<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/NCT03779711</url>
  </required_header>
  <id_info>
    <org_study_id>18-004753</org_study_id>
    <nct_id>NCT03779711</nct_id>
  </id_info>
  <brief_title>Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Repair of Hypoplastic Left Heart Syndrome</brief_title>
  <official_title>Phase IIb Study of Intramyocardial Injection of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Stage II Surgical Repair of Right Ventricular Dependent Variants of Hypoplastic Left Heart Syndrome (AutoCell-S2)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Timothy J Nelson, MD, PhD</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>University of Oklahoma</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospital of Philadelphia</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospital Los Angeles</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospital Colorado</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospitals and Clinics of Minnesota</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Ochsner Health System</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's of Alabama</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospital Medical Center, Cincinnati</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>ReGen Theranostics, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Researchers want to better understand what happens to the heart when the stem cells are&#xD;
      injected directly into the muscle of the right side of the heart during the Stage II&#xD;
      palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS)&#xD;
      or HLHS variant. Researchers want to see if there are changes in the heart's&#xD;
      structure/function following this stem cell-based therapy and compared to children that have&#xD;
      not had cell-based therapy.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a Phase IIb trial to determine if the delivery of the autologous UCB-MNC product into&#xD;
      the myocardium of the right ventricle of the heart at the time of Stage II surgical repair&#xD;
      will provide an improvement in cardiac function, reaching growth and developmental&#xD;
      milestones, and quality of life, while also providing a reduction in the cumulative days of&#xD;
      hospitalization following Stage II surgical repair. Long-term improvement in cardiac&#xD;
      function, reaching growth and developmental milestones, reaching Stage III surgical repair&#xD;
      pre-op work-up, prolonging time to cardiac transplantation or death, and improving quality of&#xD;
      life will also be determined.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">June 6, 2019</start_date>
  <completion_date type="Anticipated">February 2026</completion_date>
  <primary_completion_date type="Actual">May 22, 2022</primary_completion_date>
  <phase>Phase 2</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>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Right Ventricular Cardiac Function measured by apical fractional area change (FAC)</measure>
    <time_frame>baseline, 3 months post-Stage II surgery</time_frame>
    <description>Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Right Ventricular Cardiac Function measured by circumferential strain.</measure>
    <time_frame>baseline, 3 months post-Stage II surgery</time_frame>
    <description>Right ventricular cardiac function as measured by echocardiogram that includes studies for circumferential strain. Measured by percent.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Right Ventricular Cardiac Function measured by longitudinal strain.</measure>
    <time_frame>baseline, 3 months post-Stage II surgery</time_frame>
    <description>Right ventricular cardiac function as measured by echocardiogram that includes studies for longitudinal strain. Measured by percent.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Right Ventricular Ejection Fraction</measure>
    <time_frame>baseline, 12 months post-Stage II surgery</time_frame>
    <description>Right ventricular ejection fraction as measured by echocardiogram. Ejection Fraction is calculated by the volume of blood in the heart during systole and diastole. It is the percentage of the volume in the ventricle ejected during a cardiac cycle. Measured by percent. The normal ejection fraction is 55 to 75 percent.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in ejection fraction</measure>
    <time_frame>baseline pre-op, hospital discharge from Stage II surgery (an average of 1-6 weeks)</time_frame>
    <description>Ejection fraction as measured by echocardiogram. Ejection Fraction is calculated by the volume of blood in the heart during systole and diastole. It is the percentage of the volume in the ventricle ejected during a cardiac cycle. Measured by percent. The normal ejection fraction is 55 to 75 percent.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cumulative days of hospitalization</measure>
    <time_frame>1, 3-months post Stage II surgery</time_frame>
    <description>Cumulative days of hospitalization per patient following discharge for the Stage II surgical repair.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in weight</measure>
    <time_frame>3-months post Stage II surgery</time_frame>
    <description>Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in heart rate</measure>
    <time_frame>3-months post Stage II surgery</time_frame>
