Cannabidiol as add-on Therapy for Children With Refractory Epilepsy (CBD-uN1que), a High-quality Individualized Approach: a Series of N-of-1 Trials

Korábbi kezelés

• 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
• History of recreational or medicinal cannabis use, 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

Más betegség

• Any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (cannabis extract and refined peanut oil, generally safe for patients with peanut allergy)
• Ongoing evaluation for epilepsy surgery
• Unstable medical condition, other than refractory epilepsy, that is of significant influence on participation in the trial; significance to be determined with the treating physician/pediatric neurologist

Laborértékek

• History or current signs 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.
• Cardiac disease including: Structural heart disease with hemodynamic significance, heart failure, ischemic heart disease, Brugada syndrome, long-QT syndrome, cardiac arrythmia

Egyéb

• Pregnancy, breastfeeding, or intention to become pregnant throughout the trial or within 3 months of completing treatment
• Glaucoma
• Expected inability to undergo blood sampling due to anxiety or resistance
• Use of clobazam dosage of > 0,5 mg/kg/day in the last month

• 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 in one week) of epileptic origin, during the 4-week baseline period while receiving care as usual.
• All medication doses or other interventions for epilepsy must have been stable for one month prior to screening and the participants/parents and treating physicians are willing to maintain the current treatment regimen throughout the trial.
• Informed consent/ of legal representative/ parents.
• Presence of a consistently available patient caregiver for proxy-reports.