<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/NCT04462770</url>
  </required_header>
  <id_info>
    <org_study_id>EPX-100-001</org_study_id>
    <nct_id>NCT04462770</nct_id>
  </id_info>
  <brief_title>EPX-100 (Clemizole Hydrochloride) as Add-on Therapy to Control Convulsive Seizures in Patients With Dravet Syndrome</brief_title>
  <acronym>ARGUS</acronym>
  <official_title>A 20-Week Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of EPX-100 (Clemizole Hydrochloride) as Adjunctive Therapy in Children and Adult Participants With Dravet Syndrome (ARGUS Trial)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Epygenix</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Epygenix</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to evaluate the safety and efficacy of EPX-100 as adjunctive&#xD;
      therapy in children and adult participants with Dravet Syndrome.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a global, multicenter, randomized, double-blind, placebo-controlled study to evaluate&#xD;
      the safety and efficacy of EPX-100 as adjunctive therapy in children and adult participants&#xD;
      with Dravet Syndrome. The study consists of a 4-week observational period, a 16-week double&#xD;
      blind period and a 3-year open label extension.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">September 15, 2020</start_date>
  <completion_date type="Anticipated">December 2024</completion_date>
  <primary_completion_date type="Anticipated">December 2024</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <intervention_model_description>Patients are randomized 1:1 to EPX-100 or placebo.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Triple (Participant, Care Provider, Investigator)</masking>
    <masking_description>Matching active vs placebo solutions have been prepared including color and taste.</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>The mean percent change in countable convulsive seizure frequency (CCSF1) in the Titration and Maintenance (T+M) periods relative to baseline.</measure>
    <time_frame>20 weeks</time_frame>
    <description>The mean percent change in countable convulsive seizure frequency (CCSF1) in the Titration and Maintenance (T+M) periods relative to baseline.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The difference between EPX-100 vs placebo in the number of countable convulsive seizure-free days in the T+M periods relative to baseline.</measure>
    <time_frame>20 weeks</time_frame>
    <description>The difference between EPX-100 vs placebo in the number of countable convulsive seizure-free days in the T+M periods relative to baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The difference between EPX-100 vs placebo in proportion of participants with &gt;50% reduction in the mean CCSF in the T+M periods relative to baseline.</measure>
    <time_frame>20 weeks</time_frame>
    <description>The difference between EPX-100 vs placebo in proportion of participants with &gt;50% reduction in the mean CCSF in the T+M periods relative to baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The total change in Seizure Severity using Hague Seizure Severity Scale (HASS).</measure>
    <time_frame>20 weeks</time_frame>
    <description>The total change in Seizure Severity using Hague Seizure Severity Scale (HASS).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The improvement in Clinical Global Impression (CGI).</measure>
    <time_frame>20 weeks</time_frame>
    <description>The improvement in Clinical Global Impression (CGI).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The total change in Quality of Life in Childhood Epilepsy short form (QOLCE 55).</measure>
    <time_frame>20 weeks</time_frame>
    <description>The total change in Quality of Life in Childhood Epilepsy short form (QOLCE 55).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The difference between EPX-100 vs placebo in proportion of participants with &gt;25% reduction in the mean CCSF in the T+M periods relative to baseline.</measure>
    <time_frame>20 weeks</time_frame>
    <description>The difference between EPX-100 vs placebo in proportion of participants with &gt;25% reduction in the mean CCSF in the T+M periods relative to baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The mean difference between EPX-100 vs placebo per 28-days in the percent change of all seizures in the T+M periods relative to the baseline.</measure>
    <time_frame>20 weeks</time_frame>
    <description>The mean difference between EPX-100 vs placebo per 28-days in the percent change of all seizures in the T+M periods relative to the baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The change in the number of episodes of status epilepticus, in the T+M periods relative to the baseline.</measure>
    <time_frame>20 weeks</time_frame>
