<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/NCT01711073</url>
  </required_header>
  <id_info>
    <org_study_id>Prot001</org_study_id>
    <nct_id>NCT01711073</nct_id>
  </id_info>
  <brief_title>Mobilization of Stem Cells With G-CSF and Mozobil in Patients With End Stage Liver Disease</brief_title>
  <official_title>Mobilization of Stem Cells With G-CSF and Mozobil in Patients With End Stage Liver Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Proteonomix, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>University of Medicine and Dentistry of New Jersey</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Numoda</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Proteonomix, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      A phase I trial to study the safety of mobilization of stem cells with G-CSF and Mozobil in&#xD;
      patients with chronic liver disease.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Liver cirrhosis in humans represents the end stage of chronic liver injury. Supply of &quot;new&quot;&#xD;
      stem cells to the liver could regenerate hepatocytes and restore the lost function. Delivery&#xD;
      of Mesenchymal Stem Cells (MSCs) has been shown in animal models and limited clinical trials&#xD;
      to result in improved liver disease (MELD) score.&#xD;
&#xD;
      In preclinical studies we have demonstrated that the combination of G-CSF plus Mozobil can&#xD;
      effectively mobilize both hematopoietic stem cells (HSCs) and MSCs into the peripheral&#xD;
      circulation. While G-CSF only mobilizes HSCs.&#xD;
&#xD;
      The clinical trial will test the safety of treating patients with end stage liver disease&#xD;
      with G-CSF and Mozobil to mobilize MSCs into the peripheral circulation.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date>October 2012</start_date>
  <completion_date type="Anticipated">September 2013</completion_date>
  <primary_completion_date type="Anticipated">September 2013</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>Toxicity as measured by bone pain, hematologic parameters, GI measures and renal parameters</measure>
    <time_frame>12 months</time_frame>
    <description>The primary end point for this study is the safety of mobilization of stem cells in patients with end stage liver disease. Adverse events will be documented to assess safety.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Effects of Mobilization</measure>
    <time_frame>12 months</time_frame>
    <description>The secondary objective is to study the mobilization of stem cells, including MSCs, to the peripheral circulation and the effect on liver function. Functional assays will define the levels of heamtopoietic stem cells (CD34+ cells) and MSCs (CFU-F) in the circulation of patients.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">15</enrollment>
  <condition>End Stage Liver DIsease</condition>
  <arm_group>
    <arm_group_label>Mobilization with G-CSF plus Mozobil</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Patients will receive G-CSF (Filgrastim) plus Mozobil (Plerixafor)</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Mobilization with G-CSF and Mozobil</intervention_name>
    <description>Treatment with drugs for mobilization of MSCs</description>
    <arm_group_label>Mobilization with G-CSF plus Mozobil</arm_group_label>
    <other_name>Filgrastim</other_name>
    <other_name>Neupogen</other_name>
    <other_name>Plerixafor</other_name>
    <other_name>AMD3100</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients with a clinical diagnosis of cirrhosis Age greater than or equal to 18 years&#xD;
             MELD score less than or equal to 12 able to provide informed consent HIV and HBsAg&#xD;
             seronegative Platelet count &gt;50,000, WBC count &gt; 2,000 No history of malignancy within&#xD;
             the last 5 years, except for non-melanoma skin cancer or cervical carcinoma in situ No&#xD;
             lesions suspicious for liver cancer on CT and/or MRI within prior 4 months&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Patients with acute or subacute onset of liver disease Patients who have received a liver&#xD;
        transplant Age &lt; 18 MELD score &gt;12 Patients whose MELD scores are currently less than or&#xD;
        equal to 12 but with history of prior deterioration with MELD score &gt;12 Unable to provide&#xD;
        informed consent Patients with HIV or HBsAg seropositivity Pregnant or lactating females&#xD;
        Enrolled in another research protocol Any condition that precludes serial follow up&#xD;
        Patients with history of malignancy within the last 5 years, except for non-melanoma skin&#xD;
        cancer or cervical carcinoma in situ Any lesions suspicious for liver cancer on CT and/or&#xD;
        MRI within prior 4 months Patients with palpable splenomegaly on physical examination ANy&#xD;
        condition that in the investigators opinion would likely increase the risk of particpation&#xD;
        or would likely confound interpretation of the data&#xD;
&#xD;
        -&#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>Baburao Koneru, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Medicine and Dentistry of New Jersey</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Medicine and Dentistry of New Jersey</name>
      <address>
        <city>Newark</city>
        <state>New Jersey</state>
        <zip>07101</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Baburao Koneru, MD</last_name>
      <email>koneruba@umdnj.edu</email>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2013</verification_date>
  <study_first_submitted>October 11, 2012</study_first_submitted>
  <study_first_submitted_qc>October 19, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 22, 2012</study_first_posted>
  <last_update_submitted>January 23, 2013</last_update_submitted>
  <last_update_submitted_qc>January 23, 2013</last_update_submitted_qc>
  <last_update_posted type="Estimate">January 24, 2013</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Liver Diseases</mesh_term>
    <mesh_term>End Stage Liver Disease</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Plerixafor</mesh_term>
  </intervention_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/NCT01711073
org study id: Prot001
nct id: NCT01711073
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
A phase I trial to study the safety of mobilization of stem cells with G-CSF and Mozobil in
      patients with chronic liver disease.
Liver cirrhosis in humans represents the end stage of chronic liver injury. Supply of "new"
      stem cells to the liver could regenerate hepatocytes and restore the lost function. Delivery
      of Mesenchymal Stem Cells (MSCs) has been shown in animal models and limited clinical trials
      to result in improved liver disease (MELD) score.

      In preclinical studies we have demonstrated that the combination of G-CSF plus Mozobil can
      effectively mobilize both hematopoietic stem cells (HSCs) and MSCs into the peripheral
      circulation. While G-CSF only mobilizes HSCs.

      The clinical trial will test the safety of treating patients with end stage liver disease
      with G-CSF and Mozobil to mobilize MSCs into the peripheral circulation.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Toxicity as measured by bone pain, hematologic parameters, GI measures and renal parameters
time frame: 12 months
description: The primary end point for this study is the safety of mobilization of stem cells in patients with end stage liver disease. Adverse events will be documented to assess safety.
measure: Effects of Mobilization
time frame: 12 months
description: The secondary objective is to study the mobilization of stem cells, including MSCs, to the peripheral circulation and the effect on liver function. Functional assays will define the levels of heamtopoietic stem cells (CD34+ cells) and MSCs (CFU-F) in the circulation of patients.
arm group label: Mobilization with G-CSF plus Mozobil
arm group type: Experimental
description: Patients will receive G-CSF (Filgrastim) plus Mozobil (Plerixafor)
intervention type: Drug
intervention name: Mobilization with G-CSF and Mozobil
description: Treatment with drugs for mobilization of MSCs
arm group label: Mobilization with G-CSF plus Mozobil
other name: Filgrastim
other name: Neupogen
other name: Plerixafor
other name: AMD3100
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: No
last name: Baburao Koneru, MD
role: Principal Investigator
affiliation: University of Medicine and Dentistry of New Jersey
facility: 
status: Recruiting
contact: 
country: United States
responsible party type: Sponsor
mesh term: Liver Diseases
mesh term: End Stage Liver Disease
mesh term: Plerixafor