in anecdotal reports involving children. However, there are no data from placebo-controlled trials to prove or disprove the efficacy and safety of CBD in Dravet syndrome.
In November 2013, the US Food and Drug Administration (FDA) granted cannabidiol orphan drug status for the treatment of children with Dravet syndrome and gave approval for cannabidiol studies to begin in this patient population. GW Pharmaceuticals announced one such trial in October 2014 for Epidiolex, their cannabidiol candidate.
GW Pharmaceuticals’ phase 2/3 trial will be a two-part, randomized, double-blinded, placebo-controlled, parallel-group safety, tolerability, pharmacokinetic, and efficacy trial of single and multiple doses of Epidiolex to treat Dravet syndrome in children receiving other antiepileptic drugs. The first part of the trial comprises the pharmacokinetic and dose-finding elements of the trial in a total of 30 patients over a 3-week treatment period, whereas the second part is a placebo-controlled safety and efficacy evaluation of Epidiolex over a 3-month treatment period in a total of 80 patients. In addition, all patients who complete the study will be eligible to receive Epidiolex under a long-term open-label extension study. GW anticipates an additional phase 3 trial in Dravet syndrome in the first quarter of 2015 in parallel with part two of the first phase 2/3 trial.
“Epidiolex is the first plant-based CBD medicine to be studied in a FDA-authorized, placebo-controlled trial and we look forward to working with leading pediatric epilepsy centers across the U.S. to advance this clinical program as rapidly as possible,” said Justin Gover, chief executive officer, GW Pharmaceuticals, in a company press release.
Healthcare providers interested in learning more about this trial are encouraged to email email@example.com.