Epidiolex (cannabidiol or CBD) commences Phase III study to treat tuberous sclerosis complex- GW Pharma

GW Pharmaceuticals has commenced a Phase III clinical trial of Epidiolex (cannabidiol or CBD) as an adjunctive therapy for the treatment of seizures associated with tuberous sclerosis complex (TSC), a rare genetic disorder, the most common symptom of which is epilepsy. Epilepsy occurs in around 80–90% of TSC patients and is a significant cause of morbidity and mortality. TSC is the third indication that GW is targeting within its Epidiolex clinical development programme, which includes four Phase III pivotal trials in Dravet syndrome and Lennox-Gastaut syndrome, both rare and catastrophic forms of childhood-onset epilepsy.

In March 2016, GW announced positive results from the first of these pivotal trials in Dravet syndrome. This single Phase III dose-ranging trial is a 16-week comparison of Epidiolex versus placebo in a total of approximately 200 patients, aged 1–65, to assess its safety and efficacy as an adjunctive anti-epileptic treatment. The primary measure of this trial is the percentage change from baseline in seizure frequency during the treatment period. Primary endpoint seizures include focal motor seizures with or without impairment of consciousness or awareness, and generalized convulsive seizures. Several additional efficacy and safety secondary outcome measures will be analysed. Following completion of the study, patients may be eligible to receive Epidiolex through a long term open-label extension study.