    <description>Heart rate as measured at home by subject's caregiver as standard of care per subject's provider. Caregivers will be instructed by the site staff on the best way to perform.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in oxygen saturation</measure>
    <time_frame>3-months post Stage II surgery</time_frame>
    <description>Oxygen saturation as measured at home by subject's caregiver using the pulse oximetry device provided as standard of care by subject's provider. Caregivers will be instructed by the site to use the same pulse oximetry device for every home measurement of oxygen saturation.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in weight</measure>
    <time_frame>baseline, every 6 months post-Stage II surgery</time_frame>
    <description>Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in heart failure medication</measure>
    <time_frame>baseline, every 6 months post-Stage II surgery</time_frame>
    <description>Changes in heart failure medications will be recorded by caregiver on the concomitant medication diary throughout the subject's participation in the study starting at the time of consent.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in arrhythmia medication</measure>
    <time_frame>baseline, every 6 months post-Stage II surgery</time_frame>
    <description>Changes in arrhythmia medication will be recorded by subject's caregiver on the concomitant medication diary throughout the subject's participation in the study starting at the time of consent.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Stage III surgical repair</measure>
    <time_frame>Stage III surgery pre-op, approx. 4 years</time_frame>
    <description>The number of research subjects eligible for Stage III surgical repair</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Total time until listed for cardiac transplantation</measure>
    <time_frame>up to Stage III surgery pre-op, approx. 4 years</time_frame>
    <description>The number of days between Stage II surgery and listed on cardiac transplantation list.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in right ventricular function</measure>
    <time_frame>baseline, Stage III surgery pre-op</time_frame>
    <description>Right ventricular ejection fraction as measured by echocardiogram, and cardiac catheterization. Ejection Fraction is calculated by the volume of blood in the heart during systole and diastole. It is the percentage of the volume in the ventricle ejected during a cardiac cycle. Measured by percent. The normal ejection fraction is 55 to 75 percent.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Total time until death</measure>
    <time_frame>approx 4 years</time_frame>
    <description>The number of days between Stage II surgery and death occurrence</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">95</enrollment>
  <condition>Hypoplastic Left Heart Syndrome</condition>
  <arm_group>
    <arm_group_label>Treatment</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Autologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 1-3 million cells per kilogram body weight . This is a one time treatment at the time of Stage II Glenn surgery .</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Control</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Clinical data will be collected before, during and after the Stage II surgical standard of care procedure. Requires additional imaging studies at 3 months that are not standard of care in addition to some blood tests that would be beyond standard of care. Information will be collected to document the clinical outcomes and will be compared with the information from the group receiving the investigational treatment used in this study.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Autologous (self) mononuclear cells derived from umbilical cord blood</intervention_name>
    <description>The investigational product will be delivered into the right myocardium via sub-epicardial injections of 0.1 mL per kg body weight to achieve the target dose of 1-3 million TNC per kg body weight.at the time of Stage II surgical repair.</description>
    <arm_group_label>Treatment</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>Stage II Surgical repair</intervention_name>
    <description>This operation usually is performed about six months after Stage I surgery to divert half of the blood to the lungs when circulation through the lungs no longer needs as much pressure from the ventricle. The shunt to the pulmonary arteries is disconnected and the right pulmonary artery is connected directly to the superior vena cava, the vein that brings deoxygenated blood from the upper part of the body to the heart. This sends half of the deoxygenated blood directly to the lungs without going through the ventricle.</description>
    <arm_group_label>Control</arm_group_label>
    <arm_group_label>Treatment</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of HLHS or HLHS variant with single right ventricular dependent CHD having&#xD;
             undergone Stage I surgical repair and undergoing Stage II surgical repair.&#xD;
&#xD;
          -  Less than 13 months of age at time of Stage II surgical repair&#xD;
&#xD;
          -  Previous participation in the UCB collection protocol with autologous UCB-MNC product&#xD;
             that is acceptable for use (treatment arm only)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  History of DMSO reaction (treatment arm only).&#xD;
&#xD;