    <description>The change in the number of episodes of status epilepticus, in the T+M periods relative to the baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The incidence of rescue anti-epileptic drug (AED) use between EPX-100 and placebo, as measured by the number of days on rescue AEDs in the T+M periods relative to the baseline.</measure>
    <time_frame>20 weeks</time_frame>
    <description>The incidence of rescue anti-epileptic drug (AED) use between EPX-100 and placebo, as measured by the number of days on rescue AEDs in the T+M periods relative to the baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The individual item changes in Quality of Life in Childhood Epilepsy short form (QOLCE 55).</measure>
    <time_frame>20 weeks</time_frame>
    <description>The individual item changes in Quality of Life in Childhood Epilepsy short form (QOLCE 55).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The individual item changes in Seizure Severity using Hague Seizure Severity Scale (HASS).</measure>
    <time_frame>20 weeks</time_frame>
    <description>The individual item changes in Seizure Severity using Hague Seizure Severity Scale (HASS).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The change in the Sleep Disturbance Scale for Children (SDSC) between EPX-100 and placebo by each scheduled visit</measure>
    <time_frame>20 weeks</time_frame>
    <description>The change in the Sleep Disturbance Scale for Children (SDSC) between EPX-100 and placebo by each scheduled visit</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">100</enrollment>
  <condition>Dravet Syndrome</condition>
  <arm_group>
    <arm_group_label>Active arm with EPX-100 (Clemizole HCl)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>EPX-100 oral solution</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo arm</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Color- and taste-matched placebo oral solution dosed to match the active arm.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>EPX-100 (Clemizole HCl)</intervention_name>
    <description>Daily dose of EPX100</description>
    <arm_group_label>Active arm with EPX-100 (Clemizole HCl)</arm_group_label>
    <other_name>Clemizole Hydrochloride</other_name>
    <other_name>Clemizole HCl</other_name>
    <other_name>Clemizole</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Daily dose of Placebo</description>
    <arm_group_label>Placebo arm</arm_group_label>
    <other_name>Placebo to match EPX-100 solution</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Male and female participants 2 years and older at time of consent.&#xD;
&#xD;
          2. Participant or parent/Legally Authorized Representative (LAR) willing and able to&#xD;
             provide written informed consent, assent (if applicable) prior to initiation of any&#xD;
             study related procedures.&#xD;
&#xD;
          3. Clinical diagnosis of Dravet Syndrome. Participants must have seizures which are not&#xD;
             completely controlled by AEDs with the following criteria:&#xD;
&#xD;
               -  Onset of seizures prior to 18 months of age,&#xD;
&#xD;
               -  Normal development at onset,&#xD;
&#xD;
               -  History of seizures that are generalized, unilateral clonic, and/or hemiclonic,&#xD;
&#xD;
               -  Brain MRI without cortical malformation (not including mild atrophy associated&#xD;
                  with the natural progression of Dravet Syndrome), and&#xD;
&#xD;
               -  Genetic mutation of the SCN1A gene must be documented.&#xD;
&#xD;
          4. The participant must be approved to participate by the Independent Reviewer, in&#xD;
             collaboration with the PI. Participants will be approved for participation following&#xD;
             review of the participant's medical and seizure history, historical neuroimaging,&#xD;
             historical EEGs, genetic report confirming SCN1A mutation, and review and&#xD;
             classification of at least 28 days of baseline seizures.&#xD;
&#xD;
          5. ≥4 countable convulsive seizures within minimum 28-day screening/baseline period&#xD;
             (e.g., hemiclonic, secondarily generalized tonic-clonic, generalized tonic-clonic,&#xD;
             tonic, clonic, tonic/atonic (resulting in a drop), and focal with clear observable&#xD;
             motor signs).&#xD;
&#xD;
          6. Participants should be on a stable regimen of AEDs ≥30 days prior to Visit 1 and&#xD;
             generally in good health.&#xD;
&#xD;
          7. Participant or parent/ LAR is able and willing to maintain an accurate and complete&#xD;
             daily seizure and medication diary for the duration of the trial.&#xD;
&#xD;
          8. Sexually active women of child-bearing potential (WCBP) must be using a medically&#xD;
             acceptable method of birth control and have a negative serum or urine pregnancy test&#xD;
             at the screening (Visit 1) and Randomization (Visit 2). A WCBP is defined as a female&#xD;
             who is biologically capable of becoming pregnant. A medically acceptable method of&#xD;
