The protocol for the Cannabidiol in children with refractory epileptic encephalopathy (CARE-E) study: a phase 1
Darren Reithmeier1,2, Richard Tang-Wai1,3,4, Blair Seifert1,5
1Cannabinoid Research Initiative of Saskatchewan (CRIS), University of Saskatchewan, Saskatoon, Saskatchewan, Canada.
Insights
This study evaluated the safety and tolerability of cannabidiol (CBD)-enriched Cannabis extract in children with refractory epilepsy. The research monitored seizure frequency, duration, and quality of life improvements, providing crucial data for pediatric epilepsy treatment.
Area of Science:
- Pediatric Neurology
- Pharmacology
- Cannabinoid Research
Background:
- Pharmaceutical-grade cannabidiol (CBD) shows promise in reducing seizures and improving quality of life for children with epileptic encephalopathies.
- Limited access to pharmaceutical CBD has led to increased availability of Cannabis extracts, raising safety concerns due to other cannabinoids like Δ9-tetrahydrocannabinol (Δ9-THC).
- Physicians express reluctance to recommend Cannabis extracts due to insufficient high-quality safety data.
Purpose of the Study:
- To determine the safety and tolerability of CBD-enriched Cannabis extract in pediatric patients with refractory epilepsy.
- To monitor the impact of CBD-enriched Cannabis extract on seizure frequency and duration.
- To assess the effects on patients' quality of life.
Main Methods:
- An open-label, dose-escalation phase 1 trial involving 28 children (aged 1-10 years) with treatment-resistant epileptic encephalopathy.
- Recruitment across four Canadian cities.
- Primary objectives focused on safety, tolerability, and seizure control; secondary objectives included assessing anticonvulsant medication levels, quality of life, cannabinoid pharmacokinetics, and adverse event incidence.
Main Results:
- This paper outlines the study design for a Phase 1 trial of CBD-enriched Cannabis herbal extract in children with treatment-resistant epileptic encephalopathy.
- The study aims to provide the first high-quality analysis of the safety of CBD-enriched Cannabis herbal extract in pediatric patients.
- Analysis will focus on dosage and pharmacokinetics of active cannabinoids.
Conclusions:
- The study protocol describes a Phase 1 trial investigating CBD-enriched Cannabis herbal extract for pediatric refractory epilepsy.
- It will provide crucial data on safety, tolerability, and efficacy in a vulnerable patient population.
- Findings will inform clinical recommendations regarding the use of cannabis-based therapies in pediatric epilepsy.
Background:
Initial studies suggest pharmaceutical grade cannabidiol (CBD) can reduce the frequency of convulsive seizures and lead to improvements in quality of life in children affected by epileptic encephalopathies. With limited access to pharmaceutical CBD, Cannabis extracts in oil are becoming increasingly available. Physicians show reluctance to recommend Cannabis extracts given the lack of high quality safety data especially regarding the potential for harm caused by other cannabinoids, such as Δ9-tetrahydrocannabinol (Δ9-THC). The primary aims of the study presented in this protocol are (i) To determine whether CBD enriched Cannabis extract is safe and well-tolerated for pediatric patients with refractory epilepsy, (ii) To monitor the effects of CBD-enriched Cannabis extract on the frequency and duration of seizure types and on quality of life.
Methods:
Twenty-eight children with treatment resistant epileptic encephalopathy ranging in age from 1 to 10 years will be recruited in four Canadian cities into an open-label, dose-escalation phase 1 trial. The primary objectives for the study are (i) To determine if the CBD-enriched Cannabis herbal extract is safe and well-tolerated for pediatric patients with treatment resistant epileptic encephalopathy and (ii) To determine the effect of CBD-enriched Cannabis herbal extract on the frequency and duration of seizures. Secondary objectives include (i) To determine if CBD-enriched Cannabis herbal extracts alter steady-state levels of co-administered anticonvulsant medications. (ii) To assess the relation between dose escalation and quality of life measures, (iii) To determine the relation between dose escalation and steady state trough levels of bioactive cannabinoids. (iv) To determine the relation between dose escalation and incidence of adverse effects.
Discussion:
This paper describes the study design of a phase 1 trial of CBD-enriched Cannabis herbal extract in children with treatment-resistant epileptic encephalopathy. This study will provide the first high quality analysis of safety of CBD-enriched Cannabis herbal extract in pediatric patients in relation to dosage and pharmacokinetics of the active cannabinoids.
Trial Registration:
http://clinicaltrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). 2016 Dec 16. Identifier NCT03024827, Cannabidiol in Children with Refractory Epileptic Encephalopathy: CARE-E; 2017 Jan 19 [cited 2017 Oct]; Available from: http://clinicaltrials.gov/ct2/show/NCT03024827.
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