<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/NCT03791372</url>
  </required_header>
  <id_info>
    <org_study_id>GuangdongWCHRD</org_study_id>
    <nct_id>NCT03791372</nct_id>
  </id_info>
  <brief_title>Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy</brief_title>
  <official_title>Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Guangdong Women and Children Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Guangdong Women and Children Hospital</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in&#xD;
      the treatment of cerebral palsy.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a Phase 1 clinical trial that constitutes two time points cohorts with participants&#xD;
      who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride&#xD;
      respectively. The timing of treatment is any time after the diagnosis of cerebral palsy. The&#xD;
      investigator will proceed the groups during the same period.&#xD;
&#xD;
        1. Demographic Data and Baseline Characteristics of the Studied Group were collected:&#xD;
&#xD;
             -  Basic patient's information survey&#xD;
&#xD;
             -  Medical history&#xD;
&#xD;
             -  Physical examination&#xD;
&#xD;
             -  Basic blood test result&#xD;
&#xD;
             -  Children's developmental disorders evaluation before the treatment&#xD;
&#xD;
             -  Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRI-DTI) before the&#xD;
                treatment&#xD;
&#xD;
             -  Neurocognitive function test before the treatment&#xD;
&#xD;
        2. Assessment of clinical condition in the course by measurement of blood pressure, heart&#xD;
           and respiratory rates, temperature and adverse events was recorded.&#xD;
&#xD;
        3. Autologous cord blood doses is 20-30ml (total Mononuclear cells&gt;1*10^7/kg)，the infusion&#xD;
           speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo.&#xD;
&#xD;
        4. The follow-up: clinical test until 30th month in 3 month gaps.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">October 10, 2017</start_date>
  <completion_date type="Anticipated">April 10, 2021</completion_date>
  <primary_completion_date type="Anticipated">October 10, 2020</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <intervention_model_description>Parallel assignment</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Single (Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The Change of Gross Motor Function Classification System(GMFCS) Score</measure>
    <time_frame>up to 30 months after therapy at a 3-month interval</time_frame>
    <description>GMFCS is used to assess the gross motor function of the children who were diagnosised as cerebral palsy.The results of GMFCS are divided into five levels. Grade I represents the best outcome while grade Ⅴ represents the worst.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>The Change of Gesell Developmental Scale(GDS) Score</measure>
    <time_frame>every 3-month after therapy until 30 months</time_frame>
    <description>The GDS is used to measure the gross motor,fine action,adaptability，language and individual-society function of the children whose age ranges from 1-month to 6-year-old. The score less than 75 for each subscale represent the worse outcome and more than 75 represent the better.</description>
  </primary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">25</enrollment>
  <condition>Safety Issues</condition>
  <condition>Effect of Drugs</condition>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cord Blood Mononuclear Cells(CBMNC)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Autologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells&gt;1*10^7/kg). The infusion speed is 1ml/min.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>0.9% Sodium Chloride</intervention_name>
    <description>0.9% Sodium Chloride in control group</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Autologous Umbilical Cord Blood</intervention_name>
    <description>Autologous Umbilical Cord Blood Mononuclear Cells Therapy in Cerebral Palsy for safety and effect evaluation.</description>
    <arm_group_label>Cord Blood Mononuclear Cells(CBMNC)</arm_group_label>
    <other_name>Autologous Umbilical Cord Blood Mononuclear Cells Therapy</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  A patient who was diagnosed with a mild case, and severity case of cerebral palsy or&#xD;
             developmental disability through developmental assessment from more than two&#xD;
             specialists including a specialist in pediatrics and rehabilitation medicine..&#xD;
&#xD;
          -  In case there is a sign of the general decrease in white matter, or periventricular&#xD;
             leukomalacia near cerebral ventricles in MRI DTI test.&#xD;
&#xD;
          -  A subject who did a written consent to participation in this clinical trial through&#xD;
             the very person and a legal representative.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  In case a patient underwent a surgical procedure, or was administered autologous cord&#xD;
             blood within one year before participating in a clinical trial.&#xD;
&#xD;
          -  Accompanied by a serious disease, such as chromosome abnormality, etc.&#xD;
&#xD;
          -  In case where a patient's medical condition is judged to be maladapted by a&#xD;
             researcher.&#xD;
&#xD;
          -  In case a patient or his or her legal representative doesn't agree to participation in&#xD;
             a clinical trial.&#xD;
&#xD;
          -  A patient having a predisposition to allergies.&#xD;
&#xD;
          -  A patient having serious disorders in the liver, kidney, and cardiopulmonary function.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Months</minimum_age>
    <maximum_age>10 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Chang Yanqun, Phd</last_name>
    <role>Study Director</role>
    <affiliation>Guangdong Women and Children Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Xiao Huimei, MD</last_name>
    <phone>+862039151772</phone>
    <phone_ext>+86</phone_ext>
    <email>xiaowu2010727@163.com</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Chang Yanqun, Phd</last_name>
    <phone>+862039151772</phone>
    <phone_ext>+86</phone_ext>
    <email>sfycyq@sina.com</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Guangdong Women and Children's Hospital and Health Institute</name>
      <address>
        <city>Guangzhou</city>
        <state>Guangdong</state>
        <zip>510010</zip>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Chang Yanqun, Phd</last_name>
      <phone>+862039151772</phone>
      <email>sfycyq@sina.com</email>
    </contact>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <reference>
    <citation>Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S, Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28.</citation>
    <PMID>29080265</PMID>
  </reference>
  <verification_date>January 2019</verification_date>
  <study_first_submitted>December 18, 2018</study_first_submitted>
  <study_first_submitted_qc>December 29, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">January 2, 2019</study_first_posted>
  <last_update_submitted>January 3, 2019</last_update_submitted>
  <last_update_submitted_qc>January 3, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">January 4, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Guangdong Women and Children Hospital</investigator_affiliation>
    <investigator_full_name>Xiao Huimei</investigator_full_name>
    <investigator_title>Professor</investigator_title>
  </responsible_party>
  <keyword>Cerebral palsy</keyword>
  <keyword>Autologous Umbilical Cord Blood</keyword>
  <keyword>safety</keyword>
  <keyword>effect</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cerebral Palsy</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </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/NCT03791372
org study id: GuangdongWCHRD
nct id: NCT03791372
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in
      the treatment of cerebral palsy.
This is a Phase 1 clinical trial that constitutes two time points cohorts with participants
      who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride
      respectively. The timing of treatment is any time after the diagnosis of cerebral palsy. The
      investigator will proceed the groups during the same period.

