Verified December 2016 by The Hospital for Sick Children
Sponsor:
Collaborator:
Tilray
Information provided by (Responsible Party):
Blathnaid McCoy, The Hospital for Sick Children
ClinicalTrials.gov Identifier:
NCT02983695
First received: November 17, 2016
Last updated: December 2, 2016
Last verified: December 2016
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.
| Epilepsy | Drug: TIL-TC150 | Phase 1 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Cannabinoid Therapy in Medically Refractory Pediatric Epilepsy – Phase 1: Dosing and Tolerability Study of a Cannabidiol-Rich Whole Plant Extract of Cannabis. |
- 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 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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.
- The impact of therapy on quality of life using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire [ Time Frame: 16-52 weeks ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 20 |
| Study Start Date: | January 2017 |
| Estimated Study Completion Date: | July 2018 |
| Estimated Primary Completion Date: | June 2017 (Final data collection date for primary outcome measure) |
| 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 |
Drug: TIL-TC150 |
| Ages Eligible for Study: | 1 Year to 18 Years (Child, Adult) |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Exclusion Criteria:
- Co-morbid liver or renal disease
- Without clinically-apparent seizures
- Current or previous therapy with cannabinoids
Please refer to this study by its ClinicalTrials.gov identifier: NCT02983695
The Hospital for Sick Children
Tilray
| Principal Investigator: | Blathnaid McCoy, MB BCh BAO | The Hospital for Sick Children |
| Responsible Party: | Blathnaid McCoy, Principal Investigator, The Hospital for Sick Children |
| ClinicalTrials.gov Identifier: | NCT02983695 History of Changes |
| Other Study ID Numbers: | 1000047417 |
| Study First Received: | November 17, 2016 |
| Last Updated: | December 2, 2016 |
| Health Authority: | Canada: Health Canada Canada: Ethics Review Committee |
| Individual Participant Data | |
| Plan to Share IPD: | No |
Additional relevant MeSH terms:
| Epilepsy Brain Diseases Central Nervous System Diseases Nervous System Diseases |
ClinicalTrials.gov processed this record on December 06, 2016