A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to Epidiolex

NCT06924827 | Not Yet Recruiting Phase 4

• 1 other identifier: 3524
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to learn the best way to switch children with Lennox-Gastaut Syndrome (LGS) or Dravet Syndrome (DS) taking 'artisanal' (non pharmaceutical-grade) cannabidiol (CBD) to Epidiolex for treatment of seizures. The main questions it aims to answer are:

• How well does a gradual switch from 'artisanal' CBD to Epidiolex work?
• Does the same dose of Epidiolex as 'artisanal' CBD work best?
• What side-effects or medical problems do participants have when switching from 'artisanal' CBD to Epidiolex? Researchers will examine how successful switching from 'artisanal' CBD to Epidiolex is. Participants will:
• Gradually increase their dose of Epidiolex and reduce their dose of 'artisanal' CBD until they are taking just Epidiolex
• Visit the clinic five times over 20 weeks for checkups and tests
• Keep a diary of their seizures, symptoms and the number of times they use a rescue seizure medication

Conditions

Dravet Syndrome (DS); Lennox-Gastaut Syndrome (LGS)

Keywords

Cannabidiol (CBD); Epidiolex; Seizures; Epilepsy; Lennox-Gastaut Syndrome (LGS); Dravet Syndrome (DS)

Outcome Measures

Primary Outcomes (1)

• The success rate of transition of children from 'artisanal' CBD to Epidiolex.

  Participants successfully transitioned from 'artisanal' CBD to Epidiolex if they complete the transition protocol and continue treatment with Epidiolex at visit 2 following the two-week transition phase of the study. The success rate will be determined as the percentage of participants that complete the two-week transition phase.

  Baseline (visit 2, Day 1) through visit 4 (Day 15), the two-week transition phase.

Secondary Outcomes (16)

• The acceptability rate of Epidiolex in children previously treated with 'artisanal' CBD.

  Baseline (visit 2, Day 15) to end of treatment (visit 7, Day 85).

• The change in seizure frequency following transition of children from 'artisanal' CBD to Epidiolex.

  Baseline (visit 2, Day 1) through the end of treatment (visit 7, Day 85).

• Change in reported Pediatric Epilepsy Side-Effects Questionnaire (PESQ) score.

  Baseline (visit 2, Day 1) through visit 4 (Day 15), visit 5 (Day 29) and end of treatment (visit 7, Day 85).

• Change Clinical Global Impression of Improvement (CGI-I) score.

  Baseline (visit 2, Day 1) through visit 5 (Day 29) and end of treatment (visit 7, Day 85).

• Change in Clinical Global Impression of Seizure Intensity/Duration (CGI-CSID) score.

  Baseline (visit 2, Day 1) through visit 5 (Day 29) and end of treatment (visit 7, Day 85).

Other Outcomes (2)

• Change in Short Form 12 (SF-12) score.

  Baseline (visit 2, Day 1) through end of treatment (visit 7, Day 85).

• Motivation for switching from 'artisanal' CBD to Epidiolex.

  Baseline (visit 1, Day -28), once at beginning of study.

Study Arms (1)

Epidiolex Treatment

EXPERIMENTAL

The participant's dose of Epidiolex will be matched to their dose of 'artisanal' cannabidiol (CBD). Epidiolex will be titrated over two weeks with a concomitant taper of the participant's 'artisanal' CBD. CBD doses during transition week 1 will consist of 75% 'artisanal' and 25% Epidiolex, followed by 50% 'artisanal' and 50% Epidiolex for transition week 2. Participants will commence a CBD dose comprised of 100% Epidiolex once reaching the maintenance period of the study. Participants will remain on their matched dose of Epidiolex throughout the maintenance period, unless a dose modification is clinically indicated for efficacy, safety or tolerability. The daily dose of Epidiolex should not exceed the maximum approved dose of 20 mg/kg/day.

