<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/NCT02235844</url>
  </required_header>
  <id_info>
    <org_study_id>IND 16026 DMD Single Patient</org_study_id>
    <nct_id>NCT02235844</nct_id>
  </id_info>
  <brief_title>Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)</brief_title>
  <official_title>Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Allergy and Asthma Consultants, Wichita, Kansas</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Aidan Foundation</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Neil H. Riordan PhD</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Allergy and Asthma Consultants, Wichita, Kansas</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
  </oversight_info>
  <brief_summary>
    <textblock>
      This research study is designed to evaluate the effects of human umbilical cord mesenchymal&#xD;
      stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle&#xD;
      regenerative and anti-inflammatory properties of UC MSCs position them as a possible&#xD;
      treatment option for DMD. Both of these properties could lead to potential benefits for a DMD&#xD;
      patient.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">September 2014</start_date>
  <completion_date type="Actual">September 30, 2017</completion_date>
  <primary_completion_date type="Actual">September 30, 2017</primary_completion_date>
  <phase>Phase 1</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>Adverse Events</measure>
    <time_frame>3 months after final treatment</time_frame>
    <description>No occurrence of adverse events</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of weight</measure>
    <time_frame>3 months after final treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change of muscle diameter (circumferential measurements) from baseline</measure>
    <time_frame>3 months after final treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of Pulmonary Maximum Expiratory Pressure</measure>
    <time_frame>3 months after final treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of Pulmonary Forced Vital Capacity</measure>
    <time_frame>3 months after final treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Maximum Change from baseline of Predicted Inspiratory Pressure %</measure>
    <time_frame>3 months after final treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of Predicted Maximum Expiratory Pressure %</measure>
    <time_frame>3 months after final treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline of Predicted Forced Vital Capacity %</measure>
    <time_frame>3 months after final treatment</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">1</enrollment>
  <condition>Duchenne's Muscular Dystrophy</condition>
  <arm_group>
    <arm_group_label>Mesenchymal Stem Cells</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Umbilical Cord Mesenchymal Stem Cells</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Umbilical Cord Mesenchymal Stem Cells</intervention_name>
    <arm_group_label>Mesenchymal Stem Cells</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Duchenne's Muscular Dystrophy&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  None&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>28 Years</minimum_age>
    <maximum_age>31 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Maurice HV Strickland, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Allergy and Asthma Consultants of Wichita, KS</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Asthma and Allergy Consultants</name>
      <address>
        <city>Wichita</city>
        <state>Kansas</state>
        <zip>67205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2019</verification_date>
  <study_first_submitted>September 8, 2014</study_first_submitted>
  <study_first_submitted_qc>September 8, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 10, 2014</study_first_posted>
  <last_update_submitted>September 12, 2019</last_update_submitted>
  <last_update_submitted_qc>September 12, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">September 16, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Mesenchymal</keyword>
  <keyword>Stem Cells</keyword>
  <keyword>Umbilical Cord</keyword>
  <keyword>Duchenne's</keyword>
  <keyword>Muscular Dystrophy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophy, Duchenne</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/NCT02235844
org study id: IND 16026 DMD Single Patient
nct id: NCT02235844
lead sponsor: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: Yes
This research study is designed to evaluate the effects of human umbilical cord mesenchymal
      stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle
      regenerative and anti-inflammatory properties of UC MSCs position them as a possible
      treatment option for DMD. Both of these properties could lead to potential benefits for a DMD
      patient.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Adverse Events
time frame: 3 months after final treatment
description: No occurrence of adverse events
measure: Change from baseline of weight
time frame: 3 months after final treatment
measure: Change of muscle diameter (circumferential measurements) from baseline
time frame: 3 months after final treatment
measure: Change from baseline of Pulmonary Maximum Expiratory Pressure
time frame: 3 months after final treatment
measure: Change from baseline of Pulmonary Forced Vital Capacity
time frame: 3 months after final treatment
measure: Maximum Change from baseline of Predicted Inspiratory Pressure %
time frame: 3 months after final treatment
measure: Change from baseline of Predicted Maximum Expiratory Pressure %
time frame: 3 months after final treatment
measure: Change from baseline of Predicted Forced Vital Capacity %
time frame: 3 months after final treatment
arm group label: Mesenchymal Stem Cells
arm group type: Experimental
description: Umbilical Cord Mesenchymal Stem Cells
intervention type: Biological
intervention name: Umbilical Cord Mesenchymal Stem Cells
arm group label: Mesenchymal Stem Cells
criteria: 
gender: Male
minimum age: 28 Years
maximum age: 31 Years
healthy volunteers: No
last name: Maurice HV Strickland, MD
role: Principal Investigator
affiliation: Allergy and Asthma Consultants of Wichita, KS
facility: 
country: United States
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophy, Duchenne