        1. Demographic Data and Baseline Characteristics of the Studied Group were collected:

             -  Basic patient's information survey

             -  Medical history

             -  Physical examination

             -  Basic blood test result

             -  Children's developmental disorders evaluation before the treatment

             -  Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRI-DTI) before the
                treatment

             -  Neurocognitive function test before the treatment

        2. Assessment of clinical condition in the course by measurement of blood pressure, heart
           and respiratory rates, temperature and adverse events was recorded.

        3. Autologous cord blood doses is 20-30ml (total Mononuclear cells>1*10^7/kg)，the infusion
           speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo.

        4. The follow-up: clinical test until 30th month in 3 month gaps.
allocation: Non-Randomized
intervention model: Parallel Assignment
intervention model description: Parallel assignment
primary purpose: Treatment
masking: Single (Outcomes Assessor)
measure: The Change of Gross Motor Function Classification System(GMFCS) Score
time frame: up to 30 months after therapy at a 3-month interval
description: GMFCS is used to assess the gross motor function of the children who were diagnosised as cerebral palsy.The results of GMFCS are divided into five levels. Grade I represents the best outcome while grade Ⅴ represents the worst.
measure: The Change of Gesell Developmental Scale(GDS) Score
time frame: every 3-month after therapy until 30 months
description: The GDS is used to measure the gross motor,fine action,adaptability，language and individual-society function of the children whose age ranges from 1-month to 6-year-old. The score less than 75 for each subscale represent the worse outcome and more than 75 represent the better.
arm group label: Placebo
arm group type: Placebo Comparator
description: 0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min.
arm group label: Cord Blood Mononuclear Cells(CBMNC)
arm group type: Experimental
description: Autologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells>1*10^7/kg). The infusion speed is 1ml/min.
intervention type: Drug
intervention name: 0.9% Sodium Chloride
description: 0.9% Sodium Chloride in control group
arm group label: Placebo
intervention type: Biological
intervention name: Autologous Umbilical Cord Blood
description: Autologous Umbilical Cord Blood Mononuclear Cells Therapy in Cerebral Palsy for safety and effect evaluation.
arm group label: Cord Blood Mononuclear Cells(CBMNC)
other name: Autologous Umbilical Cord Blood Mononuclear Cells Therapy
criteria: 
gender: All
minimum age: 6 Months
maximum age: 10 Years
healthy volunteers: No
last name: Chang Yanqun, Phd
role: Study Director
affiliation: Guangdong Women and Children Hospital
last name: Xiao Huimei, MD
phone: +862039151772
phone ext: +86
email: xiaowu2010727@163.com
last name: Chang Yanqun, Phd
phone: +862039151772
phone ext: +86
email: sfycyq@sina.com
facility: 
status: Recruiting
contact: 
country: China
citation: Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S, Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28.
PMID: 29080265
responsible party type: Principal Investigator
investigator affiliation: Guangdong Women and Children Hospital
investigator full name: Xiao Huimei
investigator title: Professor
mesh term: Cerebral Palsy
sharing ipd: Undecided