Cannabidiol in refractory childhood epilepsy, a personalized high-quality approach

Prior treatment

• History of recreational or medicinal cannabis, or cannabinoid-based medications, within three months prior to screening and the patient is unwilling to abstain for the duration of the study.
• Planned intervention under general anesthesia interfering with the baseline period; or any planned major surgery within the duration of the trial.
• Implementation of vagal nerve stimulation within 3 months prior to screening and unwillingness to delay inclusion until stable settings of vagal nerve stimulation are reached.
• Starting a ketogenic diet within 3 months prior to screening and unwillingness to delay inclusion until a stable ketogenic diet is reached.

Other illness

• Any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP).
• Structural heart disease.
• Ongoing evaluation for epilepsy surgery.
• Unstable medical condition(s), other than refractory epilepsy, that are of significant influence on participation in the trial; significance to be determined with the treating physician/pediatric neurologist.

Lab values

• History or current symptoms of significantly impaired liver function, such as bilirubin level ≥ 2 x upper limit of normal; or presence of liver damage as indicated by levels of alanine aminotransferase and/or aspartate aminotransferase ≥ 3 x upper limit of normal.

Other

• Expected inability to undergo blood sampling due to anxiety or resistance.
• Pregnancy, breastfeeding, or intention to become pregnant throughout the trial or within 3 months of completing treatment.
• Glaucoma.

• Minimum age of 1 year old and maximum age of 18 years old.
• Confirmed diagnosis of refractory epilepsy according to ILAE criteria.
• At least 4 countable seizures (not all on one day) during the 4-week baseline period while receiving at least 1 ASM.
• All medications or interventions for epilepsy must have been stable dosed for one month prior to screening and the participant is willing to maintain the current ASM regimen throughout the trial.
• Informed consent/assent of legal representative/ parents.
• Presence of a consistently available patient caregiver for proxy-reports.