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

Devinsky, O. et al.


What You Need to Know:

In patients with the Dravet syndrome, treatment with cannabidiol was effective in reducing convulsive-seizure frequency but increased the risk of adverse events compared to placebo.

Trial Design:

  • Randomized, double-blind, placebo-controlled trial evaluated the efficacy of cannabidiol oral solution in patients with the Dravet syndrome and drug-resistant seizures
  • 120 children and young adults randomized to receive cannabidiol 20mg/kg of body weight daily or placebo, in addition to receiving standard antiepileptic therapy
  • Primary endpoint: convulsive-seizure frequency change over the 14-week treatment period

Key Outcomes:

  • Larger decrease in the median frequency of convulsive seizures per month observed in patients receiving cannabidiol vs placebo (12.4 to 5.9 vs 14.9 to 14.1 seizures per month, respectively)
  • Adjusted median difference between groups: -22.8%; 95% CI: -41.1, -5.4; P=0.01
  • 43% of cannabidiol patients had a 50% decrease in convulsive-seizure frequency vs 27% of placebo patients (OR 2.00; 95% CI: 0.93, 4.30; P=0.08)
  • 62% of cannabidiol patients improved by 1 category on the Caregiver Global Impression of Change Scale vs 34% of placebo patients (P=0.02)
  • No significant reduction seen in frequency of nonconvulsive seizures
  • Reduction in the frequency of seizures of all types was seen in the cannabidiol group (P=0.03)
  • 5% of cannabidiol patients became seizure-free vs 0% with placebo (P=0.08)
  • Increased risk of withdrawal from the study as well as adverse events in the cannabidiol group (diarrhea, vomiting, fatigue, pyrexia, somnolence, abnormal liver-function test results)