<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/NCT01684761</url>
  </required_header>
  <id_info>
    <org_study_id>Protocol Number 2012-00</org_study_id>
    <nct_id>NCT01684761</nct_id>
  </id_info>
  <brief_title>Study of Tcelna (Imilecleucel-T) in Secondary Progressive Multiple Sclerosis</brief_title>
  <acronym>Abili-T</acronym>
  <official_title>A Phase 2 Double-Blind, Placebo Controlled Multi-Center Study to Evaluate the Efficacy and Safety of Tcelna in Subjects With Secondary Progressive Multiple Sclerosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Opexa Therapeutics, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Opexa Therapeutics, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to determine whether Tcelna (imilecleucel-T, autologous T-Cell&#xD;
      Immunotherapy) is effective in the treatment of secondary progressive multiple sclerosis&#xD;
      (SPMS).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Subjects whose myelin reactive T-cell can be identified by EPA will are randomized and&#xD;
      provide blood to manufacture Tcelna. Approximately 5 weeks after receipt of the subject's&#xD;
      whole blood procurement, the subjects will receive either Tcelna or placebo and will complete&#xD;
      baseline assessments and will receive study treatments at Weeks 0, 4, 8, 12, and 24 (Visits&#xD;
      3-7), totaling 5 doses in year one.&#xD;
&#xD;
      Approximately one month prior to the Week 52 visit a second blood procurement will be&#xD;
      performed and the subject will receive the second series of treatments as received in the&#xD;
      first year study schedule. Subjects will be evaluated for changes in disability and cognitive&#xD;
      function every 3 months, and radiographic changes annually.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>August 2012</start_date>
  <completion_date type="Actual">October 2016</completion_date>
  <primary_completion_date type="Actual">October 2016</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>Brain Atrophy</measure>
    <time_frame>2 Years</time_frame>
    <description>The percentage of brain volume change (atrophy) as measured on 24 month MRIs calculated by the central MRI facility.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Disease Progression</measure>
    <time_frame>2 Years</time_frame>
    <description>The percentage of subjects with sustained progression with definitions of sustained effect at 3 months and 6 months.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">183</enrollment>
  <condition>Autoimmune Diseases of the Nervous System</condition>
  <condition>Multiple Sclerosis</condition>
  <condition>Secondary Progressive Multiple Sclerosis</condition>
  <condition>Disease Progression</condition>
  <condition>Brain Atrophy</condition>
  <arm_group>
    <arm_group_label>Tcelna</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>30-45 x 10E6 total cells in 2 ml. Subjects receive two annual courses of 5 subcutaneous doses each year (at 0, 4, 8, 12 and 24 weeks).</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Tcelna inactive ingredients (without cells) totaling 2 ml per dose. Administered subcutaneously with same two year treatment regimen as experimental treatment arm.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Tcelna</intervention_name>
    <description>Autologous pool of myelin reactive T-cells (MRTC) expanded ex vivo with immunodominant epitopes selected from the three myelin antigens, MBP, PLP and MOG on a per subject basis. Attenuated by irradiation to prevent further proliferation before releasing product for administration.</description>
    <arm_group_label>Tcelna</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>2 ml of Tcelna excipients, prepared daily as individual doses and irradiated before releasing product for administration.</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosed with MS as defined by the modified McDonald criteria&#xD;
&#xD;
          -  SPMS defined as relapsing-remitting disease with recent progression in MS-related&#xD;
             neurological deficits&#xD;
&#xD;
          -  EDSS score 3.0 - 6.0, inclusively&#xD;
&#xD;
          -  Presence of myelin reactive T-cells&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosed with primary progressive MS&#xD;
&#xD;
          -  Treatment with beta-interferon, glatiramer acetate or dimethyl fumarate 30 days prior&#xD;
             to screening&#xD;
&#xD;
          -  Treatment with ACTH, any over-the-counter or prescription corticosteroids 60 days&#xD;
             prior to screening&#xD;
&#xD;
          -  Treatment with IVIG, plasmapheresis or cytopheresis 90 days prior to screening&#xD;
&#xD;
          -  Treatment with mitoxantrone, teriflunomide, fingolimod, natalizumab, azathioprine,&#xD;
             cyclosporine, methotrexate or mycophenolate mofetil 1 year prior to baseline&#xD;
&#xD;
          -  Any prior treatment with cladribine, cyclophosphamide, total lymphoid irradiation, T&#xD;
             cell or T cell receptor products, or any therapeutic monoclonal antibody, except&#xD;
             natalizumab&#xD;
&#xD;
          -  Previous treatment with any other MS investigational drug 1 year prior to screening&#xD;
&#xD;
          -  All non-MS investigational drugs must have a minimum washout of 30 days prior to&#xD;
             screening or 5 half-lives, whatever is the longest period of time.&#xD;
&#xD;
          -  HIV or hepatitis infection&#xD;
&#xD;
          -  History of cancer&#xD;
