<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/NCT03019419</url>
  </required_header>
  <id_info>
    <org_study_id>A2016-J000-001</org_study_id>
    <nct_id>NCT03019419</nct_id>
  </id_info>
  <brief_title>Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)</brief_title>
  <official_title>Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS): A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 2 Trials</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Tokyo Medical University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Tokyo Medical University</source>
  <oversight_info>
    <has_dmc>Yes</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>
      To investigate the safety and the efficacy of perampanel in patients with sporadic&#xD;
      amyotrophic lateral sclerosis&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      To evaluate the effect of peramanel for 48 weeks on progression of disease in subjects with&#xD;
      ALS, as measured by ALS Functional Rating Scale-Revised (ALSFRS-R)&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 24, 2017</start_date>
  <completion_date type="Actual">December 23, 2020</completion_date>
  <primary_completion_date type="Actual">December 23, 2020</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>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in ALS Functional rating scale</measure>
    <time_frame>48 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in ALS Functional rating scale</measure>
    <time_frame>12, 24, 36, and 48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Manual Muscle Test</measure>
    <time_frame>12, 24, 36, and 48 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percent-predicted forced vital capacity</measure>
    <time_frame>12, 24, 36, and 48 weeks</time_frame>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">66</enrollment>
  <condition>ALS</condition>
  <arm_group>
    <arm_group_label>Perampanel 4mg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Once daily 4mg of perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Perampanel 8mg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Once daily 8mg of perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Once daily placebo for control for 48 weeks</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Perampanel</intervention_name>
    <description>4mg/d or 8mg/d</description>
    <arm_group_label>Perampanel 4mg</arm_group_label>
    <arm_group_label>Perampanel 8mg</arm_group_label>
    <other_name>non-competitive AMAP antagonist</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>placebo</intervention_name>
    <description>placebo</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        [Eligibility Criteria for Interim Registration]&#xD;
&#xD;
          -  Patients who are able to submit written informed consent. If patients are duly capable&#xD;
             of study consent but are unable to sign by themselves due to aggravation of disease&#xD;
             condition, written informed consent can be obtained from a legally authorized&#xD;
             representative who can sign on behalf of the patients after confirming the patients'&#xD;
             agreement to study participation.&#xD;
&#xD;
          -  Patients who are male or female aged 40 years to 78 years at the time of obtaining&#xD;
             informed consent&#xD;
&#xD;
          -  Patients who have clinically definite ALS, clinically probable ALS, or clinically&#xD;
             probable-laboratory supported ALS as specified in the revised El Escorial Airlie House&#xD;
             diagnostic criteria.&#xD;
&#xD;
          -  The sum of the 3 respiratory items of the ALSFRS-R must total 12 points or more&#xD;
&#xD;
          -  Patients within 2-year elapsed time period from disease onset at the time of obtaining&#xD;
             informed consent&#xD;
&#xD;
          -  Patients who can visit study site for out-patient treatment&#xD;
&#xD;
        [Eligibility Criteria for Registration]&#xD;
&#xD;
        Subjects who meet the following criteria in addition to the inclusion criteria for the&#xD;
        interim registration&#xD;
&#xD;
          -  The progression on score of ALSFRS-R during 12 weeks of observation period must be&#xD;
             between -2 and -5&#xD;
&#xD;
          -  Patients who has not initiated newly introduced riluzole therapy after starting the&#xD;
             observation period. Or those who has not received dose escalation or resumed&#xD;
             administration of riluzole therapy after previous down titration or discontinuation&#xD;
&#xD;
          -  Patients who has not initiated newly introduced edaravone therapy after starting the&#xD;
             observation period&#xD;
&#xD;
          -  Patients who are judged to be eligible for continuation of the study by the&#xD;
             investigators&#xD;
&#xD;
        [Exclusion Criteria]&#xD;
&#xD;
          -  Patients who underwent tracheostomy.&#xD;
&#xD;
          -  Patients who experienced non-invasive positive pressure ventilation.&#xD;
&#xD;
          -  Patients whose percent-predicted forced vital capacity (%FVC) is ≤80%.&#xD;