          -  Parent(s) and/or legal guardian(s) unwilling to have their child participate or&#xD;
             unwilling to follow the study procedures.&#xD;
&#xD;
          -  Severe chronic diseases at the discretion of the treating physician.&#xD;
&#xD;
          -  Extensive extra-cardiac syndromic features.&#xD;
&#xD;
          -  Known history of cancer.&#xD;
&#xD;
          -  Any of the following complications of his/her congenital heart disease:&#xD;
&#xD;
               1. Any condition requiring urgent, or unplanned interventional procedure within 15&#xD;
                  days prior to Stage II surgical repair, unless complete and full cardiac recovery&#xD;
                  is documented by site investigator&#xD;
&#xD;
               2. Severe pulmonary hypertension (reported in the medical record as &gt;70% systemic&#xD;
                  pressure)&#xD;
&#xD;
               3. Other clinical concerns as documented by a site investigator that would predict&#xD;
                  (more likely to happen than not to happen) a risk of severe complications or very&#xD;
                  poor outcome, not directly related to the stem cell product or its injection&#xD;
                  procedure, during or after Stage II surgical repair.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>12 Months</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Frank Cetta, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Mayo Clinic</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Harold M. Burkhart, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Oklahoma University Medical Center</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Joseph W. Rossano, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital of Philadelphia</affiliation>
  </overall_official>
  <overall_official>
    <last_name>David M. Overman, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Minnesota</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Ram Kumar Subramanyan, M.D., Ph.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Los Angeles</affiliation>
  </overall_official>
  <overall_official>
    <last_name>James Jaggers, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Colorado</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Benjamin Peeler, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Ochsner Health System</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Waldemar Carlo, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Alabama at Birmingham</affiliation>
  </overall_official>
  <overall_official>
    <last_name>James Tweddell, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Medical Center, Cincinnati</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's of Alabama</name>
      <address>
        <city>Birmingham</city>
        <state>Alabama</state>
        <zip>35233</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Los Angeles</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90027</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ocshner Medical Center</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70121</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospitals of Minnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55404</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital and Medical Center</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45529</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oklahoma University Medical Center</name>
      <address>
        <city>Oklahoma City</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.mayo.edu/research/clinical-trials</url>
    <description>Mayo Clinic Clinical Trials</description>
  </link>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>December 4, 2018</study_first_submitted>
  <study_first_submitted_qc>December 15, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 19, 2018</study_first_posted>
  <last_update_submitted>May 5, 2023</last_update_submitted>
  <last_update_submitted_qc>May 5, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 9, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>ReGen Theranostics, Inc.</investigator_affiliation>
    <investigator_full_name>Timothy J Nelson, MD, PhD</investigator_full_name>
    <investigator_title>Program Director</investigator_title>
  </responsible_party>
  <keyword>Hypoplastic Left Heart Syndrome</keyword>
  <keyword>HLHS</keyword>
  <keyword>Congenital Heart Disease</keyword>
  <keyword>Umbilical Cord Blood</keyword>
  <keyword>UCB</keyword>
  <keyword>Cord blood</keyword>
  <keyword>Stem cells</keyword>
  <keyword>Regenerative therapy</keyword>
  <keyword>Stage II Glenn</keyword>
  <keyword>Glenn Surgery</keyword>
  <keyword>single ventricle</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hypoplastic Left Heart Syndrome</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</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/NCT03779711
org study id: 18-004753
nct id: NCT03779711
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
Researchers want to better understand what happens to the heart when the stem cells are
      injected directly into the muscle of the right side of the heart during the Stage II
      palliative surgery for single ventricle patients with hypoplastic left heart syndrome (HLHS)
      or HLHS variant. Researchers want to see if there are changes in the heart's
      structure/function following this stem cell-based therapy and compared to children that have
      not had cell-based therapy.
