<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/NCT01708863</url>
  </required_header>
  <id_info>
    <org_study_id>12-002887</org_study_id>
    <nct_id>NCT01708863</nct_id>
  </id_info>
  <brief_title>A Prospective Study of Patients With Hypoplastic Left Heart Syndrome (HLHS) Following Stage II Surgical Palliation</brief_title>
  <official_title>A Prospective Study of Patients With Hypoplastic Left Heart Syndrome (HLHS) Following Stage II Surgical Palliation</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>Mayo Clinic</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>ReGen Theranostics, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Hypoplastic left heart syndrome (HLHS) is a severe form of congenital heart disease that&#xD;
      consists of multiple obstructions to flow through the left heart and aorta, as well as&#xD;
      hypoplasia of the left ventricle. Most patients require a three-stage surgical protocol&#xD;
      starting within days of birth. Stage I of this process is the Norwood reconstruction (within&#xD;
      the first few days of life), Stage II (usually required within 3-8 months) involves creation&#xD;
      of a direct connection between the patient's superior vena cava and the pulmonary arterial&#xD;
      confluence (bidirectional Glenn anastomosis), and the last stage is creation of a Fontan&#xD;
      circulation (typically within the first 2-4 years). This &quot;single ventricle&quot; approach requires&#xD;
      the right ventricle to perform as the only circulatory pump for the entire body.&#xD;
&#xD;
      Our long-term goal is to develop regenerative strategies to strengthen and augment the right&#xD;
      ventricular muscle of the single-ventricle heart following surgical palliation in HLHS&#xD;
      patients. To determine the safety and feasibility of a cell-based therapeutic intervention at&#xD;
      the Stage II surgery, we aim to document the natural history of post-surgical care in HLHS&#xD;
      patients having undergone standard of care with protocol specific follow-up over the course&#xD;
      of a 6-month period.&#xD;
&#xD;
      This prospective study will document the natural history in patients with HLHS after planned&#xD;
      Stage II surgical palliation with a focus on cardiovascular parameters within 6 months&#xD;
      following surgery in 10 patients.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 2012</start_date>
  <completion_date type="Actual">May 15, 2020</completion_date>
  <primary_completion_date type="Actual">May 2020</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">4</enrollment>
  <condition>Hypoplastic Left Heart Syndrome</condition>
  <arm_group>
    <arm_group_label>Children with Hypoplastic Left Heart Syndrome (HLHS)</arm_group_label>
    <description>HLHS patients requiring Stage II Glenn surgery</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Children with HLHS up to 18 months of age requiring a planned Stage II bi-directional Glenn&#xD;
        surgery&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Individuals with HLHS having undergone Stage I surgical palliation and undergoing&#xD;
             planned Stage II palliative Glenn surgery.&#xD;
&#xD;
          -  Individuals up to 18 months of age are eligible if written informed consent can be&#xD;
             obtained from both parents and/or legal guardians, unless one parent is not reasonably&#xD;
             available.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Severe chronic diseases, extensive extra-cardiac syndromic features, or history of&#xD;
             cancer&#xD;
&#xD;
        The following complications of congenital heart disease:&#xD;
&#xD;
          -  Any condition requiring urgent, or unplanned procedure within 15 days prior to Stage&#xD;
             II surgical repair.&#xD;
&#xD;
          -  Severe pulmonary hypertension (reported in the medical record as &gt;70% systemic&#xD;
             pressure).&#xD;
&#xD;
          -  Other clinical concerns as documented by a site investigator that would predict (more&#xD;
             likely to happen than not to happen) a risk of severe complications or very poor&#xD;
             outcome during or after Stage II surgical repair.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>0 Months</minimum_age>
    <maximum_age>18 Months</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Timothy J Nelson, MD, PhD</last_name>
    <role>Study Director</role>
    <affiliation>Mayo Clinic</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Muhammad Y Qureshi, MBBS</last_name>
    <role>Principal Investigator</role>
    <affiliation>Mayo Clinic</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55901</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oklahoma University Children's Hospital</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>October 2020</verification_date>
  <study_first_submitted>October 15, 2012</study_first_submitted>
  <study_first_submitted_qc>October 16, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 17, 2012</study_first_posted>
  <last_update_submitted>October 2, 2020</last_update_submitted>
  <last_update_submitted_qc>October 2, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">October 5, 2020</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>Stage II palliative surgery</keyword>
  <keyword>Glenn procedure</keyword>
  <keyword>Congenital heart defect</keyword>
  <keyword>Congenital heart disease</keyword>
  <keyword>Underdeveloped left 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>
  <!-- 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/NCT01708863
org study id: 12-002887
nct id: NCT01708863
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
Hypoplastic left heart syndrome (HLHS) is a severe form of congenital heart disease that
      consists of multiple obstructions to flow through the left heart and aorta, as well as
      hypoplasia of the left ventricle. Most patients require a three-stage surgical protocol
      starting within days of birth. Stage I of this process is the Norwood reconstruction (within
      the first few days of life), Stage II (usually required within 3-8 months) involves creation
      of a direct connection between the patient's superior vena cava and the pulmonary arterial
      confluence (bidirectional Glenn anastomosis), and the last stage is creation of a Fontan
      circulation (typically within the first 2-4 years). This "single ventricle" approach requires
      the right ventricle to perform as the only circulatory pump for the entire body.

      Our long-term goal is to develop regenerative strategies to strengthen and augment the right
      ventricular muscle of the single-ventricle heart following surgical palliation in HLHS
      patients. To determine the safety and feasibility of a cell-based therapeutic intervention at
      the Stage II surgery, we aim to document the natural history of post-surgical care in HLHS
      patients having undergone standard of care with protocol specific follow-up over the course
      of a 6-month period.

      This prospective study will document the natural history in patients with HLHS after planned
      Stage II surgical palliation with a focus on cardiovascular parameters within 6 months
      following surgery in 10 patients.
observational model: Cohort
time perspective: Prospective
arm group label: Children with Hypoplastic Left Heart Syndrome (HLHS)
description: HLHS patients requiring Stage II Glenn surgery
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 0 Months
maximum age: 18 Months
healthy volunteers: No
last name: Timothy J Nelson, MD, PhD
role: Study Director
affiliation: Mayo Clinic
last name: Muhammad Y Qureshi, MBBS
role: Principal Investigator
affiliation: Mayo Clinic
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