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

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.

Conditions

• Refractory Epilepsy in Children
• Refractory Epilepsy
• Epilepsy

Interventions

DRUG

Cannabidiol (CBD) oral solution

Bedrolite, which is a full spectrum cannabis extract with 10%CBD/0.4%THC. Cannabidiol will be titrated up to a dosage of 2.5-20mg/kg/day, dosed twice daily.

OTHER

Placebo

Cannabis extract with less than 0.5% CBD/0.02%THC

Sponsors & Collaborators

• Stichting Epilepsie Instellingen Nederland

  collaborator OTHER

• Epilepsiecentrum Kempenhaeghe

  collaborator INDUSTRY

• Erasmus Medical Center

  collaborator OTHER

• Maastricht University Medical Center

  collaborator OTHER

• ZonMw: The Netherlands Organisation for Health Research and Development

  collaborator OTHER

• UMC Utrecht

  lead OTHER

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

CROSSOVER

Eligibility

Min Age

1 Year

Max Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-02-06

Primary Completion

2027-11-30

Completion

2027-12-31

Countries

• Netherlands

Study Locations