             birth control includes intrauterine devices in place for at least 3 months, surgical&#xD;
             sterilization, or adequate barrier methods (e.g., diaphragm and foam). Use of oral&#xD;
             contraceptives in combination with another method (e.g., a spermicidal cream) is&#xD;
             acceptable. In participants who are not sexually active, abstinence is an acceptable&#xD;
             form of birth control and urine will be tested per protocol. Women who are of&#xD;
             non-child-bearing potential, i.e., post-menopause, must have this condition captured&#xD;
             in their medical history. Pregnant women are excluded from this study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        The presence of any of the following excludes a participant from study enrollment:&#xD;
&#xD;
          1. Known sensitivity, allergy, or previous exposure to EPX-100 (clemizole HCl).&#xD;
&#xD;
          2. Exposure to any investigational drug or device &lt;90 days prior to screening or plans to&#xD;
             participate in another drug or device trial at any time during the study.&#xD;
&#xD;
          3. Seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating&#xD;
             disease, degenerative neurological disease, or central nervous system (CNS) disease&#xD;
             deemed progressive, metabolic illness, or progressive degenerative disease.&#xD;
&#xD;
          4. Concurrent use of drugs known to interfere with EPX-100, including moderate or severe&#xD;
             inducers or inhibitors of CYP3A4/5/7. Specifically, concurrent use of carbamazepine,&#xD;
             oxcarbazepine and/or phenytoin, as well as refraining from grapefruits and grapefruit&#xD;
             juice during the study period. Refer to Appendix 1 for a list of prohibited drugs.&#xD;
&#xD;
          5. Prior or concurrent use of lorcaserin.&#xD;
&#xD;
          6. Concurrent use of fenfluramine. Participants with prior use of fenfluramine within the&#xD;
             previous 3 months, or without proper documentation of an echocardiogram, at minimum 3&#xD;
             months following the last dose of fenfluramine, to ensure that the participant does&#xD;
             not meet any criteria for drug-related (fenfluramine) cardiac valvular heart disease&#xD;
             and/or drug-related pulmonary arterial hypertension (PAH) as indicated by any of the&#xD;
             following:&#xD;
&#xD;
               -  documented mild or greater aortic regurgitation [AR] or moderate or greater&#xD;
                  mitral regurgitation [MR]&#xD;
&#xD;
               -  significant (greater than mild) tricuspid regurgitation&#xD;
&#xD;
               -  abnormally thickened cardiac valve and/or has restricted motion of the valve&#xD;
                  leaflets&#xD;
&#xD;
               -  elevated right heart/pulmonary artery pressure &gt;35mmHg&#xD;
&#xD;
          7. Has any medical condition that, in the PI's judgment, is considered to be clinically&#xD;
             significant and could potentially affect participant safety or study outcome,&#xD;
             including but not limited to: clinically significant cardiac disease (including&#xD;
             angina, congestive heart failure, uncontrolled hypertension, and history of&#xD;
             arrhythmias), renal, pulmonary, gastrointestinal, hematologic or hepatic conditions;&#xD;
             or a condition that affects the absorption, distribution, metabolism, or excretion of&#xD;
             drugs.&#xD;
&#xD;
          8. Has an active suicidal plan/intent or have had active suicidal thoughts in the past 6&#xD;
             months or a suicide attempt in the past 3 years.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Lorianne Masouka, MD</last_name>
    <role>Study Director</role>
    <affiliation>Epygenix Therapeutics, Inc.</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Lorianne Masuoka, M.D.</last_name>
    <phone>(415) 933-0826</phone>
    <email>lm@epygenix.com</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Rebekah DeVitry Fries</last_name>
    <email>rfries@epygenix.com</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Children's Hospital of Los Angeles</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90027</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Martha Arellano-Garcia</last_name>
      <phone>323-361-5812</phone>
      <email>margarcia@chla.usc.edu</email>
    </contact>
    <investigator>
      <last_name>Deborah L Holder, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>UCSF Medical Center</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94158</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Kristina Pfeifer</last_name>
      <phone>415-353-8440</phone>
      <email>kristina.pfeifer@ucsf.edu</email>
    </contact>
    <investigator>
      <last_name>Ernesto Gonzalez-Giraldo, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Nemours Children's Hospital-DE</name>
      <address>
        <city>Wilmington</city>
        <state>Delaware</state>
