Back to resultsCannabinoid Therapy for Pediatric Epilepsy
Primary Purpose
Epilepsy
Status
Completed
Phase
Phase 1
Locations
Canada
Study Type
Interventional
Intervention
TIL-TC150
Sponsored by
About this trial
This is an interventional treatment trial for Epilepsy
Eligibility Criteria
Inclusion Criteria:
- Drug resistant epilepsy due to Dravet syndrome
- Must have clinically apparent seizures
- Must be able to tolerate administration of medication orally or enterally via gastrostomy tube
- Was never on Cannabinoid therapy or have not been treated with Cannabinoid products for at least last 60 days (confirmed by negative urine test for Tetrahydrocannabidiol (THC), only for those who had been treated with CBD).
Exclusion Criteria:
- Co-morbid liver or renal disease
- Without clinically-apparent seizures
- Currently taking any Cannabinoid products.
Sites / Locations
- The Hospital for Sick Children
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
treatment
Arm Description
all participants will be in this arm and will receive study drug 'Cannabidiol-Rich whole Plant Extract (TIL-TC150) to assess dosing and tolerability according to study protocol.TIL-TC150 Oil is the study product The active ingredients in TIL-TC150 Oil are THC and CBD, present in a 1:50 ratio. These active ingredients are derived from Cannabis sativa L. strains produced by Tilray, and suspended in a grape seed oil. This suspension is administered at a dose of 2mg/kg/day CBD divided BID and titrated up to a maximal dose of 16mg/kg/day CBD (or maximal tolerated).
Outcomes
Primary Outcome Measures
Establishment of tolerability of TIL-TC150 by measuring the number of participants with adverse events and / or abnormal laboratory values that are related to treatment
During this study, the dose of TIL-TC150 will be titrated according to study protocol and participant tolerance. Tolerance will be assessed as documented in title, by evaluating for adverse events clinically and by parent report of adverse event every week during the first 20 weeks of the study, also by blood work at weeks 0, 4, and between 8-16 weeks.
For those choose to continue the study drug after 20 week period of assessment, tolerance will be evaluated by assessing for adverse events at weeks 28, 40,52 and 64.
Establishment of tolerability of TIL-TC150 by standardised side effects questionnaire -pediatric epilepsy side effects questionnaire (PESQ)
During this study, tolerance will also be assessed by the standardised side effects questionnaire- PESQ in clinic at weeks 2,4,8,12 and 16. For those choosing to continue therapy will be reassessed by PESQ in clinic at week 28,40,52 and 64.
Secondary Outcome Measures
The impact of therapy on quality of life using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire
The QOLCE will be completed by parents at clinic at first visit or baseline(week 0), at maximal dose (8 weeks) and at 16 weeks for primary analysis. For those choosing to continue therapy will be reassessed at 28 weeks and 52 weeks. Parents will be provided with a research report detailing the assessment findings at study completion.
The impact of therapy on everyday behaviours using the Vineland Adaptive Behavior Scales, Second Edition.
Vineland Adaptive Behaviour Scale, Second Edition: is a standardized parent interview to complete either in person at the clinic or by telephone on week 0, prior to starting the TIL-TC150. The interview will also be completed again at the primary evaluation period anytime between weeks 16 and 20 and again for those choosing to continue the therapy will be reassessed during the longitudinal follow-up anytime between 52 and 64 weeks. This will be done by study team. Parents will be provided with a research report detailing the assessment findings at study completion.
change in seizure frequency from baseline
Full Information
NCT ID
NCT02983695
First Posted
November 17, 2016
Last Updated
November 12, 2021
Sponsor
The Hospital for Sick Children
Collaborators
Tilray
1. Study Identification
Unique Protocol Identification Number
NCT02983695
Brief Title
Cannabinoid Therapy for Pediatric Epilepsy
Official Title
Cannabinoid Therapy in Medically Refractory Pediatric Epilepsy - Phase 1: Dosing and Tolerability Study of a Cannabidiol-Rich Whole Plant Extract of Cannabis.