This is a Phase IIb trial to determine if the delivery of the autologous UCB-MNC product into
      the myocardium of the right ventricle of the heart at the time of Stage II surgical repair
      will provide an improvement in cardiac function, reaching growth and developmental
      milestones, and quality of life, while also providing a reduction in the cumulative days of
      hospitalization following Stage II surgical repair. Long-term improvement in cardiac
      function, reaching growth and developmental milestones, reaching Stage III surgical repair
      pre-op work-up, prolonging time to cardiac transplantation or death, and improving quality of
      life will also be determined.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change in Right Ventricular Cardiac Function measured by apical fractional area change (FAC)
time frame: baseline, 3 months post-Stage II surgery
description: Right ventricular cardiac function as measured by echocardiogram that includes studies for apical fractional area change (FAC). Measured by percent.
measure: Change in Right Ventricular Cardiac Function measured by circumferential strain.
time frame: baseline, 3 months post-Stage II surgery
description: Right ventricular cardiac function as measured by echocardiogram that includes studies for circumferential strain. Measured by percent.
measure: Change in Right Ventricular Cardiac Function measured by longitudinal strain.
time frame: baseline, 3 months post-Stage II surgery
description: Right ventricular cardiac function as measured by echocardiogram that includes studies for longitudinal strain. Measured by percent.
measure: Change in Right Ventricular Ejection Fraction
time frame: baseline, 12 months post-Stage II surgery
description: Right ventricular ejection fraction as measured by echocardiogram. Ejection Fraction is calculated by the volume of blood in the heart during systole and diastole. It is the percentage of the volume in the ventricle ejected during a cardiac cycle. Measured by percent. The normal ejection fraction is 55 to 75 percent.
measure: Change in ejection fraction
time frame: baseline pre-op, hospital discharge from Stage II surgery (an average of 1-6 weeks)
description: Ejection fraction as measured by echocardiogram. Ejection Fraction is calculated by the volume of blood in the heart during systole and diastole. It is the percentage of the volume in the ventricle ejected during a cardiac cycle. Measured by percent. The normal ejection fraction is 55 to 75 percent.
measure: Cumulative days of hospitalization
time frame: 1, 3-months post Stage II surgery
description: Cumulative days of hospitalization per patient following discharge for the Stage II surgical repair.
measure: Change in weight
time frame: 3-months post Stage II surgery
description: Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.
measure: Change in heart rate
time frame: 3-months post Stage II surgery
description: Heart rate as measured at home by subject's caregiver as standard of care per subject's provider. Caregivers will be instructed by the site staff on the best way to perform.
measure: Change in oxygen saturation
time frame: 3-months post Stage II surgery
description: Oxygen saturation as measured at home by subject's caregiver using the pulse oximetry device provided as standard of care by subject's provider. Caregivers will be instructed by the site to use the same pulse oximetry device for every home measurement of oxygen saturation.
measure: Change in weight
time frame: baseline, every 6 months post-Stage II surgery
description: Weight as measured by scale provided as part of standard of care by subject's provider. Measured at home by subject's caregiver using the same scale for every home measurement of body weight. Caregivers will be instructed by the site to use the same scale for every home measurement of body weight. Scales will vary based on the institution's standard name/type used.
measure: Change in heart failure medication
time frame: baseline, every 6 months post-Stage II surgery
description: Changes in heart failure medications will be recorded by caregiver on the concomitant medication diary throughout the subject's participation in the study starting at the time of consent.
measure: Change in arrhythmia medication
time frame: baseline, every 6 months post-Stage II surgery
description: Changes in arrhythmia medication will be recorded by subject's caregiver on the concomitant medication diary throughout the subject's participation in the study starting at the time of consent.
