Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome
Status:
Recruiting
Trial end date:
2021-06-01
Target enrollment:
Participant gender:
Summary
The clinical trial "A Prospective Single-Center Single-Arm Clinical Trial on Cognitive Effect
of Cannabidiol (CBD-OS®) on Dravet syndrome and Lennox-Gastaut Syndrome" is a single-group
phase III study done in single tertiary referral center in Seoul, Korea.
Chief investigator is Dr. Hoon-Chul Kang of Severance Hospital, Yonsei University College of
Medicine. Associate investigators are Dr. Heung Dong Kim, Joon Soo Lee, Se Hee Kim, Han Som
Choi, Ji Hoon Na, Dong Hwa Yang, and Hee Jung Kang, of Severance Hospital, Yonsei University
College of Medicine.
The aim of the study is to evaluate the effect of cannabidiol (CBD-OS®) on cognitive
functions in patients aged from 2 to 18 years old diagnosed with Dravet syndrome or
Lennox-Gastaut syndrome. The duration of study is planned as one year, after patient
recruitment of 6 months. The intervention period in each patient is 24 weeks, with 2 weeks of
medication titration, stabilization period of 10 weeks, and maintenance period of 12 weeks.
The recruitment goal of patient number is 104, considering the study power of 90 percent.
Primary outcomes are improvement of cognitive and development and improvement of seizure
outcome. Secondary outcomes are improvement in behavior and quality of life.
Safety monitoring criteria are adverse event profiles and physician's and caregiver's global
assessment.
Statistical analysis of outcomes is subject only to the patients who completed the 24-week
medication and 2 times of tests before and after treatment of cannabidiol. Evaluation of
seizure outcomes would include all patients who completed the 24-week medication and those
who dropped out of the study, either by follow-up loss or discontinuation of medication due
to incomplete seizure control or adverse effect of the medication.
To evaluate safety, the investigators would measure adverse events and dropout rates by
percentage. The investigators would analyze overall evaluation of the caregivers and
investigator. Serious adverse events would be noted after causality evaluation.