<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/NCT01700166</url>
  </required_header>
  <id_info>
    <org_study_id>JB IND #14576</org_study_id>
    <nct_id>NCT01700166</nct_id>
  </id_info>
  <brief_title>Umbilical Cord Blood in the Treatment of Stroke in Children.</brief_title>
  <acronym>Pedi Stroke</acronym>
  <official_title>Safety of Autologous Human Umbilical Cord Blood in the Treatment of Stroke in Children.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>James E.Baumgartner, MD</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>The Institute for Rehabilitaion and Research Foundation</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Cord Blood Registry, Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>The University of Texas Health Science Center, Houston</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>M.D. Anderson Cancer Center - Houston</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Baylor College of Medicine</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Memorial Hermann Health System</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The specific aims of this study are:&#xD;
&#xD;
        1. To determine if Human Umbilical Cord Blood (hUCB) infusion is safe in children with&#xD;
           perinatal arterial ischemic stroke (AIS).&#xD;
&#xD;
        2. To determine if late functional outcome, physiologic response, and anatomic findings are&#xD;
           changed following hUCB infusion in children with perinatal AIS.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This study will enroll ten pediatric patients who have their umbilical cord blood banked with&#xD;
      Cord Blood Registry, Inc. (CBR), who have been diagnosed with an arterial ischemic stroke&#xD;
      (AIS), and whose caregivers contacted CBR, Inc. to notify them that the child has experienced&#xD;
      an AIS around the time of birth.&#xD;
&#xD;
      After receiving permission to release the patient's contact information, the CBR team will&#xD;
      then relay the contact information to the Stem Cell Study Group at the University of Texas -&#xD;
      Houston (UT) / Children's Memorial Hermann Hospital (CMHH) who will contact the family,&#xD;
      explain the study, and send a consent form for their review.&#xD;
&#xD;
      Several weeks prior to the scheduled procedure date, the patient will go to their family&#xD;
      doctor for a physical evaluation and have blood drawn to rule out problems with the child's&#xD;
      health.&#xD;
&#xD;
      After successfully meeting initial pre-screening criteria, the patient's family will make&#xD;
      their own arrangements to travel to Houston for the study procedures.&#xD;
&#xD;
      Once the patient is in Houston, he/she will undergo baseline assessments, including physical,&#xD;
      neurological exams and tests, speech therapy testing, laboratory tests, and imaging (MRI) of&#xD;
      the brain. If any of the blood tests are abnormal, or the patient has an active infection,&#xD;
      the procedure may be postponed or the patient may possibly be excluded from the study.&#xD;
&#xD;
      If the patient continues to meet all criteria, the frozen cord blood will be shipped&#xD;
      overnight to a special lab in Houston, TX for processing. Once release criteria have been&#xD;
      met, the cells will be brought to the CMHH for infusion into the patient's vein. The patient&#xD;
      will remain at CHMM for 24 hours of monitoring, after which they will be allowed to go home.&#xD;
      Patients will be called daily by the research staff through Day 14, and once a month after&#xD;
      that.&#xD;
&#xD;
      Patients will return to Houston, Texas for 6 month, 1 year and 2 year follow-up visits for&#xD;
      repeat assessments, tests, and exams just like at the baseline visit. The MRI of the brain&#xD;
      will only be repeated at the 6 month visit.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    Principal Investigator relocated; Study may resume in Fl Hosp. for Children, Orlando FL&#xD;
  </why_stopped>
  <start_date>September 2012</start_date>
  <completion_date type="Anticipated">December 2016</completion_date>
  <primary_completion_date type="Actual">August 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>Functional outcome measure</measure>
    <time_frame>Two years</time_frame>
    <description>Physiological (e.g., SSEP, EEG) and anatomic outcome (MRI) changes will be evaluated by repeating assessments, clinical tests and imaging exams at the pre-treatment and follow-up visits.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Speech Therapy Specific Neuropsychological outcome measures</measure>
    <time_frame>Two years</time_frame>
    <description>Age appropriate Neurologic examinations and speech therapy specific standardized testing will be performed pre-treatment and at all follow-up visits.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Arterial Ischemic Stroke (AIS) in Children</condition>
  <arm_group>
    <arm_group_label>Biologic; Cord Blood Stem Cells; Intravenous injection</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Autologous Human Umbilical Cord Blood derived Stem Cell injection</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Autologous Human Cord Blood derived Stem Cell injection</intervention_name>
