<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/NCT03239691</url>
  </required_header>
  <id_info>
    <org_study_id>AC-083-103</org_study_id>
    <nct_id>NCT03239691</nct_id>
  </id_info>
  <brief_title>A Study to Evaluate the Effect of ACT-709478 in Photosensitive Epilepsy Patients</brief_title>
  <official_title>A Phase 2a, Multi-center, Single-blind, Within-subject, Placebo-controlled Study to Assess the Pharmacodynamics of ACT-709478 in Subjects With Photosensitive Epilepsy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Idorsia Pharmaceuticals Ltd.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Idorsia Pharmaceuticals Ltd.</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>
      The main study objective is to assess the effect of ACT-709478 in male and female subjects&#xD;
      with photosensitive epilepsy following single dose administration&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 6, 2017</start_date>
  <completion_date type="Actual">April 25, 2018</completion_date>
  <primary_completion_date type="Actual">April 25, 2018</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Other</primary_purpose>
    <masking>Single (Participant)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Individual evaluation of the response to intermittent photic stimulation</measure>
    <time_frame>From Day 2 to Day 10</time_frame>
    <description>Positive response described as complete suppression of photoparoxysmal response (PPR) or a clinically relevant reduction in the standardized photosensitive range (SPR)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Time to onset of positive response</measure>
    <time_frame>From Day 2 to Day 10</time_frame>
    <description>Defined by the first time point after ACT-709478 administration at which complete suppression of PPR or clinically relevant reduction in SPR is achieved</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Duration of positive response</measure>
    <time_frame>From Day 2 to Day 10</time_frame>
    <description>Defined as the time elapsed between the time point of onset of the positive response and the last time point of the positive response after ACT-709478 administration</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Maximum SPR reduction</measure>
    <time_frame>From Day 2 to Day 10</time_frame>
    <description>Defined as the largest reduction in SPR achieved at any time point compared to baseline during the positive response after ACT-709478 administration</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to maximum SPR reduction</measure>
    <time_frame>From Day 2 to Day 10</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">5</enrollment>
  <condition>Photosensitive Epilepsy</condition>
  <arm_group>
    <arm_group_label>ACT-709478 - Single dose administration</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Up to 16 subjects with photosensitive epilepsy will be studied across a maximum of 4 dose levels. Each dose level will initially be investigated in cohorts of 4 subjects undergoing a fixed sequence of study treatment administration in fed condition</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo will be administered on two study days</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ACT-709478 for oral use</intervention_name>
    <description>Hard gelatin capsules for oral administration formulated at strengths of 10 mg and 100 mg</description>
    <arm_group_label>ACT-709478 - Single dose administration</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Matching placebo available as matching capsules for oral administration</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Signed informed consent in the local language prior to any study-mandated procedure&#xD;
&#xD;
          -  Male and female subjects aged between 18 and 60 years (inclusive) at screening&#xD;
&#xD;
          -  Photosensitive epilepsy and a generalized PPR in response to IPS of at least 4 units&#xD;
             on a SPR in at least 1 condition (eye closure, eyes closed, or eyes open) on 2&#xD;
             occasions at screening with at least 1 hour interval and reproducible on Day -1 (less&#xD;
             than 3 units difference in SPR between screening and Day -1)&#xD;
&#xD;
          -  Healthy on the basis of physical examination, cardiovascular assessments and&#xD;
             laboratory tests&#xD;
&#xD;
          -  Women of childbearing potential must have a negative serum pregnancy test at screening&#xD;
             and a negative urine pregnancy test on Day -1. They must consistently and correctly&#xD;
             use a reliable method of contraception with a failure rate of &lt;1% per year, be&#xD;
             sexually inactive, or have a vasectomized partner. Hormonal contraceptives must be&#xD;
             initiated at least 1 month before first study treatment administration&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Lactating women&#xD;
&#xD;
          -  Known hypersensitivity to any of the excipients of the study treatment formulation&#xD;
&#xD;
          -  History or clinical evidence of any disease other than epilepsy and/or existence of&#xD;
             any surgical or medical condition, which might interfere with the absorption,&#xD;
             distribution, metabolism, or excretion of the study treatment (appendectomy and&#xD;
             herniotomy allowed, cholecystectomy not allowed)&#xD;
&#xD;
