<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/NCT02983708</url>
  </required_header>
  <id_info>
    <org_study_id>CP-PB-2011</org_study_id>
    <nct_id>NCT02983708</nct_id>
  </id_info>
  <brief_title>Neuroregenerative Potential of Intravenous G-CSF and Autologous Peripheral Blood Stem Cells</brief_title>
  <official_title>Neuroregenerative Potential of Intravenous G-CSF and Autologous Peripheral Blood Stem Cells in Children With Cerebral Palsy: a Randomized, Double-blind Cross-over Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Hanyang University Seoul Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Ministry of Health &amp; Welfare, Korea</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Hanyang University Seoul Hospital</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The current study describes a randomized, double-blind, cross-over study of intravenous G-CSF&#xD;
      followed by infusion with autologous mobilized peripheral blood mononuclear cells (mPBMCs) in&#xD;
      children with cerebral palsy (CP) to determine the safety and feasibility of the procedure,&#xD;
      as well as the potential efficacy for improving neurological impairment.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      We hypothesized that mobilized peripheral blood mononuclear cells (mPBMCs) would be a better&#xD;
      source of cell therapy for children with CP, if these cells had a similar neuroregenerative&#xD;
      potential to bone marrow/cord blood mononuclear cells (MNCs). Multipotent precursor cells&#xD;
      exist in peripheral blood, and a fraction of elutriated blood cells from normal individuals&#xD;
      contains MNCs that have the potential to be MSCs. There are several advantages to using&#xD;
      mPBMCs for cell therapy in children with CP: the G-CSF that is used to mPBMCs has&#xD;
      neuroregenerative potential; the collection and fractionation of stem cells can be repeated;&#xD;
      and, the therapy is suitable for most children with CP.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>August 2011</start_date>
  <completion_date type="Actual">September 2014</completion_date>
  <primary_completion_date type="Actual">September 2014</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>Randomized</allocation>
    <intervention_model>Crossover Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Overall improvement as a score changes in GMFM &gt; 4 points</measure>
    <time_frame>6 months</time_frame>
  </primary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">57</enrollment>
  <condition>Neurodegeneration</condition>
  <condition>G-CSF</condition>
  <condition>Peripheral Blood Mononuclear Cells</condition>
  <condition>Cerebral Palsy</condition>
  <arm_group>
    <arm_group_label>mPBMC group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. mPBMCs group would be included all patients who received mPBMCs at M1 or M7.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo group</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. Placebo group would be included all patients who received placebo at M1 or M7.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Peripheral blood mononuclear cells (mPBMC)</intervention_name>
    <arm_group_label>mPBMC group</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>G-CSF</intervention_name>
    <arm_group_label>Placebo group</arm_group_label>
    <arm_group_label>mPBMC group</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <arm_group_label>Placebo group</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Non severe type of cerebral palsy&#xD;
&#xD;
          -  Evidences of abnormal MRI findings such as periventricular leukomalacia&#xD;
&#xD;
          -  Collected mobilized peripheral blood mononuclear cell counts &gt; 1×10^8/kg or CD34+ cell&#xD;
             counts &gt; 1×10^6/kg&#xD;
&#xD;
          -  Consent form&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Previous trials of autologous cord blood infusion or erythropoietin/G-CSF&#xD;
&#xD;
          -  Chromosomal abnormalities&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>10 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <verification_date>December 2016</verification_date>
  <study_first_submitted>December 2, 2016</study_first_submitted>
  <study_first_submitted_qc>December 2, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 6, 2016</study_first_posted>
  <last_update_submitted>December 5, 2016</last_update_submitted>
  <last_update_submitted_qc>December 5, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">December 7, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Hanyang University Seoul Hospital</investigator_affiliation>
    <investigator_full_name>Young-Ho Lee</investigator_full_name>
    <investigator_title>Professor</investigator_title>
  </responsible_party>
  <keyword>Neuroregeneration</keyword>
  <keyword>G-CSF</keyword>
  <keyword>Peripheral blood mononuclear cells</keyword>
  <keyword>Cerebral palsy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cerebral Palsy</mesh_term>
    <mesh_term>Nerve Degeneration</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
    <ipd_description>We have to get individual permissions to make IPD available to other researchers.</ipd_description>
  </patient_data>
  <!-- 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/NCT02983708
org study id: CP-PB-2011
nct id: NCT02983708
lead sponsor: 
collaborator: 
has dmc: Yes
The current study describes a randomized, double-blind, cross-over study of intravenous G-CSF
      followed by infusion with autologous mobilized peripheral blood mononuclear cells (mPBMCs) in
      children with cerebral palsy (CP) to determine the safety and feasibility of the procedure,
      as well as the potential efficacy for improving neurological impairment.
We hypothesized that mobilized peripheral blood mononuclear cells (mPBMCs) would be a better
      source of cell therapy for children with CP, if these cells had a similar neuroregenerative
      potential to bone marrow/cord blood mononuclear cells (MNCs). Multipotent precursor cells
      exist in peripheral blood, and a fraction of elutriated blood cells from normal individuals
      contains MNCs that have the potential to be MSCs. There are several advantages to using
      mPBMCs for cell therapy in children with CP: the G-CSF that is used to mPBMCs has
      neuroregenerative potential; the collection and fractionation of stem cells can be repeated;
      and, the therapy is suitable for most children with CP.
allocation: Randomized
intervention model: Crossover Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Overall improvement as a score changes in GMFM > 4 points
time frame: 6 months
arm group label: mPBMC group
arm group type: Experimental
description: G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. mPBMCs group would be included all patients who received mPBMCs at M1 or M7.
arm group label: Placebo group
arm group type: Placebo Comparator
description: G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. Placebo group would be included all patients who received placebo at M1 or M7.
intervention type: Biological
intervention name: Peripheral blood mononuclear cells (mPBMC)
arm group label: mPBMC group
intervention type: Drug
intervention name: G-CSF
arm group label: Placebo group
arm group label: mPBMC group
intervention type: Drug
intervention name: Placebo
arm group label: Placebo group
criteria: 
gender: All
minimum age: 2 Years
maximum age: 10 Years
healthy volunteers: No
responsible party type: Principal Investigator
investigator affiliation: Hanyang University Seoul Hospital
investigator full name: Young-Ho Lee
investigator title: Professor
mesh term: Cerebral Palsy
mesh term: Nerve Degeneration
sharing ipd: Undecided
ipd description: We have to get individual permissions to make IPD available to other researchers.