Drug: Epidiolex 100 mg/mL Oral Solution

Interventions

Epidiolex 100 mg/mL Oral Solution DRUG

The participant's 'artisanal' CBD and Epidiolex dose should be taken consistently with food or consistently without food throughout the entire study. The participant's dosing with or without food should be consistent with their method of dosing of 'artisanal' CBD prior to screening. Oral administration is recommended. When necessary, Epidiolex can be enterally administered via silicone feeding tubes, such as nasogastric or gastrostomy tubes.

Epidiolex Treatment

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or female aged 2 through 18 years, inclusive.
• Clinical diagnosis of Dravet or Lennox Gastaut Syndrome:
• Clinical diagnosis of Dravet Syndrome supported by:
• Onset of seizures within the first year of life.
• Initial seizures present as fever-induced or fever-triggered seizures, hemi-clonic, generalized tonic-clonic, prolonged seizures (more than 15 minutes).
• Emergence of other seizure types after 1 year of age.
• Normal development within the first year of age, then emergence of neurodevelopmental difficulties or delay.
• Clinical diagnosis of Lennox Gastaut Syndrome supported by:
• a. History of an EEG with slow/disorganized background and slow (\<2.5 Hz or less) spike and wave activity or generalized paroxysmal fast activity (GPFA).
• b. History of more than 1 type of generalized seizures, including drop seizures (tonic, atonic or tonic-clonic).
• Participant must be willing and able to give written informed consent for participation. If the participant is not qualified or unable to provide written consent based on age, development, intellectual capacity or other factors, the parent or legally authorized representative must provide written informed consent on their behalf.
• Must be on a stable dose of a licensed artisanal cannabidiol (CBD) product as maintenance therapy for seizure control for a minimum of 3 months prior to screening (visit 1).
• 'Artisanal' CBD dose must be between 5 mg/kg/day and 20mg/kg/day.
• 'Artisanal' CBD preparation must be a high CBD to THC formulation defined as a minimum CBD:THC ratio of 20:1.
• Must be taking a minimum of 1 other anti-seizure medication (ASM) in addition to an 'artisanal' form of CBD.

You may not qualify if:

• Previous or current exposure to Epidiolex.
• Supplemental use of cannabinoid-containing products, including but not limited to:
• Recreational use of cannabis.
• Use of artisanal CBD as a seizure rescue medication.
• Use of more than one formulation of 'artisanal' CBD (e.g. THC supplementation).
• Pregnant or breastfeeding.
• Any clinically significant, unstable medical condition other than epilepsy that, in the opinion of the investigator, could place the participant at increased risk or interfere with the results of the study.
• Hepatic impairment at screening (visit 1) defined as either of the following conditions:
• ALT or AST \> 5x upper limit of normal (ULN).
• ALT or AST \> 3x ULN and total bilirubin \>2x ULN (or international normalized ratio \>1.5).
• Known sensitivity to any ingredient in Epidiolex, including sesame and sesame oil.
• Unwillingness to refrain from alcohol consumption throughout the duration of the study.
• Unwillingness of females of childbearing potential to use a highly effective form of birth control. Acceptable methods include: hormonal contraceptives, intra-uterine devices, bilateral tube occlusion, vasectomized partner and sexual abstinence.
• Currently enrolled in another clinical trial.
• Have suicidal plan/intent, active suicidal thoughts, or a suicide attempt in the past 6 month prior to screening.

Sponsors & Collaborators

• Elizabeth Donner: lead
• Jazz Pharmaceuticals: collaborator
• Alberta Children's Hospital: collaborator
• BC Children's Hospital Research Institute: collaborator

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

MeSH Terms

Conditions

Epilepsies, Myoclonic; Lennox Gastaut Syndrome; Seizures; Epilepsy

Interventions

Cannabidiol; Solutions

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals; Pharmaceutical Preparations

Study Officials

• Elizabeth Donner, MD

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Laura MacDougall, PhD

CONTACT

416-813-7996; laura.macdougall@sickkids.ca

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Head, Division of Neurology

Study Record Dates

• First Submitted: April 4, 2025
• First Posted: April 11, 2025
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2029
• Last Updated: March 17, 2026

Record last verified: 2026-03

Locations

CA (1)