<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/NCT03020797</url>
  </required_header>
  <id_info>
    <org_study_id>Eisai-01</org_study_id>
    <nct_id>NCT03020797</nct_id>
  </id_info>
  <brief_title>A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)</brief_title>
  <official_title>A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Stony Brook University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Eisai Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Stony Brook University</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The&#xD;
      investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved&#xD;
      by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel&#xD;
      will be used off-label for adults with ALS at an oral medication dose on the low end of the&#xD;
      recommended dose range for epilepsy. This study will consist of two treatments arms:&#xD;
      perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication&#xD;
      for 9 months.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    1) Enrolment below study target and 2) too many subjects had early termination due to disease&#xD;
    progression.&#xD;
  </why_stopped>
  <start_date type="Actual">January 12, 2017</start_date>
  <completion_date type="Actual">February 8, 2023</completion_date>
  <primary_completion_date type="Actual">January 31, 2023</primary_completion_date>
  <phase>N/A</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>Incidence of treatment-emergent adverse events</measure>
    <time_frame>9 months</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);</measure>
    <time_frame>9 months</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">12</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>perampanel</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks</description>
  </arm_group>
  <arm_group>
    <arm_group_label>placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>placebo 1 tablet QD for 2 weeks placebo 2 tablets QD for 2 weeks placebo 3 tablets QD for 2 weeks placebo 4 tablets QD for 2 weeks</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Perampanel</intervention_name>
    <arm_group_label>perampanel</arm_group_label>
    <other_name>Fycompa</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo Oral Tablet</intervention_name>
    <arm_group_label>placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. diagnosis of ALS&#xD;
&#xD;
          2. first clinical weakness within past 3 years&#xD;
&#xD;
          3. slow vital capacity &gt;= 60% of predicted within 1 month of treatment&#xD;
&#xD;
          4. may be on stable dose of riluzole for at least 30 days, or otherwise agree to not&#xD;
             initiate riluzole for duration of the trial&#xD;
&#xD;
          5. may be on stable dose of edaravone for at least 30 days, otherwise agree to not&#xD;
             initiate edaravone for duration of the trial&#xD;
&#xD;
          6. can travel to Stony Brook to receive medical care&#xD;
&#xD;
          7. must have a monitor who can be contacted at regular intervals to report on subject's&#xD;
             clinical/psychiatric status&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. use of tracheostomy or mechanical ventilation within last 3 months&#xD;
&#xD;
          2. hepatic insufficiency or abnormal liver function&#xD;
&#xD;
          3. renal insufficiency&#xD;
&#xD;
          4. clinically significant psychiatric disorder&#xD;
&#xD;
          5. active malignancy&#xD;
&#xD;
          6. history of HIV, clinically significant chronic hepatitis, or other active infection&#xD;
&#xD;
          7. history of stomach or intestinal surgery or condition that could interfere with&#xD;
             absorption, distribution, metabolism or secretion of study drug&#xD;
&#xD;
          8. history of alcohol or substance abuse within 3 months prior to entry (subjects will be&#xD;
             instructed to refrain from alcohol during the study)&#xD;
&#xD;
          9. use of strong cytochrome P4503A inhibitors or inducers, anticonvulsants or other drugs&#xD;
             known to interact strongly with perampanel.&#xD;
&#xD;
         10. pregnancy or lactation&#xD;
&#xD;
         11. clinically significant medical condition (other than ALS) that would pose a risk to&#xD;
             the subject if they were to participate&#xD;
&#xD;
         12. know hypersensitivity to perampanel&#xD;
&#xD;
         13. currently participating, or has participated in a study with an investigation or&#xD;
             marketed compound within 3 months of entry&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Nurcan Gursoy, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Stony Brook Medicine Dept. of Neurology</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Stony Brook University Medical Center</name>
      <address>
        <city>Stony Brook</city>
        <state>New York</state>
        <zip>11794-8121</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>December 15, 2016</study_first_submitted>
  <study_first_submitted_qc>January 11, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 13, 2017</study_first_posted>
  <last_update_submitted>May 3, 2023</last_update_submitted>
  <last_update_submitted_qc>May 3, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 6, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Stony Brook University</investigator_affiliation>
    <investigator_full_name>Nurcan Gursoy</investigator_full_name>
    <investigator_title>Clinical Assistant Professor</investigator_title>
  </responsible_party>
  <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>
    <mesh_term>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/NCT03020797
org study id: Eisai-01
nct id: NCT03020797
lead sponsor: 
collaborator: 
has dmc: Yes
This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The
      investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved
      by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel
      will be used off-label for adults with ALS at an oral medication dose on the low end of the
      recommended dose range for epilepsy. This study will consist of two treatments arms:
      perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication
      for 9 months.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Incidence of treatment-emergent adverse events
time frame: 9 months
measure: Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);
time frame: 9 months
arm group label: perampanel
arm group type: Experimental
description: perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks
arm group label: placebo
arm group type: Placebo Comparator
description: placebo 1 tablet QD for 2 weeks placebo 2 tablets QD for 2 weeks placebo 3 tablets QD for 2 weeks placebo 4 tablets QD for 2 weeks
intervention type: Drug
intervention name: Perampanel
arm group label: perampanel
other name: Fycompa
intervention type: Drug
intervention name: Placebo Oral Tablet
arm group label: placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: No
last name: Nurcan Gursoy, MD
role: Principal Investigator
affiliation: Stony Brook Medicine Dept. of Neurology
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Stony Brook University
investigator full name: Nurcan Gursoy
investigator title: Clinical Assistant Professor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis
sharing ipd: No