        <zip>19803</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Kimberly Klipner, RN</last_name>
      <phone>302-344-1528</phone>
      <email>kklipner@nemours.org</email>
    </contact>
    <investigator>
      <last_name>Stephen Falchek, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Ann &amp; Robert H. Lurie Children's Hospital of Chicago</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Maya Stosic, MD</last_name>
      <phone>312-227-4525</phone>
      <email>mstosic@luriechildrens.org</email>
    </contact>
    <investigator>
      <last_name>Linda C Laux, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Michigan- Mott Children's Hospital</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Brittany Nordhaus</last_name>
      <phone>734-936-4179</phone>
      <email>nbrittan@med.umich.edu</email>
    </contact>
    <investigator>
      <last_name>Julie Ziobro, MD, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Northeast Regional Epilepsy Group</name>
      <address>
        <city>Hackensack</city>
        <state>New Jersey</state>
        <zip>07601</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Terminated</status>
  </location>
  <location>
    <facility>
      <name>Wake Forest Baptist Health Sciences Department of Neurology</name>
      <address>
        <city>Winston-Salem</city>
        <state>North Carolina</state>
        <zip>27157</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Carolyn Hedrick</last_name>
      <phone>336-716-8694</phone>
      <email>cwhedric@wakehealth.edu</email>
    </contact>
    <investigator>
      <last_name>Gautam Popli, Professor</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Samantha Ballard, BSN, RN, CPN</last_name>
      <phone>513-803-3177</phone>
      <email>samantha.ballard@cchmc.org</email>
    </contact>
    <investigator>
      <last_name>Gewalin Aungaroon, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Cleveland Clinic</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <contact>
      <last_name>Honglian Huang, PhD</last_name>
      <phone>216-445-2366</phone>
      <email>huangh2@ccf.org</email>
    </contact>
    <investigator>
      <last_name>Elia Margarita Pestana Knight, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jennie Minnick</last_name>
      <phone>215-419-3212</phone>
      <email>minnick@email.chop.edu</email>
    </contact>
    <investigator>
      <last_name>Eric Marsh, MD, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Le Bonheur Children's Hospital</name>
      <address>
        <city>Memphis</city>
        <state>Tennessee</state>
        <zip>38103</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Kenishia Guy</last_name>
      <phone>901-287-7484</phone>
      <email>Kenishia.Guy@lebonheur.org</email>
    </contact>
    <investigator>
      <last_name>James Wheless, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Child Neurology Consultants of Austin</name>
      <address>
        <city>Austin</city>
        <state>Texas</state>
        <zip>78757</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Victoria Henderson</last_name>
      <phone>210-416-4163</phone>
      <email>Victoria.Henderson@mednax.com</email>
    </contact>
    <investigator>
      <last_name>Karen Keough, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84112</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Laura Beeler</last_name>
      <phone>801-585-9266</phone>
      <email>laura.beeler@hsc.utah.edu</email>
    </contact>
    <investigator>
      <last_name>Angela Peters, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Seattle Children's</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Laurie Guidry</last_name>
      <phone>206-987-0058</phone>
      <email>laurie.guidry@seattlechildrens.org</email>
    </contact>
    <investigator>
      <last_name>Russell P Saneto, DO, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>UBC Children's Hospital Research Institute</name>
      <address>
        <city>Vancouver</city>
        <state>British Columbia</state>
        <zip>V6H 3V4</zip>
        <country>Canada</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Inderpal Gill</last_name>
      <phone>604-875-2345</phone>
      <phone_ext>6834</phone_ext>
      <email>inderpal.gill@cw.bc.ca</email>
    </contact>
    <investigator>
      <last_name>Mary Connolly, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Eastern Ontario</name>
      <address>
        <city>Ottawa</city>
        <state>Ontario</state>
        <zip>K1H 8L1</zip>
        <country>Canada</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sarah Healy</last_name>
      <phone>613-738-4297</phone>
      <email>shealy@cheo.on.ca</email>
    </contact>
    <investigator>
      <last_name>Sharon Whiting, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <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>
    <status>Recruiting</status>
    <contact>
      <last_name>Laura MacDougall, PhD, CCRP</last_name>
      <phone>416-813-7996</phone>
