Study of CBD Oil as Add-On Treatment for Children Aged 1 to 18 With Severe Refractory Epilepsy Using Personalized N-of-1 Trial Design

What this Trial Is About

Researchers are evaluating whether cannabidiol CBD oil can effectively treat severe epilepsy in children aged 1 to 18 years with difficult-to-control seizures. This phase 3 clinical trial aims to determine if CBD reduces seizure frequency and influences other outcomes such as behavior, sleep, communication, and daily activities. The study also examines the safety of cannabidiol and monitors medical problems that may arise from its use. Participants will receive both cannabidiol and a placebo oil in different treatment periods within one to three cycles. Each cycle lasts about 15 weeks and includes two periods of approximately 7 weeks each, with titration phases to determine the tolerated dose of CBD, up to 20 mgkgday or a maximum of 1000 mg daily. The placebo resembles the CBD oil but contains minimal active ingredients. Participants will alternate between CBD and placebo during the cycles. Throughout the study, participants will record seizures daily using a digital app during up to 6 periods. They will have blood tests every 7 weeks to monitor safety and possible side effects. Clinic visits occur every 15 weeks for checkups and additional tests. Questionnaires measuring quality of life, functioning, sleep, behavior, and epilepsy severity are completed regularly. The main outcome measured is seizure frequency from baseline through treatment cycles, with secondary outcomes including seizure-free days and various patient-centered scales. An open-label extension of up to 6 months may follow for continued monitoring.

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