Cannabidiol as add-on therapy for children with refractory epilepsy (CBD-uN1que): an individualized N-of-1 series

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This interventional study uses a series of N-of-1 trials to determine if cannabidiol (CBD) as an add-on can reduce seizure frequency in children aged 1 to 18 with refractory epilepsy. Each participant experiences both CBD and placebo in alternating periods, allowing an individualized comparison of seizure outcomes and potential effects on behavior, sleep, and communication. Safety and tolerability of CBD are also assessed. Seizure data are collected via a digital tracking tool, and results are interpreted per patient to identify who may benefit most from CBD. The study is conducted at institutions in the Netherlands, led by UMC Utrecht, with collaboration from Epilepsie Instellingen Nederland and Kempenhaeghe, Erasmus Medical Center.

Why it matters

Who can join: the full rules

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 epilepsy surgery
* 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
* 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
* 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
* 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

Where it's running

• University Medical Center Utrecht
  Utrecht, Utrecht, Netherlands Recruiting

Study contacts

• Study coordinator: Marit van de Wiel, MD
• Email: m.vandewiel@umcutrecht.nl
• Phone: 088 755 5555

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.