Study Type
Interventional
2. Study Status
Record Verification Date
November 2021
Overall Recruitment Status
Completed
Study Start Date
February 2, 2017 (Actual)
Primary Completion Date
August 24, 2018 (Actual)
Study Completion Date
July 20, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The Hospital for Sick Children
Collaborators
Tilray
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a Phase 1 trial to determine the tolerability and optimal dose of CBD rich cannabis extract as an adjunct treatment in children with severe drug resistant epilepsy due to Dravet Syndrome. This is an open label intervention. Study duration is 20 weeks to primary analysis with continued follow-up until 64 weeks completed.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epilepsy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
treatment
Arm Type
Experimental
Arm Description
all participants will be in this arm and will receive study drug 'Cannabidiol-Rich whole Plant Extract (TIL-TC150) to assess dosing and tolerability according to study protocol.TIL-TC150 Oil is the study product The active ingredients in TIL-TC150 Oil are THC and CBD, present in a 1:50 ratio. These active ingredients are derived from Cannabis sativa L. strains produced by Tilray, and suspended in a grape seed oil. This suspension is administered at a dose of 2mg/kg/day CBD divided BID and titrated up to a maximal dose of 16mg/kg/day CBD (or maximal tolerated).
Intervention Type
Drug
Intervention Name(s)
TIL-TC150
Intervention Description
The active ingredients in TIL-TC150 are tetrahydrocannabinol (THC) and Cannabidiol (CBD), isolated from the Cannabis sativa L. strains produced by Tilray, and formulated into an oral liquid drug product.
Primary Outcome Measure Information:
Title
Establishment of tolerability of TIL-TC150 by measuring the number of participants with adverse events and / or abnormal laboratory values that are related to treatment
Description
During this study, the dose of TIL-TC150 will be titrated according to study protocol and participant tolerance. Tolerance will be assessed as documented in title, by evaluating for adverse events clinically and by parent report of adverse event every week during the first 20 weeks of the study, also by blood work at weeks 0, 4, and between 8-16 weeks.
For those choose to continue the study drug after 20 week period of assessment, tolerance will be evaluated by assessing for adverse events at weeks 28, 40,52 and 64.
Time Frame
20-64 weeks
Title
Establishment of tolerability of TIL-TC150 by standardised side effects questionnaire -pediatric epilepsy side effects questionnaire (PESQ)
Description
During this study, tolerance will also be assessed by the standardised side effects questionnaire- PESQ in clinic at weeks 2,4,8,12 and 16. For those choosing to continue therapy will be reassessed by PESQ in clinic at week 28,40,52 and 64.
Time Frame
16-64 weeks
Secondary Outcome Measure Information:
Title
The impact of therapy on quality of life using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire
Description
The QOLCE will be completed by parents at clinic at first visit or baseline(week 0), at maximal dose (8 weeks) and at 16 weeks for primary analysis. For those choosing to continue therapy will be reassessed at 28 weeks and 52 weeks. Parents will be provided with a research report detailing the assessment findings at study completion.
Time Frame
16-52 weeks
Title
The impact of therapy on everyday behaviours using the Vineland Adaptive Behavior Scales, Second Edition.
Description
Vineland Adaptive Behaviour Scale, Second Edition: is a standardized parent interview to complete either in person at the clinic or by telephone on week 0, prior to starting the TIL-TC150. The interview will also be completed again at the primary evaluation period anytime between weeks 16 and 20 and again for those choosing to continue the therapy will be reassessed during the longitudinal follow-up anytime between 52 and 64 weeks. This will be done by study team. Parents will be provided with a research report detailing the assessment findings at study completion.
Time Frame
20-64 weeks
Title
change in seizure frequency from baseline
Time Frame
20 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Drug resistant epilepsy due to Dravet syndrome
Must have clinically apparent seizures
Must be able to tolerate administration of medication orally or enterally via gastrostomy tube
Was never on Cannabinoid therapy or have not been treated with Cannabinoid products for at least last 60 days (confirmed by negative urine test for Tetrahydrocannabidiol (THC), only for those who had been treated with CBD).
Exclusion Criteria:
Co-morbid liver or renal disease
Without clinically-apparent seizures
Currently taking any Cannabinoid products.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Blathnaid McCoy, MB BCh BAO
Organizational Affiliation
The Hospital for Sick Children
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Cannabinoid Therapy for Pediatric Epilepsy
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