    <description>One time intravenous (in the vein) injection with two year follow-up</description>
    <arm_group_label>Biologic; Cord Blood Stem Cells; Intravenous injection</arm_group_label>
    <other_name>Patient's own cord blood</other_name>
    <other_name>Patient's own stem cells</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Between 6 weeks and 6 years of age on the day of study cord blood infusion.&#xD;
&#xD;
          2. MRI documented single arterial distribution infarction.&#xD;
&#xD;
          3. Initial injury occurring in the pre-natal or perinatal period.&#xD;
&#xD;
          4. Ability of caregivers to understand and speak English&#xD;
&#xD;
          5. Ability of child and caregiver to travel to Houston, and stay for at least 4 days, and&#xD;
             to return for all Follow-up visits (patient is responsible for cost of travel and&#xD;
             lodging while in Houston)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Inability to obtain all pertinent medical records, including pertinent physician notes,&#xD;
        laboratory findings, and radiographic images, related to the original injury,&#xD;
        hospitalization and rehabilitation - must be sent to research team at least 14 days prior&#xD;
        to scheduled study cord blood treatment.&#xD;
&#xD;
          1. Recent radiographic evidence (imaging performed within past 2 weeks) of extensive&#xD;
             stroke as evidenced by &gt;100ml lesion.&#xD;
&#xD;
          2. Multifocal infarctions on screening MRI.&#xD;
&#xD;
          3. Evidence of hypoxic-ischemic encephalopathy on screening MRI.&#xD;
&#xD;
          4. Uncorrected coagulopathy during the baseline period defined as INR &gt; 1.4; PTT&gt; 35 sec;&#xD;
             PLT &lt; 100,000.&#xD;
&#xD;
          5. Known history of:&#xD;
&#xD;
               1. Recently diagnosed infection (within past 2 weeks) requiring treatment and/or&#xD;
                  medical intervention.&#xD;
&#xD;
               2. Renal disease or altered renal function as defined by serum creatinine &gt; 1.5&#xD;
                  mg/dL at admission.&#xD;
&#xD;
               3. Hepatic disease or altered liver function as defined by SGPT &gt; 150 U/L, and/or T.&#xD;
                  Bilirubin &gt;1.3 mg/dL at enrollment.&#xD;
&#xD;
               4. Malignancy.&#xD;
&#xD;
               5. Immunosuppression as defined by WBC &lt; 3 (10x3) at admission.&#xD;
&#xD;
               6. HIV, Hepatitis B, Hepatitis C.&#xD;
&#xD;
          6. Pneumonia, or chronic lung disease requiring oxygen.&#xD;
&#xD;
          7. Cord blood sample contamination.&#xD;
&#xD;
          8. Participation in a concurrent intervention study.&#xD;
&#xD;
          9. Desire for organ-donation in the event of death.&#xD;
&#xD;
         10. Unwillingness or inability to stay for at least four days following cord blood&#xD;
             infusion (should any problems arise following the infusion) and to return for 6 month,&#xD;
             1 year, and 2 year follow-up visits.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Weeks</minimum_age>
    <maximum_age>6 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Timothy C. Foster, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>The University of Texas Health Science Center, Houston</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Linda S. Baumgartner, MS, CCC-SLP, LSLS, Cert.AVT</last_name>
    <role>Principal Investigator</role>
    <affiliation>Florida Hospital for Children - Orlando</affiliation>
  </overall_official>
  <overall_official>
    <last_name>James E. Baumgartner, MD</last_name>
    <role>Study Chair</role>
    <affiliation>Florida Hospital for Children - Orlando</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's Memorial Hermann Hospital; University of Texas Health Science Center - Houston</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2014</verification_date>
  <study_first_submitted>October 2, 2012</study_first_submitted>
  <study_first_submitted_qc>October 2, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 4, 2012</study_first_posted>
  <last_update_submitted>January 21, 2014</last_update_submitted>
  <last_update_submitted_qc>January 21, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">January 23, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Memorial Hermann Health System</investigator_affiliation>
    <investigator_full_name>James E.Baumgartner, MD</investigator_full_name>
    <investigator_title>Clinical Associate Professor; Board-certified Pediatric Neurosurgeon</investigator_title>
  </responsible_party>
  <keyword>Autologous</keyword>
  <keyword>Cord Blood</keyword>
  <keyword>Stem Cells</keyword>
  <keyword>Pediatric stroke</keyword>
  <keyword>Children with ischemic stroke</keyword>
  <keyword>Cerebral Palsy</keyword>
  <keyword>Perinatal AIS</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Stroke</mesh_term>
    <mesh_term>Ischemic Stroke</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/NCT01700166
org study id: JB IND #14576
nct id: NCT01700166
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: Yes
The specific aims of this study are:

        1. To determine if Human Umbilical Cord Blood (hUCB) infusion is safe in children with
           perinatal arterial ischemic stroke (AIS).

        2. To determine if late functional outcome, physiologic response, and anatomic findings are
           changed following hUCB infusion in children with perinatal AIS.
This study will enroll ten pediatric patients who have their umbilical cord blood banked with
      Cord Blood Registry, Inc. (CBR), who have been diagnosed with an arterial ischemic stroke
      (AIS), and whose caregivers contacted CBR, Inc. to notify them that the child has experienced
      an AIS around the time of birth.

      After receiving permission to release the patient's contact information, the CBR team will
      then relay the contact information to the Stem Cell Study Group at the University of Texas -
      Houston (UT) / Children's Memorial Hermann Hospital (CMHH) who will contact the family,
      explain the study, and send a consent form for their review.

      Several weeks prior to the scheduled procedure date, the patient will go to their family
      doctor for a physical evaluation and have blood drawn to rule out problems with the child's
      health.

      After successfully meeting initial pre-screening criteria, the patient's family will make
      their own arrangements to travel to Houston for the study procedures.

      Once the patient is in Houston, he/she will undergo baseline assessments, including physical,
      neurological exams and tests, speech therapy testing, laboratory tests, and imaging (MRI) of
      the brain. If any of the blood tests are abnormal, or the patient has an active infection,
      the procedure may be postponed or the patient may possibly be excluded from the study.

      If the patient continues to meet all criteria, the frozen cord blood will be shipped
      overnight to a special lab in Houston, TX for processing. Once release criteria have been
      met, the cells will be brought to the CMHH for infusion into the patient's vein. The patient
      will remain at CHMM for 24 hours of monitoring, after which they will be allowed to go home.
      Patients will be called daily by the research staff through Day 14, and once a month after
      that.

      Patients will return to Houston, Texas for 6 month, 1 year and 2 year follow-up visits for
      repeat assessments, tests, and exams just like at the baseline visit. The MRI of the brain
      will only be repeated at the 6 month visit.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Functional outcome measure
time frame: Two years
description: Physiological (e.g., SSEP, EEG) and anatomic outcome (MRI) changes will be evaluated by repeating assessments, clinical tests and imaging exams at the pre-treatment and follow-up visits.
measure: Speech Therapy Specific Neuropsychological outcome measures
time frame: Two years
description: Age appropriate Neurologic examinations and speech therapy specific standardized testing will be performed pre-treatment and at all follow-up visits.
arm group label: Biologic; Cord Blood Stem Cells; Intravenous injection
arm group type: Experimental
description: Autologous Human Umbilical Cord Blood derived Stem Cell injection
intervention type: Biological
intervention name: Autologous Human Cord Blood derived Stem Cell injection
description: One time intravenous (in the vein) injection with two year follow-up
arm group label: Biologic; Cord Blood Stem Cells; Intravenous injection
other name: Patient's own cord blood
other name: Patient's own stem cells
criteria: 
gender: All
minimum age: 6 Weeks
maximum age: 6 Years
healthy volunteers: No
last name: Timothy C. Foster, MD
role: Principal Investigator
affiliation: The University of Texas Health Science Center, Houston
last name: Linda S. Baumgartner, MS, CCC-SLP, LSLS, Cert.AVT
role: Principal Investigator
affiliation: Florida Hospital for Children - Orlando
last name: James E. Baumgartner, MD
role: Study Chair
affiliation: Florida Hospital for Children - Orlando
facility: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Memorial Hermann Health System
investigator full name: James E.Baumgartner, MD
investigator title: Clinical Associate Professor; Board-certified Pediatric Neurosurgeon
mesh term: Stroke
mesh term: Ischemic Stroke