&#xD;
          -  Patients with progressive bulbar palsy type.&#xD;
&#xD;
          -  Patients with cognitive impairment, severe disease in the renal, cardiovascular, or&#xD;
             hematological system.&#xD;
&#xD;
          -  Patients with hepatic disease.&#xD;
&#xD;
          -  Patients with malignant tumor.&#xD;
&#xD;
          -  Pregnant women or women with a possibility of becoming pregnant.&#xD;
&#xD;
          -  Patients who participated in another clinical study within 12 weeks before starting&#xD;
             the observation period.&#xD;
&#xD;
          -  Patients who has initiated perampanel therapy in the past or at present.&#xD;
&#xD;
          -  Patients who are judged to be ineligible for study entry by the investigators.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>40 Years</minimum_age>
    <maximum_age>78 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Tomohiro Haga</last_name>
    <role>Study Director</role>
    <affiliation>The University of Tokyo Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Kitasato University East Hospital</name>
      <address>
        <city>Kanagawa</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kumamoto Saishunso National Hospital</name>
      <address>
        <city>Kumamoto</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nagoya University Hospital</name>
      <address>
        <city>Nagoya</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Okayama University Hospital</name>
      <address>
        <city>Okayama</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hokkaido University Hospital</name>
      <address>
        <city>Sapporo</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tohoku University Hospital</name>
      <address>
        <city>Sendai</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shiga University of Medical Science Hospital</name>
      <address>
        <city>Shiga</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tokyo Medical University</name>
      <address>
        <city>Tokyo</city>
        <zip>160-0023</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Tokyo Hospital</name>
      <address>
        <city>Tokyo</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tokyo Metropolitan Neurological Hospital</name>
      <address>
        <city>Tokyo</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Tsukuba Hospital</name>
      <address>
        <city>Tsukuba</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Yamaguchi University Hospital</name>
      <address>
        <city>Yamaguchi</city>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Japan</country>
  </location_countries>
  <verification_date>January 2021</verification_date>
  <study_first_submitted>January 10, 2017</study_first_submitted>
  <study_first_submitted_qc>January 10, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 12, 2017</study_first_posted>
  <last_update_submitted>January 14, 2021</last_update_submitted>
  <last_update_submitted_qc>January 14, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">January 15, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>sporadic ALS</keyword>
  <keyword>perampanel</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Motor Neuron Disease</mesh_term>
    <mesh_term>Amyotrophic Lateral Sclerosis</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/NCT03019419
org study id: A2016-J000-001
nct id: NCT03019419
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
To investigate the safety and the efficacy of perampanel in patients with sporadic
      amyotrophic lateral sclerosis
To evaluate the effect of peramanel for 48 weeks on progression of disease in subjects with
      ALS, as measured by ALS Functional Rating Scale-Revised (ALSFRS-R)
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Change in ALS Functional rating scale
time frame: 48 weeks
measure: Change in ALS Functional rating scale
time frame: 12, 24, 36, and 48 weeks
measure: Manual Muscle Test
time frame: 12, 24, 36, and 48 weeks
measure: Percent-predicted forced vital capacity
time frame: 12, 24, 36, and 48 weeks
arm group label: Perampanel 4mg
arm group type: Experimental
description: Once daily 4mg of perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks
arm group label: Perampanel 8mg
arm group type: Experimental
description: Once daily 8mg of perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks
arm group label: Placebo
arm group type: Placebo Comparator
description: Once daily placebo for control for 48 weeks
intervention type: Drug
intervention name: Perampanel
description: 4mg/d or 8mg/d
arm group label: Perampanel 4mg
arm group label: Perampanel 8mg
other name: non-competitive AMAP antagonist
intervention type: Drug
intervention name: placebo
description: placebo
arm group label: Placebo
criteria: 
gender: All
minimum age: 40 Years
maximum age: 78 Years
healthy volunteers: No
last name: Tomohiro Haga
role: Study Director
affiliation: The University of Tokyo Hospital
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Japan
responsible party type: Sponsor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
sharing ipd: No