&#xD;
          -  Any other significant medical condition that, in the opinion of the investigator,&#xD;
             could cause CNS tissue damage or limit its repair.&#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>Jessica Jackson</last_name>
    <role>Study Director</role>
    <affiliation>Opexa Therapeutics, Inc.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>HOPE Research Institute</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85050</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Northwest NeuroSpecialists, LLC</name>
      <address>
        <city>Tucson</city>
        <state>Arizona</state>
        <zip>85741</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Alta Bates Summit Medical Center, The Research and Education Development Institute</name>
      <address>
        <city>Berkeley</city>
        <state>California</state>
        <zip>94705</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurology Associates, P.A.</name>
      <address>
        <city>Maitland</city>
        <state>Florida</state>
        <zip>32751</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Miami</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33136</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Collier Neurologic Specialists, LLC</name>
      <address>
        <city>Naples</city>
        <state>Florida</state>
        <zip>34102</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurological Services of Orlando</name>
      <address>
        <city>Orlando</city>
        <state>Florida</state>
        <zip>32806</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Meridien Research</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33606</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vero Beach Neurology</name>
      <address>
        <city>Vero Beach</city>
        <state>Florida</state>
        <zip>32960</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shepherd Center</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30309</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Consultants In Neurology, Ltd.</name>
      <address>
        <city>Northbrook</city>
        <state>Illinois</state>
        <zip>60062</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fort Wayne Neurological Center</name>
      <address>
        <city>Fort Wayne</city>
        <state>Indiana</state>
        <zip>46804</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Josephson Wallack Munshower Neurology, PC</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46256</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Kansas Medical Center</name>
      <address>
        <city>Kansas City</city>
        <state>Kansas</state>
        <zip>66160</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Associates in Neurology</name>
      <address>
        <city>Lexington</city>
        <state>Kentucky</state>
        <zip>40503</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Elizabeth's Medical Center</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02135</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Island Neurological Assoicates, PC</name>
      <address>
        <city>Plainview</city>
        <state>New York</state>
        <zip>11803</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital and Medical Center Stony Brook New York</name>
      <address>
        <city>Stony Brook</city>
        <state>New York</state>
        <zip>11794-8121</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Neurological Institute, PA</name>
      <address>
        <city>Charlotte</city>
        <state>North Carolina</state>
        <zip>28204</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Carolinas Medical Center Neurology</name>
      <address>
        <city>Charlotte</city>
        <state>North Carolina</state>
        <zip>28207</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>PMG Research of Charlotte</name>
      <address>
        <city>Charlotte</city>
        <state>North Carolina</state>
        <zip>28209</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurology Specialists, Inc</name>
      <address>
        <city>Dayton</city>
        <state>Ohio</state>
        <zip>45408</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Providence Medical Group - Medford</name>
      <address>
        <city>Medford</city>
        <state>Oregon</state>
        <zip>97504</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Providence St. Vincent Medical Center - Northwest MS Center</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97225</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Pennsylvania</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Maxine Mesinger MS Clinic/Baylor College of Medicine</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Central Texas Neurology</name>
      <address>
        <city>Round Rock</city>
        <state>Texas</state>
        <zip>78681</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Integra Clinical Research, LLC</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fletcher Allen Health Care - Neurology Service</name>
      <address>
        <city>Burlington</city>
        <state>Vermont</state>
        <zip>05401</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hampton Roads Neurology</name>
      <address>
        <city>Newport News</city>
        <state>Virginia</state>
        <zip>23601</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurological Associates, Inc</name>
      <address>
