<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/NCT02239276</url>
  </required_header>
  <id_info>
    <org_study_id>2014-047</org_study_id>
    <nct_id>NCT02239276</nct_id>
  </id_info>
  <brief_title>Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies</brief_title>
  <official_title>Expanded Access Use of Stiripentol in Participants With Dravet Syndrome or Epileptic Encephalopathies Associated With Sodium Channel Mutations</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Cook Children's Health Care System</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Cook Children's Health Care System</source>
  <brief_summary>
    <textblock>
      This is an expanded access use of Stiripentol in Dravet Syndrome or epileptic&#xD;
      encephalopathies associated with sodium channel mutations who have failed other drugs in an&#xD;
      effort to give them the best chance at seizure control and quality of life. As a treatment&#xD;
      protocol and not a research study, children will only be monitored on a clinical basis for&#xD;
      seizure improvement and side effects predominantly by parent and caregiver report.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The initial dose of Stiripentol will be determined by the prescribing neurologist and&#xD;
      titrated up to an initial goal dose of 50 mg/kg/day divided into 2 to 3 doses per day.&#xD;
      Further dose increases by 10-20 mg/kg/day increments up to a max of 100 mg/kg/day or 4000 mg&#xD;
      total daily dose may be necessary for improved seizure control.&#xD;
&#xD;
      Stiripentol is available as gelatin capsules and powder sachets (250 mg, 500 mg). The same&#xD;
      granule formulation (i.e. active, PVP and portion of sodium starch glycolate) used for the&#xD;
      capsule is used in the final powder blend with a few additional excipients. Depending upon&#xD;
      patient weight, the 250 mg or 500 mg formulation will be utilized for each participant.&#xD;
&#xD;
      Caretakers will be queried about common adverse effects including drowsiness, tremor, ataxis,&#xD;
      nausea, anorexia, weight loss, and emesis. Intolerable adverse effects will prompt dose&#xD;
      reduction or withholding medication.&#xD;
&#xD;
      Monitoring of these and other potential AEs will occur during study visits and&#xD;
      participant-initiated telephone calls throughout the study. Safety events and tolerability&#xD;
      will be recorded as adverse events (AE) or serious adverse events (SAE).&#xD;
&#xD;
      Physical examination, weight, vital signs, and laboratory tests (cbc, complete metabolic&#xD;
      panel, and AED levels) will be conducted at baseline and at least every 6 months and as&#xD;
      clinically warranted.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>No longer available</overall_status>
  <study_type>Expanded Access</study_type>
  <condition>Dravet Syndrome</condition>
  <condition>Epileptic Encephalopathies Associated With SCN1A Mutations</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Stiripentol</intervention_name>
    <description>The initial dose of Stiripentol will be determined by the prescribing neurologist and titrated up to an initial goal dose of 50 mg/kg/day divided into 2 to 3 doses per day. Further dose increases by 10-20 mg/kg/day increments up to a max of 100 mg/kg/day or 4000 mg total daily dose may be necessary for improved seizure control.</description>
    <other_name>Diacomit</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  6 months and older&#xD;
&#xD;
          -  Diagnosis of Dravet Syndrome or epileptic encephalopathies associated with SCN1A&#xD;
             mutations defined as:&#xD;
&#xD;
               -  A documented gene mutation reported to result in Dravet syndrome phenotype; OR&#xD;
&#xD;
               -  Clinical confirmation of Dravet syndrome by two pediatric neurologists; OR&#xD;
&#xD;
               -  Clinical confirmation of other epileptic encephalopathies associated with sodium&#xD;
                  channel mutations&#xD;
&#xD;
          -  Failure of at least 2 therapeutic anticonvulsants (excluding Na Channel blockers)&#xD;
             indicative of intractable seizures&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Hypersensitivity to the active substance or to any of the excipients&#xD;
&#xD;
          -  Past history of psychoses in the form of episodes of delirium&#xD;
&#xD;
          -  Impaired hepatic and/or renal function, defined as creatinine &gt;2 and/or transaminase&#xD;
             &gt;4xULN&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Months</minimum_age>
    <maximum_age>18 Years</maximum_age>
  </eligibility>
  <overall_official>
    <last_name>Scott Perry, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Cook Children's Health Care System</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Cook Children's Medical Center</name>
      <address>
        <city>Fort Worth</city>
        <state>Texas</state>
        <zip>76104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.cookchildrens.org/Pages/Default.aspx</url>
    <description>Cook Children's Health Care System website</description>
  </link>
  <verification_date>January 2020</verification_date>
  <study_first_submitted>September 10, 2014</study_first_submitted>
  <study_first_submitted_qc>September 10, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 12, 2014</study_first_posted>
  <last_update_submitted>January 31, 2020</last_update_submitted>
  <last_update_submitted_qc>January 31, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">February 5, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Stiripentol</keyword>
  <keyword>Dravet syndrome</keyword>
  <keyword>Epilepsy</keyword>
  <keyword>Epileptic Encephalopathies</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Brain Diseases</mesh_term>
    <mesh_term>Epilepsy</mesh_term>
    <mesh_term>Epilepsies, Myoclonic</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Stiripentol</mesh_term>
  </intervention_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/NCT02239276
org study id: 2014-047
nct id: NCT02239276
lead sponsor: 
This is an expanded access use of Stiripentol in Dravet Syndrome or epileptic
      encephalopathies associated with sodium channel mutations who have failed other drugs in an
      effort to give them the best chance at seizure control and quality of life. As a treatment
      protocol and not a research study, children will only be monitored on a clinical basis for
      seizure improvement and side effects predominantly by parent and caregiver report.
The initial dose of Stiripentol will be determined by the prescribing neurologist and
      titrated up to an initial goal dose of 50 mg/kg/day divided into 2 to 3 doses per day.
      Further dose increases by 10-20 mg/kg/day increments up to a max of 100 mg/kg/day or 4000 mg
      total daily dose may be necessary for improved seizure control.

