A Prospective, Observational Cohort Study to Assess Long-Term Safety in Patients Prescribed Epidyolex® with a Focus on Drug-induced Liver Injury (DILI)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  EPIDYOLEX
• Study drug International non-proprietary name (INN) or common name:
  CANNABIDIOL
• Anatomical Therapeutic Chemical (ATC) code:
  (N03AX24) cannabidiol

  cannabidiol
• Medical condition to be studied:
  Lennox-Gastaut syndrome

  Severe myoclonic epilepsy of infancy

  Tuberous sclerosis complex

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  230

Study design details

• Main study objective:
  To assess DILI and its risk factors of Epidyolex in patients prescribed Epidyolex in standard clinical practice.
• Outcomes:
  To assess frequency, severity, and outcome of DILI and its risk factors as determined and assessed by standard serum liver tests (Serum ALT, AST, ALP, and total bilirubin levels with fractionation if routinely done) • Incidence of Hy’s Law cases, Number and nature of SAEs and related AEs specific to suicidality (class effect), aggression, euphoria, and impact on cognitive development. • Number and nature of other SAEs • Number and nature of AEs • Changes in weight from baseline • Change in CGIC from baseline.
• Data analysis plan:
  The statistical analyses will be descriptive in nature. Each variable will be summarized descriptively by age group (paediatric versus adults), overall, by prior treatment (whether naïve to treatment or not), and by follow-up study year. Unless stated otherwise, descriptive statistics for continuous variables will include the number of non-missing values (n), mean, SD, median, minimum, maximum, interquartile range, and 95% CI where appropriate. Categorical variables will be summarized by the number and percentage of patients falling in each category. The primary analyses will include all patients from EU member countries who received at least 1 dose of Epidyolex. Supplementary analysis will be performed including EU member countries and the UK.