Date: 2016-04-11
Type of information: Initiation of the trial
phase: 3
Announcement: initiation of the trial
Company: GW Pharmaceuticals (UK)
Product: Epidiolex® (cannabidiol (2-[(1R,6R)-3-Methyl-6-(1-methylethenyl)-2-cyclohexen-1-yl]-5-pentyl-1,3-benzenediol)
Action
mechanism: cannabinol derivative/endocannabinoid modulator.
Disease: tuberous sclerosis complex
Therapeutic area: Cancer - Oncology - Rare diseases
Country: USA
Trial
details: (NCT02544763 )
Latest
news: * On April 11, 2016, GW Pharmaceuticals announced that it has commenced a Phase 3 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 program, which includes four Phase 3 pivotal trials in Dravet syndrome and Lennox-Gastaut syndrome, both rare and catastrophic forms of childhood-onset epilepsy.
At the 69th Annual Meeting of the American Epilepsy Society in December 2015, safety and efficacy data on 10 patients diagnosed with TSC from the physician-led open-label Epidiolex® expanded access program were presented by Massachusetts General Hospital for Children (Geffrey et al 2015) on Epidiolex® treatment of refractory epilepsy in these patients. The percent of patients who reported a 50% or greater reduction in seizures were 50%, 50%, 40%, 60% and 66% at 2, 3, 6, 9, and 12 months of treatment with Epidiolex®, respectively. Side effects were seen in five patients (50%) and most were resolved with anti-epileptic drug or CBD dose adjustment.
This single Phase 3 dose-ranging trial is a 16-week comparison of Epidiolex versus placebo in a total of approximately 200 patients, aged one to 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.