measure: Stage III surgical repair
time frame: Stage III surgery pre-op, approx. 4 years
description: The number of research subjects eligible for Stage III surgical repair
measure: Total time until listed for cardiac transplantation
time frame: up to Stage III surgery pre-op, approx. 4 years
description: The number of days between Stage II surgery and listed on cardiac transplantation list.
measure: Change in right ventricular function
time frame: baseline, Stage III surgery pre-op
description: Right ventricular ejection fraction as measured by echocardiogram, and cardiac catheterization. Ejection Fraction is calculated by the volume of blood in the heart during systole and diastole. It is the percentage of the volume in the ventricle ejected during a cardiac cycle. Measured by percent. The normal ejection fraction is 55 to 75 percent.
measure: Total time until death
time frame: approx 4 years
description: The number of days between Stage II surgery and death occurrence
arm group label: Treatment
arm group type: Experimental
description: Autologous (self) mononuclear cells derived from umbilical cord blood and that meet all release criteria are injected into the surface of the right heart muscle to achieve the target dose of 1-3 million cells per kilogram body weight . This is a one time treatment at the time of Stage II Glenn surgery .
arm group label: Control
arm group type: Active Comparator
description: Clinical data will be collected before, during and after the Stage II surgical standard of care procedure. Requires additional imaging studies at 3 months that are not standard of care in addition to some blood tests that would be beyond standard of care. Information will be collected to document the clinical outcomes and will be compared with the information from the group receiving the investigational treatment used in this study.
intervention type: Biological
intervention name: Autologous (self) mononuclear cells derived from umbilical cord blood
description: The investigational product will be delivered into the right myocardium via sub-epicardial injections of 0.1 mL per kg body weight to achieve the target dose of 1-3 million TNC per kg body weight.at the time of Stage II surgical repair.
arm group label: Treatment
intervention type: Procedure
intervention name: Stage II Surgical repair
description: This operation usually is performed about six months after Stage I surgery to divert half of the blood to the lungs when circulation through the lungs no longer needs as much pressure from the ventricle. The shunt to the pulmonary arteries is disconnected and the right pulmonary artery is connected directly to the superior vena cava, the vein that brings deoxygenated blood from the upper part of the body to the heart. This sends half of the deoxygenated blood directly to the lungs without going through the ventricle.
arm group label: Control
arm group label: Treatment
criteria: 
gender: All
minimum age: N/A
maximum age: 12 Months
healthy volunteers: No
last name: Frank Cetta, MD
role: Principal Investigator
affiliation: Mayo Clinic
last name: Harold M. Burkhart, M.D.
role: Principal Investigator
affiliation: Children's Hospital Oklahoma University Medical Center
last name: Joseph W. Rossano, M.D.
role: Principal Investigator
affiliation: Children's Hospital of Philadelphia
last name: David M. Overman, M.D.
role: Principal Investigator
affiliation: Children's Minnesota
last name: Ram Kumar Subramanyan, M.D., Ph.D.
role: Principal Investigator
affiliation: Children's Hospital Los Angeles
last name: James Jaggers, M.D.
role: Principal Investigator
affiliation: Children's Hospital Colorado
last name: Benjamin Peeler, M.D.
role: Principal Investigator
affiliation: Ochsner Health System
last name: Waldemar Carlo, M.D.
role: Principal Investigator
affiliation: University of Alabama at Birmingham
last name: James Tweddell, M.D.
role: Principal Investigator
affiliation: Children's Hospital Medical Center, Cincinnati
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
url: https://www.mayo.edu/research/clinical-trials
description: Mayo Clinic Clinical Trials
responsible party type: Sponsor-Investigator
investigator affiliation: ReGen Theranostics, Inc.
investigator full name: Timothy J Nelson, MD, PhD
investigator title: Program Director
mesh term: Hypoplastic Left Heart Syndrome
mesh term: Syndrome
sharing ipd: No