      <email>laura.macdougall@sickkids.ca</email>
    </contact>
    <contact_backup>
      <last_name>Christine Kowal</last_name>
      <phone>416-813-7653</phone>
      <email>christine.kowal@sickkids.ca</email>
    </contact_backup>
    <investigator>
      <last_name>Elizabeth Donner, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Toronto Western Hospital, University Health Network</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5T 2S8</zip>
        <country>Canada</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sarah Selvadurai, MD</last_name>
      <phone>(416) 603-5800</phone>
      <phone_ext>5906</phone_ext>
      <email>sarah.selvadurai2@uhn.ca</email>
    </contact>
    <investigator>
      <last_name>Danielle M Andrade, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>LEPL Tbilisi State Medical University, Givi Zhvania Academic, Clinic of Pediatry</name>
      <address>
        <city>Tbilisi</city>
        <zip>0159</zip>
        <country>Georgia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Tinatin Nadiradze</last_name>
      <phone>00995592552929</phone>
      <email>t.nadiradze@tsmu.edu</email>
    </contact>
    <investigator>
      <last_name>Sophia Bakhtadze, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>LTD Medi Club Georgia LLC</name>
      <address>
        <city>Tbilisi</city>
        <zip>0160</zip>
        <country>Georgia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Tamar Gachechiladze</last_name>
      <phone>00995598357909</phone>
      <email>tamogachechiladze@yahoo.com</email>
    </contact>
    <investigator>
      <last_name>Gia Melikishvili, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>LTD Institute of Neurology and Neuropsychology</name>
      <address>
        <city>Tbilisi</city>
        <zip>0186</zip>
        <country>Georgia</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sopiko Digmelashvili</last_name>
      <phone>00995551516066</phone>
      <email>sopikodigmelashvili@gmail.com</email>
    </contact>
    <investigator>
      <last_name>David Kvernadze, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Semmelweis University First Department of Pediatrics, Division of Child Neurology</name>
      <address>
        <city>Budapest</city>
        <zip>1083</zip>
        <country>Hungary</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Andrásné Koród</last_name>
      <phone>0036 06 20 825 8208</phone>
      <email>korodi.kata@@gmail.com</email>
    </contact>
    <investigator>
      <last_name>Victor Farkas, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Hospital de la Santa Creu i Sant Pau</name>
      <address>
        <city>Barcelona</city>
        <state>Catalonia</state>
        <zip>08025</zip>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Roser Ribosa</last_name>
      <phone>+ 34618846 138</phone>
      <email>RRibosa@santpau.cat</email>
    </contact>
    <investigator>
      <last_name>Susana Boronat, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Infantil Niño Jesús</name>
      <address>
        <city>Madrid</city>
        <state>Madrid Provincia</state>
        <zip>28009</zip>
        <country>Spain</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Nuria Ventosino</last_name>
      <phone>+34 616 531 224</phone>
      <phone_ext>7351</phone_ext>
      <email>nuria.ventosino@salud.madrid.org</email>
    </contact>
    <investigator>
      <last_name>Victor Soto, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>Georgia</country>
    <country>Hungary</country>
    <country>Spain</country>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2022</verification_date>
  <study_first_submitted>July 6, 2020</study_first_submitted>
  <study_first_submitted_qc>July 6, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 8, 2020</study_first_posted>
  <last_update_submitted>June 22, 2023</last_update_submitted>
  <last_update_submitted_qc>June 22, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 23, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Clemizole Hydrochloride</keyword>
  <keyword>Convulsive Seizure</keyword>
  <keyword>Pediatric epilepsy</keyword>
  <keyword>Dravet</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Epilepsies, Myoclonic</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT04462770
org study id: EPX-100-001
nct id: NCT04462770
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to evaluate the safety and efficacy of EPX-100 as adjunctive
      therapy in children and adult participants with Dravet Syndrome.
This is a global, multicenter, randomized, double-blind, placebo-controlled study to evaluate
      the safety and efficacy of EPX-100 as adjunctive therapy in children and adult participants
      with Dravet Syndrome. The study consists of a 4-week observational period, a 16-week double
      blind period and a 3-year open label extension.
allocation: Randomized
intervention model: Parallel Assignment
intervention model description: Patients are randomized 1:1 to EPX-100 or placebo.
