July 4, 2016
GW Pharmaceuticals released results of a
randomized, double-blind, placebo-controlled
phase III trial of Epidiolex (cannabidiol or CBD)
for Lennox-Gastaut syndrome (LGS), a rare
and severe form of childhood-onset epilepsy.
The trial randomized 171 patients into two
arms, where Epidiolex 20mg/kg/day (n=86)
or placebo (n=85) was added to current antiepileptic
drug (AEDs) treatment. The average
age of trial participants was 15 years (34%
were 18 years or older). During the treatment
period, patients taking Epidiolex achieved a
median reduction in monthly drop seizures
of 44% compared with a reduction of 22% in
patients receiving placebo, and the difference
between treatments was statistically significant
(p=0.0135). Epidiolex has Orphan Drug
Designation from the FDA for the treatment
of LGS and Dravet syndrome. The company
anticipates advancing Epidiolex toward NDA
submission with the FDA in the first half of
2017. In addition to this first phase III trial of
Epidiolex in LGS, GW is conducting a second
phase III dose-ranging trial of Epidiolex for the
treatment of LGS.
March 28, 2016
GW Pharmaceuticals reported results of the first pivotal phase III study of Epidiolex (cannabidiol or CBD) for the treatment of Dravet syndrome. The study randomized 120 patients into two arms, Epidiolex 20mg/kg/day (n=61) and placebo (n=59). Epidiolex or placebo was added to current anti-epileptic drug (AED) treatment regimens. On average, patients were taking approximately three AEDs, having previously tried and failed an average of more than four other AEDs. The average age of trial participants was 10 years and 30% of patients were less than 6 years of age. The median baseline convulsive seizure frequency per month was 13. Patients taking Epidiolex achieved a median reduction in monthly convulsive seizures of 39% compared with a reduction on placebo of 13%, which was highly statistically significant (p=0.01). The difference between Epidiolex and placebo emerged during the first month of treatment and was sustained during the entire treatment period. Epidiolex was generally well-tolerated in the study. The most common adverse events (occurring in greater than 10% of Epidiolex-treated patients) were somnolence, diarrhea, decreased appetite, fatigue, pyrexia, vomiting, lethargy, upper respiratory tract infection and convulsion. Of those patients on Epidiolex that reported an adverse event, 84% reported it to be mild or moderate. GW is currently conducting a second phase III trial in Dravet syndrome, which is recruiting 150 patients. Epidiolex has both Orphan Drug designation and Fast Track designation from the FDA in the treatment of Dravet syndrome.
December 13, 2010
H. Lundbeck released positive results from a phase III trial of clobazam for the treatment of Lennox-Gastaut syndrome (LGS). This multicenter, randomized, double-blind, placebo-controlled, parallel-group study enrolled 238 subjects who were randomized to receive either placebo or low (0.25 mg/kg/day up to a maximum of 10 mg per day), medium (0.5 mg/kg/day; maximum daily dosage of 20 mg per day) or high (1.0 mg/kg/day; maximum daily dosage of 40 mg) doses clobazam for up to 23 weeks. Of the 238 randomized subjects, a total of 217 comprised the modified intent to treat (mITT) population. The primary endpoint was the reduction in the average weekly rate of drop seizures from the 4-week baseline period compared to the 12-week maintenance period. Data showed that the high and medium dosages of clobazam met this endpoint with statistical significance over placebo (p≡0.01). Subjects in the high-dosage clobazam group achieved a mean decrease in average weekly rate of drop seizures of 68.3% (p<0.0001 vs. placebo) while those in the medium-dosage arm had an average decrease of 49.4% (p≡0.0015 vs. placebo). Secondary endpoints, including responder rates and the effect of clobazam in decreasing total seizures, were also significantly improved in the high- and medium-dose clobazam arms versus placebo.