          -  Relevant history of a suicide attempt or suicidal behavior. Any recent suicidal&#xD;
             ideation within the last 6 months (categories 4 or 5), or any suicidal behavior within&#xD;
             the last 2 years, except for &quot;Non-Suicidal Self-Injurious Behavior&quot; (item also&#xD;
             included in the Suicidal Behavior section), as judged by the investigator using the&#xD;
             Columbia-Suicide Severity Rating Scale (C-SSRS) at screening&#xD;
&#xD;
          -  History of status epilepticus during the last 12 months&#xD;
&#xD;
          -  History of non-epileptic seizures that cannot be differentiated from the participant's&#xD;
             epileptic seizures&#xD;
&#xD;
          -  History of generalized tonic-clonic seizures triggered by IPS&#xD;
&#xD;
          -  Previous history of repeated fainting, syncope, orthostatic hypotension, or vasovagal&#xD;
             reactions in the past 5 years&#xD;
&#xD;
          -  Any circumstances or conditions, which, in the opinion of the investigator, may affect&#xD;
             full participation in the study or compliance with the protocol&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Clinical Trials</last_name>
    <role>Study Director</role>
    <affiliation>Idorsia Pharmaceuticals Ltd.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>HOSP - Bicêtre Neurologie</name>
      <address>
        <city>Le Kremlin-Bicêtre</city>
        <zip>94275</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Bethel Epilepsy Center, Mara Hospital</name>
      <address>
        <city>Bielefeld</city>
        <zip>33617</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Epilepsy Center Frankfurt</name>
      <address>
        <city>Frankfurt</city>
        <zip>60528</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Epilepsiezentrum Kork</name>
      <address>
        <city>Kehl</city>
        <zip>77694</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kleinwachau, Sächsisches Epilepsiezentrum Radeberg</name>
      <address>
        <city>Radeberg</city>
        <zip>01454</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Germany</country>
  </location_countries>
  <verification_date>August 2018</verification_date>
  <study_first_submitted>August 2, 2017</study_first_submitted>
  <study_first_submitted_qc>August 2, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 4, 2017</study_first_posted>
  <last_update_submitted>August 30, 2018</last_update_submitted>
  <last_update_submitted_qc>August 30, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">August 31, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Epilepsy</mesh_term>
    <mesh_term>Epilepsy, Reflex</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>ACT-709478</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/NCT03239691
org study id: AC-083-103
nct id: NCT03239691
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The main study objective is to assess the effect of ACT-709478 in male and female subjects
      with photosensitive epilepsy following single dose administration
allocation: Non-Randomized
intervention model: Single Group Assignment
primary purpose: Other
masking: Single (Participant)
measure: Individual evaluation of the response to intermittent photic stimulation
time frame: From Day 2 to Day 10
description: Positive response described as complete suppression of photoparoxysmal response (PPR) or a clinically relevant reduction in the standardized photosensitive range (SPR)
measure: Time to onset of positive response
time frame: From Day 2 to Day 10
description: Defined by the first time point after ACT-709478 administration at which complete suppression of PPR or clinically relevant reduction in SPR is achieved
measure: Duration of positive response
time frame: From Day 2 to Day 10
description: Defined as the time elapsed between the time point of onset of the positive response and the last time point of the positive response after ACT-709478 administration
measure: Maximum SPR reduction
time frame: From Day 2 to Day 10
description: Defined as the largest reduction in SPR achieved at any time point compared to baseline during the positive response after ACT-709478 administration
measure: Time to maximum SPR reduction
time frame: From Day 2 to Day 10
arm group label: ACT-709478 - Single dose administration
arm group type: Experimental
description: Up to 16 subjects with photosensitive epilepsy will be studied across a maximum of 4 dose levels. Each dose level will initially be investigated in cohorts of 4 subjects undergoing a fixed sequence of study treatment administration in fed condition
arm group label: Placebo
arm group type: Placebo Comparator
description: Placebo will be administered on two study days
intervention type: Drug
intervention name: ACT-709478 for oral use
description: Hard gelatin capsules for oral administration formulated at strengths of 10 mg and 100 mg
arm group label: ACT-709478 - Single dose administration
intervention type: Drug
intervention name: Placebo
description: Matching placebo available as matching capsules for oral administration
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: 60 Years
healthy volunteers: No
last name: Clinical Trials
role: Study Director
affiliation: Idorsia Pharmaceuticals Ltd.
facility: 
facility: 
facility: 
facility: 
facility: 
country: France
country: Germany
responsible party type: Sponsor
mesh term: Epilepsy
mesh term: Epilepsy, Reflex
mesh term: ACT-709478