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
masking description: Matching active vs placebo solutions have been prepared including color and taste.
measure: The mean percent change in countable convulsive seizure frequency (CCSF1) in the Titration and Maintenance (T+M) periods relative to baseline.
time frame: 20 weeks
description: The mean percent change in countable convulsive seizure frequency (CCSF1) in the Titration and Maintenance (T+M) periods relative to baseline.
measure: The difference between EPX-100 vs placebo in the number of countable convulsive seizure-free days in the T+M periods relative to baseline.
time frame: 20 weeks
description: The difference between EPX-100 vs placebo in the number of countable convulsive seizure-free days in the T+M periods relative to baseline.
measure: The difference between EPX-100 vs placebo in proportion of participants with >50% reduction in the mean CCSF in the T+M periods relative to baseline.
time frame: 20 weeks
description: The difference between EPX-100 vs placebo in proportion of participants with >50% reduction in the mean CCSF in the T+M periods relative to baseline.
measure: The total change in Seizure Severity using Hague Seizure Severity Scale (HASS).
time frame: 20 weeks
description: The total change in Seizure Severity using Hague Seizure Severity Scale (HASS).
measure: The improvement in Clinical Global Impression (CGI).
time frame: 20 weeks
description: The improvement in Clinical Global Impression (CGI).
measure: The total change in Quality of Life in Childhood Epilepsy short form (QOLCE 55).
time frame: 20 weeks
description: The total change in Quality of Life in Childhood Epilepsy short form (QOLCE 55).
measure: The difference between EPX-100 vs placebo in proportion of participants with >25% reduction in the mean CCSF in the T+M periods relative to baseline.
time frame: 20 weeks
description: The difference between EPX-100 vs placebo in proportion of participants with >25% reduction in the mean CCSF in the T+M periods relative to baseline.
measure: The mean difference between EPX-100 vs placebo per 28-days in the percent change of all seizures in the T+M periods relative to the baseline.
time frame: 20 weeks
description: The mean difference between EPX-100 vs placebo per 28-days in the percent change of all seizures in the T+M periods relative to the baseline.
measure: The change in the number of episodes of status epilepticus, in the T+M periods relative to the baseline.
time frame: 20 weeks
description: The change in the number of episodes of status epilepticus, in the T+M periods relative to the baseline.
measure: The incidence of rescue anti-epileptic drug (AED) use between EPX-100 and placebo, as measured by the number of days on rescue AEDs in the T+M periods relative to the baseline.
time frame: 20 weeks
description: The incidence of rescue anti-epileptic drug (AED) use between EPX-100 and placebo, as measured by the number of days on rescue AEDs in the T+M periods relative to the baseline.
measure: The individual item changes in Quality of Life in Childhood Epilepsy short form (QOLCE 55).
time frame: 20 weeks
description: The individual item changes in Quality of Life in Childhood Epilepsy short form (QOLCE 55).
measure: The individual item changes in Seizure Severity using Hague Seizure Severity Scale (HASS).
time frame: 20 weeks
description: The individual item changes in Seizure Severity using Hague Seizure Severity Scale (HASS).
measure: The change in the Sleep Disturbance Scale for Children (SDSC) between EPX-100 and placebo by each scheduled visit
time frame: 20 weeks
description: The change in the Sleep Disturbance Scale for Children (SDSC) between EPX-100 and placebo by each scheduled visit
arm group label: Active arm with EPX-100 (Clemizole HCl)
arm group type: Experimental
description: EPX-100 oral solution
arm group label: Placebo arm
arm group type: Placebo Comparator
description: Color- and taste-matched placebo oral solution dosed to match the active arm.
intervention type: Drug
intervention name: EPX-100 (Clemizole HCl)
description: Daily dose of EPX100
arm group label: Active arm with EPX-100 (Clemizole HCl)
other name: Clemizole Hydrochloride
other name: Clemizole HCl
other name: Clemizole
intervention type: Drug
intervention name: Placebo
description: Daily dose of Placebo
arm group label: Placebo arm
other name: Placebo to match EPX-100 solution
criteria: 
gender: All
minimum age: 2 Years
maximum age: N/A
healthy volunteers: No
last name: Lorianne Masouka, MD
role: Study Director
affiliation: Epygenix Therapeutics, Inc.
last name: Lorianne Masuoka, M.D.
phone: (415) 933-0826
email: lm@epygenix.com
last name: Rebekah DeVitry Fries
email: rfries@epygenix.com
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Terminated
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Not yet recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
country: Canada
country: Georgia
country: Hungary
country: Spain
country: United States
responsible party type: Sponsor
mesh term: Epilepsies, Myoclonic
mesh term: Syndrome
sharing ipd: No