<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/NCT02199925</url>
  </required_header>
  <id_info>
    <org_study_id>GMXAUT01</org_study_id>
    <nct_id>NCT02199925</nct_id>
  </id_info>
  <brief_title>An Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum</brief_title>
  <official_title>A Phase IV, Single-site, Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Isaac Melamed</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Bio Products Laboratory</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>IMMUNOe Research Centers</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      If autism is the consequence of a chronic inflammatory process preventing the infant brain&#xD;
      from forming the proper neural connections, then treatment that reduces inflammation might&#xD;
      have a positive impact on autism. IGIV treatment has been known to suppress inflammation and&#xD;
      has been used in the treatment of inflammatory conditions and autoimmune diseases.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      We have come to believe that an immature immune system with abnormal signaling and altered&#xD;
      apoptotic pathways may result in a process of neuroinflammation with a clinical presentation&#xD;
      of Autism Spectrum Disorder. If Autism Spectrum Disorders are the consequence of a chronic&#xD;
      inflammatory process that prevents the infant brain from forming the proper neural&#xD;
      connections, then treatment that reduces inflammation might have a positive impact on autism.&#xD;
      We believe that there is a correlation between immunological abnormalities and the&#xD;
      development of the autism.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date>June 2013</start_date>
  <completion_date type="Anticipated">July 2015</completion_date>
  <primary_completion_date type="Anticipated">April 2015</primary_completion_date>
  <phase>Phase 4</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction.</measure>
    <time_frame>1 year</time_frame>
    <description>The primary endpoint is disease improvement, as evidenced by standardized test measures across 3 intervals: screening for baseline measurements, visit 5, and end of study visit measurements. Primary efficacy variables will be measured using the following standardized tests&#xD;
Caregiver Scales (SRS, CCC-2, ABC)&#xD;
CGI-S and CGI - I&#xD;
PPVT&#xD;
ADOS</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The modulatory effect of IVIG on neuroinflammation will be assessed by laboratory measurements which we believe are indicative of immune dysregulation.</measure>
    <time_frame>1 year</time_frame>
  </secondary_outcome>
  <other_outcome>
    <measure>To improve development in autistic spectrum</measure>
    <time_frame>1 year</time_frame>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">30</enrollment>
  <condition>Autism</condition>
  <condition>Autistic Disorder</condition>
  <condition>Asperger's Disorder</condition>
  <condition>PDD</condition>
  <condition>Pervasive Developmental Disorder</condition>
  <arm_group>
    <arm_group_label>Gammaplex 5% IGIV</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Gammaplex 5% IGIV administered intravenously</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Gammaplex 5%</intervention_name>
    <arm_group_label>Gammaplex 5% IGIV</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  The subject is between the ages of or equal to 4 and 12 years of age, of either&#xD;
             gender, and belonging to any ethnic group.&#xD;
&#xD;
          -  The subject has a diagnosis, for a minimum of 6 months prior to screening, of either&#xD;
             autistic disorder, Asperger disorder, or pervasive developmental disorder (PDD).&#xD;
&#xD;
          -  Normal physical test results.&#xD;
&#xD;
          -  Immunological lab results showing reduced levels of activated CD40L or abnormal&#xD;
             lymphocyte stimulation, or T or B cell dysfunction (hypgammaglobulinemia, primary&#xD;
             immunodeficiency (PIDD), or common variable immune deficiency (CVID).&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  A diagnosis of isolated IGA deficiency&#xD;
&#xD;
          -  Allergic reactions to blood products&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>4 Years</minimum_age>
    <maximum_age>12 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Isaac Melamed, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>IMMUNOe International Clinical Research Centers</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>IMMUNOe International Research Centers</name>
      <address>
        <city>Centennial</city>
        <state>Colorado</state>
        <zip>80112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2015</verification_date>
  <study_first_submitted>July 21, 2014</study_first_submitted>
  <study_first_submitted_qc>July 23, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 25, 2014</study_first_posted>
  <last_update_submitted>April 15, 2015</last_update_submitted>
  <last_update_submitted_qc>April 15, 2015</last_update_submitted_qc>
  <last_update_posted type="Estimate">April 16, 2015</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>IMMUNOe Research Centers</investigator_affiliation>
    <investigator_full_name>Isaac Melamed</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <keyword>Autism</keyword>
  <keyword>Autism spectrum</keyword>
  <keyword>Autism children</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Autistic Disorder</mesh_term>
    <mesh_term>Developmental Disabilities</mesh_term>
    <mesh_term>Autism Spectrum Disorder</mesh_term>
    <mesh_term>Asperger Syndrome</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>gamma-Globulins</mesh_term>
    <mesh_term>Immunoglobulins, Intravenous</mesh_term>
    <mesh_term>Rho(D) Immune Globulin</mesh_term>
  </intervention_browse>
  <pending_results>
    <submitted>May 7, 2018</submitted>
    <returned>June 6, 2018</returned>
    <submitted>March 1, 2019</submitted>
    <returned>March 28, 2019</returned>
  </pending_results>
  <!-- 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/NCT02199925
org study id: GMXAUT01
nct id: NCT02199925
lead sponsor: 
collaborator: 
has dmc: Yes
If autism is the consequence of a chronic inflammatory process preventing the infant brain
      from forming the proper neural connections, then treatment that reduces inflammation might
      have a positive impact on autism. IGIV treatment has been known to suppress inflammation and
      has been used in the treatment of inflammatory conditions and autoimmune diseases.
We have come to believe that an immature immune system with abnormal signaling and altered
      apoptotic pathways may result in a process of neuroinflammation with a clinical presentation
      of Autism Spectrum Disorder. If Autism Spectrum Disorders are the consequence of a chronic
      inflammatory process that prevents the infant brain from forming the proper neural
      connections, then treatment that reduces inflammation might have a positive impact on autism.
      We believe that there is a correlation between immunological abnormalities and the
      development of the autism.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction.
time frame: 1 year
description: The primary endpoint is disease improvement, as evidenced by standardized test measures across 3 intervals: screening for baseline measurements, visit 5, and end of study visit measurements. Primary efficacy variables will be measured using the following standardized tests
Caregiver Scales (SRS, CCC-2, ABC)
CGI-S and CGI - I
PPVT
ADOS
measure: The modulatory effect of IVIG on neuroinflammation will be assessed by laboratory measurements which we believe are indicative of immune dysregulation.
time frame: 1 year
measure: To improve development in autistic spectrum
time frame: 1 year
arm group label: Gammaplex 5% IGIV
arm group type: Experimental
description: Gammaplex 5% IGIV administered intravenously
intervention type: Drug
intervention name: Gammaplex 5%
arm group label: Gammaplex 5% IGIV
criteria: 
gender: All
minimum age: 4 Years
maximum age: 12 Years
healthy volunteers: No
last name: Isaac Melamed, MD
role: Principal Investigator
affiliation: IMMUNOe International Clinical Research Centers
facility: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: IMMUNOe Research Centers
investigator full name: Isaac Melamed
investigator title: Principal Investigator
mesh term: Autistic Disorder
mesh term: Developmental Disabilities
mesh term: Autism Spectrum Disorder
mesh term: Asperger Syndrome
mesh term: gamma-Globulins
mesh term: Immunoglobulins, Intravenous
mesh term: Rho(D) Immune Globulin
submitted: May 7, 2018
returned: June 6, 2018
submitted: March 1, 2019
returned: March 28, 2019