        <city>Richmond</city>
        <state>Virginia</state>
        <zip>23226</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Swedish Neuroscience Institute</name>
      <address>
        <city>Issaquah</city>
        <state>Washington</state>
        <zip>98029</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Swedish Neuroscience Institute</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98122</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Ottawa</name>
      <address>
        <city>Ottawa</city>
        <state>Ontario</state>
        <zip>K1H 8L6</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Recherche Sepmus Inc.</name>
      <address>
        <city>Greenfield Park</city>
        <state>Quebec</state>
        <zip>J4V 2J2</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Montreal Neurological Institute and Hospital</name>
      <address>
        <city>Montreal</city>
        <state>Quebec</state>
        <zip>H3A 2B4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.nationalmssociety.org</url>
    <description>Click here for more information about MS and current research.</description>
  </link>
  <verification_date>January 2017</verification_date>
  <study_first_submitted>September 11, 2012</study_first_submitted>
  <study_first_submitted_qc>September 12, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 13, 2012</study_first_posted>
  <last_update_submitted>January 9, 2017</last_update_submitted>
  <last_update_submitted_qc>January 9, 2017</last_update_submitted_qc>
  <last_update_posted type="Estimate">January 10, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Multicenter Study</keyword>
  <keyword>Randomized Controlled Trial</keyword>
  <keyword>Individualized Medicine</keyword>
  <keyword>Immunotherapy</keyword>
  <keyword>Myelin Proteins</keyword>
  <keyword>Biological Agents</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neoplasm Metastasis</mesh_term>
    <mesh_term>Multiple Sclerosis</mesh_term>
    <mesh_term>Multiple Sclerosis, Chronic Progressive</mesh_term>
    <mesh_term>Nervous System Diseases</mesh_term>
    <mesh_term>Autoimmune Diseases of the Nervous System</mesh_term>
    <mesh_term>Autoimmune Diseases</mesh_term>
    <mesh_term>Sclerosis</mesh_term>
    <mesh_term>Disease Progression</mesh_term>
  </condition_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/NCT01684761
org study id: Protocol Number 2012-00
nct id: NCT01684761
lead sponsor: 
has dmc: Yes
The purpose of this study is to determine whether Tcelna (imilecleucel-T, autologous T-Cell
      Immunotherapy) is effective in the treatment of secondary progressive multiple sclerosis
      (SPMS).
Subjects whose myelin reactive T-cell can be identified by EPA will are randomized and
      provide blood to manufacture Tcelna. Approximately 5 weeks after receipt of the subject's
      whole blood procurement, the subjects will receive either Tcelna or placebo and will complete
      baseline assessments and will receive study treatments at Weeks 0, 4, 8, 12, and 24 (Visits
      3-7), totaling 5 doses in year one.

      Approximately one month prior to the Week 52 visit a second blood procurement will be
      performed and the subject will receive the second series of treatments as received in the
      first year study schedule. Subjects will be evaluated for changes in disability and cognitive
      function every 3 months, and radiographic changes annually.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Brain Atrophy
time frame: 2 Years
description: The percentage of brain volume change (atrophy) as measured on 24 month MRIs calculated by the central MRI facility.
measure: Disease Progression
time frame: 2 Years
description: The percentage of subjects with sustained progression with definitions of sustained effect at 3 months and 6 months.
arm group label: Tcelna
arm group type: Experimental
description: 30-45 x 10E6 total cells in 2 ml. Subjects receive two annual courses of 5 subcutaneous doses each year (at 0, 4, 8, 12 and 24 weeks).
arm group label: Placebo
arm group type: Placebo Comparator
description: Tcelna inactive ingredients (without cells) totaling 2 ml per dose. Administered subcutaneously with same two year treatment regimen as experimental treatment arm.
intervention type: Biological
intervention name: Tcelna
description: Autologous pool of myelin reactive T-cells (MRTC) expanded ex vivo with immunodominant epitopes selected from the three myelin antigens, MBP, PLP and MOG on a per subject basis. Attenuated by irradiation to prevent further proliferation before releasing product for administration.
arm group label: Tcelna
intervention type: Biological
intervention name: Placebo
description: 2 ml of Tcelna excipients, prepared daily as individual doses and irradiated before releasing product for administration.
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: 60 Years
healthy volunteers: No
last name: Jessica Jackson
role: Study Director
affiliation: Opexa Therapeutics, Inc.
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country: Canada
country: United States
url: http://www.nationalmssociety.org
description: Click here for more information about MS and current research.
responsible party type: Sponsor
mesh term: Neoplasm Metastasis
mesh term: Multiple Sclerosis
mesh term: Multiple Sclerosis, Chronic Progressive
mesh term: Nervous System Diseases
mesh term: Autoimmune Diseases of the Nervous System
mesh term: Autoimmune Diseases
mesh term: Sclerosis
mesh term: Disease Progression