      Stiripentol is available as gelatin capsules and powder sachets (250 mg, 500 mg). The same
      granule formulation (i.e. active, PVP and portion of sodium starch glycolate) used for the
      capsule is used in the final powder blend with a few additional excipients. Depending upon
      patient weight, the 250 mg or 500 mg formulation will be utilized for each participant.

      Caretakers will be queried about common adverse effects including drowsiness, tremor, ataxis,
      nausea, anorexia, weight loss, and emesis. Intolerable adverse effects will prompt dose
      reduction or withholding medication.

      Monitoring of these and other potential AEs will occur during study visits and
      participant-initiated telephone calls throughout the study. Safety events and tolerability
      will be recorded as adverse events (AE) or serious adverse events (SAE).

      Physical examination, weight, vital signs, and laboratory tests (cbc, complete metabolic
      panel, and AED levels) will be conducted at baseline and at least every 6 months and as
      clinically warranted.
intervention type: Drug
intervention name: Stiripentol
description: The initial dose of Stiripentol will be determined by the prescribing neurologist and titrated up to an initial goal dose of 50 mg/kg/day divided into 2 to 3 doses per day. Further dose increases by 10-20 mg/kg/day increments up to a max of 100 mg/kg/day or 4000 mg total daily dose may be necessary for improved seizure control.
other name: Diacomit
criteria: 
gender: All
minimum age: 6 Months
maximum age: 18 Years
last name: Scott Perry, MD
role: Principal Investigator
affiliation: Cook Children's Health Care System
facility: 
country: United States
url: http://www.cookchildrens.org/Pages/Default.aspx
description: Cook Children's Health Care System website
responsible party type: Sponsor
mesh term: Brain Diseases
mesh term: Epilepsy
mesh term: Epilepsies, Myoclonic
mesh term: Syndrome
mesh term: Stiripentol