GW Pharmaceuticals begins phase 3 Epidiolex study in tuberous sclerosis complex
GW Pharmaceuticals has announced the start of a Phase 3 clinical trial of Epidiolex® (a cannabidiol-based medication) as an adjunctive therapy for the treatment of epileptic seizures in the rare genetic disorder tuberous sclerosis complex (TSC).
Epilepsy occurs in around 80-90 per cent of TSC patients and is a significant cause of poor health and mortality.
GW is also running phase 3 trials within its Epidiolex clinical development programme for Dravet syndrome and Lennox-Gastaut syndrome, both rare forms of childhood-onset epilepsy.
Drug to address unmet need among patients with few treatment options
In March 2016, GW announced positive results from the first of these trials in Dravet syndrome. Justin Gover, GW's Chief Executive Officer said: " With the commencement of this trial, GW's Epidiolex development program now encompasses three rare epilepsy indications with pivotal trials fully underway. GW is committed to establishing Epidiolex as a new therapy to address the significant unmet need among patients who have few treatment options."
"In patients with TSC, epilepsy is common, presents early and can become more difficult to treat over time. The results of our open-label study of Epidiolex in children with TSC have been very encouraging and we are excited to begin this important placebo-controlled clinical trial," stated Elizabeth Thiele, MD, PhD, Director, Pediatric Epilepsy Program at Massachusetts General Hospital, Director, Herscot Center for Tuberous Sclerosis Complex and Professor of Neurology, Harvard Medical School and Principal Investigator of the trial.
Epidiolex showed greater than 40 per cent reduction in seizures
In the previous trials for Epidiolex as a treatment for TSC, results from between two and 12 months of treatment showed a greater than 40 per cent reduction in seizures in the ten study patients.
The newly announced phase 3 trial is a dose-ranging, 16 week comparison of Epidiolex versus placebo in a sample of around 200 patients between one and 65 years of age. The trial will assess the drug's safety and efficacy as an adjunctive anti-epileptic treatment. Researchers will be measuring the percentage change in baseline seizure frequency during the treatment period.
Following this study, subjects may be eligible to receive Epidiolex as part of a further, long term open-label study.