<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/NCT02192749</url>
  </required_header>
  <id_info>
    <org_study_id>TBS-UCMSC-ASD001</org_study_id>
    <nct_id>NCT02192749</nct_id>
  </id_info>
  <brief_title>Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism</brief_title>
  <official_title>Open, Prospective Trial of Treatment of Autism Spectrum Disorders (ASD) Using Intravenous Infusion of Umbilical Cord Tissue Mesenchymal Stem Cells (UC-MSC)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Translational Biosciences</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Translational Biosciences</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Allogeneic (not from the subject) human umbilical cord tissue-derived stem cells administered&#xD;
      intravenously (IV) in a series of 4 infusions every 3 months over the course of one year is&#xD;
      safe and will induce a therapeutic effect in autism patients.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>July 2014</start_date>
  <completion_date type="Actual">August 2017</completion_date>
  <primary_completion_date type="Actual">August 2017</primary_completion_date>
  <phase>Phase 1/Phase 2</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>Number of participants with adverse events</measure>
    <time_frame>89 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC)</measure>
    <time_frame>13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of participants with a change in disability as measured by the Childhood Autism Rating Scale (CARS)</measure>
    <time_frame>13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline macrophage-derived chemokine (MDC)</measure>
    <time_frame>13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline thymus and activation-regulated chemokine (TARC)</measure>
    <time_frame>13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Autism</condition>
  <arm_group>
    <arm_group_label>Umbilical cord mesenchymal stem cells</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Umbilical cord mesenchymal stem cells</intervention_name>
    <arm_group_label>Umbilical cord mesenchymal stem cells</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male or Female&#xD;
&#xD;
          -  Ages 6 to 16&#xD;
&#xD;
          -  Diagnostic and Statistical Manual of Mental Disorders (DSM IV) diagnosis of autism&#xD;
             confirmed by Autism Diagnostic Observation Schedule (ADOS) and/or Autism Diagnostic&#xD;
             Interview-Revised (ADI-R)&#xD;
&#xD;
          -  No anticipated changes in treatment for the study duration (e.g., diet, nutrients)&#xD;
&#xD;
          -  No additional biomedical treatments started 6 weeks prior to enrollment&#xD;
&#xD;
          -  No changes in dietary management for 3 months prior to enrollment&#xD;
&#xD;
          -  Ambulatory or require minimum support walking, per parent&#xD;
&#xD;
          -  Able to sit still for 5 minutes or longer with a preferred toy item, per parent&#xD;
&#xD;
          -  Adequate vision and hearing for the purposes of test administration, per parent&#xD;
&#xD;
          -  Adequate arm-hand-finger coordination (i.e., able to point) for learning and cognitive&#xD;
             tasks used in outcome measurement, per parent&#xD;
&#xD;
          -  Stable and controlled mental disorder&#xD;
&#xD;
          -  Under the care of a caregiver willing to participate by attending regularly scheduled&#xD;
             appointments and completing the necessary measures&#xD;
&#xD;
          -  Normal heavy metals test for lead and mercury levels performed within 30 days of first&#xD;
             stem cell infusion&#xD;
&#xD;
          -  Must provide name and specialty of specialist who has made Autism Spectrum Disorder&#xD;
             (ASD) diagnosis&#xD;
&#xD;
          -  Adequate financial means to cover $7,200 (US Dollars) plus travel expenses&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Significant prematurity at birth (less than 32 weeks gestation); or birth weight&#xD;
             significantly below normal for gestational age (SGA - small for gestational age)&#xD;
&#xD;
          -  mental retardation&#xD;
&#xD;
          -  seizure disorder&#xD;
&#xD;
          -  auto-immune conditions&#xD;
&#xD;
          -  history of head trauma and other neurological or medical conditions&#xD;
&#xD;
          -  Abnormal heavy metals test for lead and mercury performed within 30 days of first stem&#xD;
             cell infusion&#xD;
&#xD;
          -  Prior stem cell therapy of any kind&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>16 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Nelson Novarro, MD</last_name>
    <role>Principal Investigator</role>
  </overall_official>
  <overall_official>
    <last_name>Jorge Paz-Rodriguez, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Translational Biosciences / Stem Cell Institute Panama</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Stem Cell Institute</name>
      <address>
        <city>Panama City</city>
        <country>Panama</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Panama</country>
  </location_countries>
  <verification_date>October 2017</verification_date>
  <study_first_submitted>July 12, 2014</study_first_submitted>
  <study_first_submitted_qc>July 15, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 17, 2014</study_first_posted>
  <last_update_submitted>October 12, 2017</last_update_submitted>
  <last_update_submitted_qc>October 12, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">October 13, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>autism</keyword>
  <keyword>umbilical cord</keyword>
  <keyword>mesenchymal</keyword>
  <keyword>stem cells</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Autistic Disorder</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/NCT02192749
org study id: TBS-UCMSC-ASD001
nct id: NCT02192749
lead sponsor: 
has dmc: Yes
Allogeneic (not from the subject) human umbilical cord tissue-derived stem cells administered
      intravenously (IV) in a series of 4 infusions every 3 months over the course of one year is
      safe and will induce a therapeutic effect in autism patients.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of participants with adverse events
time frame: 89 weeks
measure: Number of participants with a change in disability as measured by the Autism Treatment Evaluation Checklist (ATEC)
time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
measure: Number of participants with a change in disability as measured by the Childhood Autism Rating Scale (CARS)
time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
measure: Change from baseline macrophage-derived chemokine (MDC)
time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
measure: Change from baseline thymus and activation-regulated chemokine (TARC)
time frame: 13 weeks, 25 weeks, 37 weeks, 49 weeks, 89 weeks
arm group label: Umbilical cord mesenchymal stem cells
arm group type: Experimental
intervention type: Biological
intervention name: Umbilical cord mesenchymal stem cells
arm group label: Umbilical cord mesenchymal stem cells
criteria: 
gender: All
minimum age: 6 Years
maximum age: 16 Years
healthy volunteers: No
last name: Nelson Novarro, MD
role: Principal Investigator
last name: Jorge Paz-Rodriguez, MD
role: Principal Investigator
affiliation: Translational Biosciences / Stem Cell Institute Panama
facility: 
country: Panama
responsible party type: Sponsor
mesh term: Autistic Disorder