A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to Epidiolex
Trial statusNot yet recruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age2-18
SponsorElizabeth Donner
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female aged 2 through 18 years, inclusive.
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.
Disqualifiers
Previous or current exposure to Epidiolex.
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).
Trial design
Design model
Single group
Treatments tested in this trial
Epidiolex 100 mg/mL Oral Solution
DrugThe 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.
Treatment groups
25 Participants
are divided into 1 treatment groupGroup A: Epidiolex TreatmentExperimental treatment 1 intervention
Trial outcomes
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.
Time frame
Baseline (visit 2, Day 1) through visit 4 (Day 15), the two-week transition phase.Secondary outcomes
1
The acceptability rate of Epidiolex in children previously treated with 'artisanal' CBD.
Participants accepted transition to Epidiolex if they continued treatment with Epidiolex after the last scheduled visit of the maintenance period (visit 7). The acceptability rate will be determined as the percentage of participants that continue treatment with Epidiolex after the last scheduled visit of the maintenance period (visit 7, Day 85).
Time frame
Baseline (visit 2, Day 15) to end of treatment (visit 7, Day 85).2
The change in seizure frequency following transition of children from 'artisanal' CBD to Epidiolex.
The percent change (increase or decrease) from baseline in 28-day seizure frequency during the maintenance period. Seizure frequency will be calculated as the total number of seizures divided by the number of days with seizure data, multiplied by 28.
Time frame
Baseline (visit 2, Day 1) through the end of treatment (visit 7, Day 85).3
Change in reported Pediatric Epilepsy Side-Effects Questionnaire (PESQ) score.
The Pediatric Epilepsy Side Effects Questionnaire (PESQ) is a rating scale to assess pediatric participant-reported side effects associated with anti-seizure medication treatment. Each side-effect is rated on a 6-point Likert scale as follows: (1) - "Not present", (2) - "Low severity", (3) - "Low-moderate severity", (4) - "Moderate severity", (5) - "Moderate-high severity", (6) - "High severity". Higher scores indicated worse side-effects.
Time frame
Baseline (visit 2, Day 1) through visit 4 (Day 15), visit 5 (Day 29) and end of treatment (visit 7, Day 85).4
Change Clinical Global Impression of Improvement (CGI-I) score.
The Clinical Global Impression of Improvement (CGI-I) is a 7-point Likert scale used to rate the overall change in seizure control, behaviour, safety and tolerability after transitioning to Epidiolex relative to baseline. The participant's overall improvement is rated by the clinician and caregiver as: 1- "very much improved", 2- "much improved', 3- "minimally improved", 4- "no change", 5- "minimally worse", 6- "much worse", and 7- "very much worse". Higher scores indicate worse condition.
Time frame
Baseline (visit 2, Day 1) through visit 5 (Day 29) and end of treatment (visit 7, Day 85).Other outcomes
1
Motivation for switching from 'artisanal' CBD to Epidiolex.
The switching questionnaire consists of two multiple choice questions, along with an open-ended question designed to understand the caregiver's primary and secondary reasons for transitioning their child from 'artisanal' CBD to Epidiolex.
Time frame
Baseline (visit 1, Day -28), once at beginning of study.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Elizabeth Donner
Lead sponsor
The Hospital for Sick Children
Sponsor institution
Jazz Pharmaceuticals
Collaborator
Source ClinicalTrials.gov
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