Cannabinoid Therapy for Pediatric Epilepsy

Cannabinoid Therapy in Medically Refractory Pediatric Epilepsy - Phase 1: Dosing and Tolerability Study of a Cannabidiol-Rich Whole Plant Extract of Cannabis.

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.

Study Overview

• Status: Completed
• Conditions: Epilepsy
• Intervention / Treatment: Drug: TIL-TC150

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 1

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

EXPERIMENTAL: treatment; 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).

Drug: TIL-TC150; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	20-64 weeks
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.	16-64 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	16-52 weeks
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.	20-64 weeks
change in seizure frequency from baseline		20 weeks

Collaborators and Investigators

• Sponsor: The Hospital for Sick Children
• Collaborators: Tilray
• Investigators: Principal Investigator: Blathnaid McCoy, MB BCh BAO, The Hospital for Sick Children

Publications and helpful links

Helpful Links

• Research Article: A prospective open-label trial of a CBD/THC cannabis oil in dravet syndrome

Study record dates

Study Major Dates

• Study Start (ACTUAL): February 2, 2017
• Primary Completion (ACTUAL): August 24, 2018
• Study Completion (ACTUAL): July 20, 2021

Study Registration Dates

• First Submitted: November 17, 2016
• First Submitted That Met QC Criteria: December 2, 2016
• First Posted (ESTIMATE): December 6, 2016

Study Record Updates

• Last Update Posted (ACTUAL): November 16, 2021
• Last Update Submitted That Met QC Criteria: November 12, 2021
• Last Verified: November 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy
• Other Study ID Numbers: 1000047417

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No