Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet Syndrome

A multinational, randomized, double-blind, placebo-controlled trial of cannabidiol was conducted to treat drug-resistant epilepsy in the Dravet syndrome (a rare genetic form of epileptic encephalopathy primarily due to loss-of-function mutations in the SCN1A gene).This trial showed that cannabidiol reduced the frequency of convulsive seizures among children and young adults (2 to 18 years of age) over a 14-week period but was associated with adverse events including somnolence and elevation of liver-enzyme levels. Additional data are needed to determine the long-term efficacy and safety of cannabidiol for the Dravet syndrome.

(Funded by GW Pharmaceuticals; ClinicalTrials.gov number, NCT02091375.)

 

Trial of Cannabidiol for Drug-Resistant Seizures in the Dravet Syndrome

N Engl J Med 2017;376:2011-20.

Orrin Devinsky, M.D., J. Helen Cross, Ph.D., F.R.C.P.C.H., Linda Laux, M.D., Eric Marsh, M.D., Ian Miller, M.D.,Rima Nabbout, M.D., Ingrid E. Scheffer, M.B., B.S., Ph.D., Elizabeth A. Thiele, M.D., Ph.D.,and Stephen Wright, M.D., for the Cannabidiol in Dravet Syndrome Study Group.