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Cannabidiol in Children With Refractory Epileptic Encephalopathy (CARE-E)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03024827
Recruitment Status : Active, not recruiting
First Posted : January 19, 2017
Last Update Posted : September 28, 2021
Information provided by (Responsible Party):
Richard Huntsman, University of Saskatchewan

Brief Summary:
This study will assess the safety and tolerability of a cannabidiol-enriched Cannabis Herbal Extract in a small group of children with refractory epileptic encephalopathy. The dosage of Cannabis Herbal Extract will be gradually increased over a four month time period.

Condition or disease Intervention/treatment Phase
Epileptic Encephalopathy Drug: CanniMed® 1:20 Phase 1

Detailed Description:

Epileptic Encephalopathies are a group of epilepsies that develop in children. These epilepsies can cause frequent and difficult to control seizures. Because of the ongoing seizures, these epilepsies can also cause cognitive impairment and neurological impairment.

In many children with these Epileptic Encephalopathies, seizures are difficult to control with medical treatment, such as anti-convulsants or non-drug treatments like the ketogenic diet (a high fat, adequate-protein, low-carbohydrate diet). This has resulted in a need to find therapies that are effective and better tolerated for children with epileptic encephalopathies.

There is very limited data regarding the use of cannabis products in children, in particular cannabidiol-enriched cannabis oil in children with epilepsy. However, hemp oil products with high cannabidiol and low tetrahydrocannabinol ratios have been reported to provide seizure relief and cognitive improvement in children who take them.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Cannabidiol in Children With Refractory Epileptic Encephalopathy: A Phase 1 Open Label Dose Escalation Study (CARE-E)
Actual Study Start Date : April 26, 2017
Actual Primary Completion Date : February 1, 2020
Estimated Study Completion Date : January 1, 2022

Arm Intervention/treatment
Experimental: Medical Cannabis Oil
CanniMed® 1:20
Drug: CanniMed® 1:20
A cannabidiol (CBD): tetrahydrocannabinol (Δ9 THC) 20:1 ratio product will be provided as an oil-based suspension.
Other Name: Medical Cannabis Oil

Primary Outcome Measures :
  1. Heart Rate [ Time Frame: Up to 6 months ]
  2. Blood Pressure [ Time Frame: Up to 6 months ]
  3. Weight [ Time Frame: Up to 6 months ]
  4. Complete Blood Count (CBC) and Differential [ Time Frame: Up to 6 months ]
  5. Sodium, potassium, chloride, calcium, magnesium, phosphate and carbon dioxide (mmol/L) [ Time Frame: Up to 6 months ]
  6. Blood Urea Nitrogen (mmol/L) [ Time Frame: Up to 6 months ]
  7. Creatinine (umol/L) [ Time Frame: Up to 6 months ]
  8. Aspartate aminotransferase (AST), Alanine aminotransferase (ALT), Alkaline phosphatase (ALP), Gamma-glutamyl transferase (GGT) and Lipase (U/L) [ Time Frame: Up to 6 months ]
  9. Total and Direct Bilirubin (umol/L) [ Time Frame: Up to 6 months ]
  10. Albumin (g/L) [ Time Frame: Up to 6 months ]
  11. Total Cholesterol and Triglyceride (mmol/L) [ Time Frame: Up to 6 months ]
  12. Clobazam and Norclobazam Levels (umol/L) [ Time Frame: Up to 6 months ]
    For participants taking clobazam who become excessively sedated

  13. Clonazepam Level (umol/L) [ Time Frame: Up to 6 months ]
    For participants taking clonazepam who become excessively sedated

  14. Urine Ketones [ Time Frame: Up to 6 months ]
    For participants on the ketogenic diet

  15. Trough Level of Concomitant Anti-Convulsants [ Time Frame: Up to 7 months ]
    Measure interactions with any anti-convulsants participants may be already on

  16. Adverse Events [ Time Frame: Through study completion, up to 7 months ]
    Side effect rating scale, includes items related to sleepiness/lethargy, irritability, nausea/vomiting and diarrhea

