<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/NCT02229032</url>
  </required_header>
  <id_info>
    <org_study_id>14-0294</org_study_id>
    <nct_id>NCT02229032</nct_id>
  </id_info>
  <brief_title>Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population</brief_title>
  <official_title>Genetic Analysis Between Charlotte's Web Responders Versus Non- Responders in a Dravet Population</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Colorado, Denver</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Denver Health Medical Center</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>University of Colorado, Denver</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      There is tremendous curiosity about medical marijuana and the treatment of epilepsy. In a&#xD;
      specific genetic epilepsy known as Dravet Syndrome, a mutation occurs affecting the SCN1A&#xD;
      gene. A specific strain of marijuana known as Charlotte's Web, available in Colorado, may&#xD;
      have activity in this catastrophic epilepsy syndrome. Anecdotal reports suggest both success&#xD;
      and lack of response with this therapy. Genetic analysis of the differences between Dravet&#xD;
      responders and non-responders may prove useful for identifying patients likely to be helped&#xD;
      by this therapy, as well as shed light on the putative mechanisms by which marijuana may&#xD;
      exert any antiepileptic effect.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 2014</start_date>
  <completion_date type="Actual">July 2017</completion_date>
  <primary_completion_date type="Actual">July 2017</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Cross-Sectional</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>genetic differences between patients with Dravet Syndrome (SCN1A gene mutation) who appear to respond to high concentration cannabidiol (CBD) oil therapy versus those who do not.</measure>
    <time_frame>1 day</time_frame>
    <description>Patients completing three months of therapy with a seizure reduction of &gt;50% will be designated a &quot;Responder&quot; and patients who discontinue therapy for any reason prior to 3 months or have a &lt;=50% seizure reduction will be designated &quot;Non-Responder&quot;. Between-groups genetic analysis will be performed</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">19</enrollment>
  <condition>Dravet Syndrome</condition>
  <arm_group>
    <arm_group_label>Dravet Sydrome</arm_group_label>
    <description>Patients with Dravet Syndrome who are self-seeking therapy with Charlotte's Web strain of medical marijuana with the assistance of a medical marijuana doctor, but are still naïve to therapy</description>
  </arm_group>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      saliva for DNA analysis&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with Dravet Syndrome, ages 1-50, who are self- seeking therapy with Charlotte's&#xD;
        Web strain of medical marijuana with the assistance of a medical marijuana doctor, but are&#xD;
        still naïve to therapy&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  age 1-50 with gene confirmed Dravet Syndrome&#xD;
&#xD;
          -  uncontrolled epilepsy, having failed at least 2 prior treatments (including&#xD;
             appropriate Anti-epileptic drugs, ketogenic diet, vagus nerve stimulation Therapy)&#xD;
&#xD;
          -  Seizure frequency should be more than 2 visually countable motor seizures per week&#xD;
             (tonic clonic, tonic, clonic, myoclonic, or astatic)&#xD;
&#xD;
          -  care providers able to identify convulsive seizures, drop attacks, or tonic seizures,&#xD;
             or any combination of countable seizure types&#xD;
&#xD;
          -  self- seeking therapy with Charlotte's Web strain of medical marijuana with the&#xD;
             assistance of a medical marijuana doctor, but are still naïve to therapy&#xD;
&#xD;
          -  care provider able to complete seizure count diary&#xD;
&#xD;
          -  able to obtain oral swab sample from subject with Dravet Syndrome&#xD;
&#xD;
          -  agree to forfeit any right to results of genetic analysis&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  subjects having non-epileptic seizures that are not distinguishable from epileptic&#xD;
             seizures by the care provider&#xD;
&#xD;
          -  prisoners&#xD;
&#xD;
          -  pregnant women&#xD;
&#xD;
          -  failure to forfeit right to results of genetic analysis&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>50 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Edward Maa, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Denver Health Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Denver Health Medical Center</name>
      <address>
        <city>Denver</city>
        <state>Colorado</state>
        <zip>80204</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2017</verification_date>
  <study_first_submitted>August 27, 2014</study_first_submitted>
  <study_first_submitted_qc>August 28, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 29, 2014</study_first_posted>
  <last_update_submitted>December 13, 2017</last_update_submitted>
  <last_update_submitted_qc>December 13, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">December 15, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Dravet Syndrome</keyword>
  <keyword>cannabidiol therapy</keyword>
  <keyword>Charlotte's Web</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Epilepsies, Myoclonic</mesh_term>
  </condition_browse>
  <!-- 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/NCT02229032
org study id: 14-0294
nct id: NCT02229032
lead sponsor: 
collaborator: 
has dmc: No
There is tremendous curiosity about medical marijuana and the treatment of epilepsy. In a
      specific genetic epilepsy known as Dravet Syndrome, a mutation occurs affecting the SCN1A
      gene. A specific strain of marijuana known as Charlotte's Web, available in Colorado, may
      have activity in this catastrophic epilepsy syndrome. Anecdotal reports suggest both success
      and lack of response with this therapy. Genetic analysis of the differences between Dravet
      responders and non-responders may prove useful for identifying patients likely to be helped
      by this therapy, as well as shed light on the putative mechanisms by which marijuana may
      exert any antiepileptic effect.
observational model: Cohort
time perspective: Cross-Sectional
measure: genetic differences between patients with Dravet Syndrome (SCN1A gene mutation) who appear to respond to high concentration cannabidiol (CBD) oil therapy versus those who do not.
time frame: 1 day
description: Patients completing three months of therapy with a seizure reduction of >50% will be designated a "Responder" and patients who discontinue therapy for any reason prior to 3 months or have a <=50% seizure reduction will be designated "Non-Responder". Between-groups genetic analysis will be performed
arm group label: Dravet Sydrome
description: Patients with Dravet Syndrome who are self-seeking therapy with Charlotte's Web strain of medical marijuana with the assistance of a medical marijuana doctor, but are still naïve to therapy
saliva for DNA analysis
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: 50 Years
healthy volunteers: No
last name: Edward Maa, MD
role: Principal Investigator
affiliation: Denver Health Medical Center
facility: 
country: United States
responsible party type: Sponsor
mesh term: Epilepsies, Myoclonic