Cannabidiol as add-on Therapy for Children With Refractory Epilepsy (CBD-uN1que), a High-quality … (NCT07668856) | Clinical Trial Compass
RecruitingPhase 3
Cannabidiol as add-on Therapy for Children With Refractory Epilepsy (CBD-uN1que), a High-quality Individualized Approach: a Series of N-of-1 Trials
Netherlands50 participantsStarted 2025-02-06
Plain-language summary
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.
Who can participate
Age range
1 Year – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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.
Exclusion Criteria:
* 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
* Glaucoma
* Ongoing evaluation for epil…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Seizure frequency
Timeframe: Daily, from start of baseline after enrollment to the end of the last treatment cycle (each cycle - consisting of two periods with run in, taper and washout phases - is 15 weeks). Adittionally, untill up to 6 months during open label extension phase.