Cannabinoid Therapy for Pediatric Epilepsy

• ClinicalTrials.gov Identifier: NCT02983695
• Recruitment Status: Completed
• First Posted: December 6, 2016
• Last Update Posted: November 16, 2021
• Sponsor: The Hospital for Sick Children
• Collaborator: Tilray
• Information provided by (Responsible Party): Carter Snead, The Hospital for Sick Children

Tracking Information

• First Submitted Date: November 17, 2016
• First Posted Date: December 6, 2016
• Last Update Posted Date: November 16, 2021
• Actual Study Start Date: February 2, 2017
• Actual Primary Completion Date: August 24, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 2, 2016)

• 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 [ Time Frame: 20-64 weeks ]
  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) [ Time Frame: 16-64 weeks ]
  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.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 2, 2016)

• The impact of therapy on quality of life using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire [ Time Frame: 16-52 weeks ]
  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. [ Time Frame: 20-64 weeks ]
  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 [ Time Frame: 20 weeks ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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.
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Epilepsy

Intervention

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.

Study Arms

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).

Intervention: Drug: TIL-TC150

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 2, 2016): 20
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: July 20, 2021
• Actual Primary Completion Date: August 24, 2018 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 1 Year to 18 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada

Administrative Information

• NCT Number: NCT02983695
• Other Study ID Numbers: 1000047417
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Carter Snead, The Hospital for Sick Children
• Original Responsible Party: Blathnaid McCoy, The Hospital for Sick Children, Principal Investigator
• Current Study Sponsor: The Hospital for Sick Children
• Original Study Sponsor: Same as current
• Collaborators: Tilray

Investigators

• Principal Investigator: Blathnaid McCoy, MB BCh BAO; The Hospital for Sick Children

• PRS Account: The Hospital for Sick Children
• Verification Date: November 2021