  17. 2-hour Electroencephalogram (EEG) Recording [ Time Frame: Up to 6 months ]

Secondary Outcome Measures :
  1. Seizure Frequency Log Book [ Time Frame: Through study completion, up to 7 months ]
  2. Modified Quality of Life in Children with Epilepsy Questionnaire (QOLCE) [ Time Frame: Through study completion, up to 7 months ]
  3. Cannabidiol (CBD) (ng/mL) [ Time Frame: Up to 7 months ]
    Levels in blood

  4. Tetrahydrocannabinol (Δ9-THC) (ng/mL) [ Time Frame: Up to 7 months ]
    Levels in blood

  5. 11-nor-9-carboxy-tetrahydrocannabinol (11-nor-9-Carboxy-THC) (ng/mL) [ Time Frame: Up to 7 months ]
    Levels in blood

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age 1-10 years
  • Epileptic Encephalopathy
  • A minimum of at least 1 major seizure per week or 4 major seizures per month. For the purposes of this research study, a major seizure would include atonic, tonic, clonic, tonic-clonic, major myoclonic, myoclonic astatic seizures and epileptic spasms (including infantile spasms)
  • Refractory to anticonvulsant medication as per the International League Against Epilepsy (ILAE) Definition of failing 2 appropriate anticonvulsants at therapeutic doses
  • The ability to attend appointments regularly
  • Negative pregnancy test at screening for females who have reached menarche

Exclusion Criteria:

  • Recent (<1 month) change in anticonvulsant therapies including anticonvulsant medications, ketogenic diet or settings on Vagal Nerve Stimulator
  • Recent (<6 months) change in intravenous immunoglobulin (IVIG) treatment
  • Initiation of ketogenic diet within 6 months (Patients must be on the ketogenic diet for at least 6 months to prevent any delayed response from the ketogenic diet affecting study results)
  • Implantation and activation of Vagal Nerve Stimulator within 12 months (Patients may have a vagal nerve stimulator for at least one year once again to prevent delayed response from the vagal nerve stimulator affecting study results)
  • Use of cannabis-based therapy within 2 months (Participants who have previously used a cannabis based therapy may be included if they have a 2 month period without use of cannabis based therapy prior to enrolment in the study)
  • Use of selective serotonin reuptake inhibitor (SSRI), tricyclic antidepressant or atypical neuroleptic medication in last month
  • Concomitant regular use of narcotics (Use of narcotics in emergency situations and supervised by a physician is allowed)
  • Initiation or dosage change of oral or injected steroids within 3 months
  • Allergy or known intolerance to any of the compounds within the study preparation
  • Inability of study participants to attend assessments on a monthly basis
  • Clinically significant cardiac, renal or hepatic disease (as assessed by the site investigator)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03024827

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Canada, British Columbia
University of British Columbia
Vancouver, British Columbia, Canada, V6T1Z4
Canada, Manitoba
University of Manitoba
Winnipeg, Manitoba, Canada, R3T 2N2
Canada, Quebec
Universite de Montreal
Montreal, Quebec, Canada, H3T1J4
Canada, Saskatchewan
University of Saskatchewan
Saskatoon, Saskatchewan, Canada, S7N5C5
Sponsors and Collaborators
University of Saskatchewan
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Principal Investigator: Richard Huntsman, MD University of Saskatchewan
Principal Investigator: Richard Tang-Wai, MD Loma Linda University
Additional Information:
Publications of Results:
Other Publications:
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Responsible Party: Richard Huntsman, MD, University of Saskatchewan
ClinicalTrials.gov Identifier: NCT03024827    
Other Study ID Numbers: CARE-E-01
First Posted: January 19, 2017    Key Record Dates
Last Update Posted: September 28, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Richard Huntsman, University of Saskatchewan:
Infantile Spasms
Lennox Gastaut Syndrome
Doose Syndrome
Continuous Spike Wave in Sleep
Landau-Kleffner Syndrome
Dravet Syndrome
Malignant Migrating Partial Seizures of Infancy (MMPSI)
Additional relevant MeSH terms:
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Brain Diseases
Central Nervous System Diseases
Nervous System Diseases