The goal of this clinical trial is to learn if cannabidiol ("CBD-oil") works to treat severe epilepsy in children. It will also learn about the safety of cannabidiol. The main questions it aims to answer are:
* Does cannabidiol lower the number of seizure in children with severe epilepsy? * Does cannabidiol effect other outcomes such as behaviour, sleep, communication, activities? * What medical problems do participants have when taking cannabidiol?
Researchers will compare cannabidiol to a placebo (a look-alike oil that contains no cannabidiol) to see if drug cannabidiol works to treat severe epilepsy. All participants will receive both the cannabidiol and the placebo during different periods. At the end of the trial, we will evaluate for each patient seperately, whether seizures were less or more frequent during cannabidiol periods or placebo periods, to see what works best for every patient seperately.
Participants will:
* Register their seizures in a digital application during 4 to 6 periods. During these periods, CBD oil of placebo will be provided * Take CBD oil during 2 to 3 periods. One period will last around 7 weeks. * Also take placebo oil during the other 2 to 3 periods * Have a blood test every 7 weeks to monitor for safety and possible adverse effects * Visit the clinic once every 15 weeks for checkups and tests * Fill in questionnaires about quality of life, functioning, sleep, behaviour, epilepsy severity every 7 weeks.
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.
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)
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.
Pregnancy, breastfeeding, or intention to become pregnant throughout the trial or within 3 months of completing treatment
Cardiac disease including: Structural heart disease with hemodynamic significance, heart failure, ischemic heart disease, Brugada syndrome, long-QT syndrome, cardiac arrythmia
UMC Utrecht
Lead sponsor
Stichting Epilepsie Instellingen Nederland
Collaborator
Epilepsiecentrum Kempenhaeghe
Collaborator
Erasmus Medical Center
Collaborator
Maastricht University Medical Center
Collaborator
ZonMw: The Netherlands Organisation for Health Research and Development
Collaborator