<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT02983695</url>
  </required_header>
  <id_info>
    <org_study_id>1000047417</org_study_id>
    <nct_id>NCT02983695</nct_id>
  </id_info>
  <brief_title>Cannabinoid Therapy for Pediatric Epilepsy</brief_title>
  <official_title>Cannabinoid Therapy in Medically Refractory Pediatric Epilepsy - Phase 1: Dosing and Tolerability Study of a Cannabidiol-Rich Whole Plant Extract of Cannabis.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>The Hospital for Sick Children</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Tilray</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>The Hospital for Sick Children</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a Phase 1 trial to determine the tolerability and optimal dose of CBD rich cannabis&#xD;
      extract as an adjunct treatment in children with severe drug resistant epilepsy due to Dravet&#xD;
      Syndrome. This is an open label intervention. Study duration is 20 weeks to primary analysis&#xD;
      with continued follow-up until 64 weeks completed.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">February 2, 2017</start_date>
  <completion_date type="Actual">July 20, 2021</completion_date>
  <primary_completion_date type="Actual">August 24, 2018</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>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</measure>
    <time_frame>20-64 weeks</time_frame>
    <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.&#xD;
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.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Establishment of tolerability of TIL-TC150 by standardised side effects questionnaire -pediatric epilepsy side effects questionnaire (PESQ)</measure>
    <time_frame>16-64 weeks</time_frame>
    <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.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The impact of therapy on quality of life using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire</measure>
    <time_frame>16-52 weeks</time_frame>
    <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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The impact of therapy on everyday behaviours using the Vineland Adaptive Behavior Scales, Second Edition.</measure>
    <time_frame>20-64 weeks</time_frame>
    <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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>change in seizure frequency from baseline</measure>
    <time_frame>20 weeks</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Epilepsy</condition>
  <arm_group>
    <arm_group_label>treatment</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <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).</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>TIL-TC150</intervention_name>
    <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.</description>
    <arm_group_label>treatment</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Drug resistant epilepsy due to Dravet syndrome&#xD;
&#xD;
          -  Must have clinically apparent seizures&#xD;
&#xD;
          -  Must be able to tolerate administration of medication orally or enterally via&#xD;
             gastrostomy tube&#xD;
&#xD;
          -  Was never on Cannabinoid therapy or have not been treated with Cannabinoid products&#xD;
             for at least last 60 days (confirmed by negative urine test for Tetrahydrocannabidiol&#xD;
             (THC), only for those who had been treated with CBD).&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Co-morbid liver or renal disease&#xD;
&#xD;
          -  Without clinically-apparent seizures&#xD;
&#xD;
          -  Currently taking any Cannabinoid products.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Blathnaid McCoy, MB BCh BAO</last_name>
    <role>Principal Investigator</role>
    <affiliation>The Hospital for Sick Children</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>The Hospital for Sick Children</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 1X8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
  </location_countries>
  <verification_date>November 2021</verification_date>
  <study_first_submitted>November 17, 2016</study_first_submitted>
  <study_first_submitted_qc>December 2, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 6, 2016</study_first_posted>
  <last_update_submitted>November 12, 2021</last_update_submitted>
  <last_update_submitted_qc>November 12, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">November 16, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>The Hospital for Sick Children</investigator_affiliation>
    <investigator_full_name>Carter Snead</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Epilepsy</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>June 25, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/95/NCT02983695/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02983695
org study id: 1000047417
nct id: NCT02983695
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: 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
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.
measure: Establishment of tolerability of TIL-TC150 by standardised side effects questionnaire -pediatric epilepsy side effects questionnaire (PESQ)
time frame: 16-64 weeks
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.
measure: The impact of therapy on quality of life using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire
time frame: 16-52 weeks
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.
measure: The impact of therapy on everyday behaviours using the Vineland Adaptive Behavior Scales, Second Edition.
time frame: 20-64 weeks
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.
measure: change in seizure frequency from baseline
time frame: 20 weeks
arm group label: treatment
arm group type: Experimental
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: TIL-TC150
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.
arm group label: treatment
criteria: 
gender: All
minimum age: 1 Year
maximum age: 18 Years
healthy volunteers: No
last name: Blathnaid McCoy, MB BCh BAO
role: Principal Investigator
affiliation: The Hospital for Sick Children
facility: 
country: Canada
responsible party type: Principal Investigator
investigator affiliation: The Hospital for Sick Children
investigator full name: Carter Snead
investigator title: Principal Investigator
mesh term: Epilepsy
sharing ipd: No
provided document: 