<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/NCT02249923</url>
  </required_header>
  <id_info>
    <org_study_id>14-0018</org_study_id>
    <secondary_id>U01HL121518</secondary_id>
    <nct_id>NCT02249923</nct_id>
  </id_info>
  <brief_title>Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry</brief_title>
  <acronym>PPHNet</acronym>
  <official_title>Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Colorado, Denver</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Heart, Lung, and Blood Institute (NHLBI)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
    <collaborator>
      <agency>Children's Hospital Colorado</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>University of Colorado, Denver</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Patients are being asked to be in this research study because medical researchers hope that&#xD;
      by gathering information about a large number of children with pulmonary hypertension over&#xD;
      time, their understanding of the disease process will increase and lead to better treatment.&#xD;
      Investigators believe that pulmonary hypertension in children is different than pulmonary&#xD;
      hypertension in adults and this study will help us understand those differences.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Pulmonary Hypertension (PH) is a syndrome characterized by vasoconstriction and abnormal&#xD;
      growth and function of endothelial and smooth muscle cells and other components within the&#xD;
      pulmonary vessels, which leads to elevation of the pulmonary artery pressure. PH may be&#xD;
      idiopathic (primary) without any known cause. Some cases of PH are familial. PH may also be&#xD;
      secondary to a specific disease process such as portal hypertension, congenital heart&#xD;
      disease, chronic lung disease, thromboembolic disease, connective tissue disease, human&#xD;
      immunodeficiency virus (HIV), and use of anorexigens. Left untreated, PH is often progressive&#xD;
      and fatal. There is no cure for PH. Therapy focuses upon treatment of secondary causes if&#xD;
      present, and reduction of the pulmonary artery pressure through medical therapy. There have&#xD;
      been many new developments within the past few years in the management of patients with PH.&#xD;
      While there is no cure for PH early detection and treatment are important for survival of&#xD;
      patients. Limited data is available that describes the etiologies, clinical course and&#xD;
      prognosis of pediatric pulmonary hypertension.&#xD;
&#xD;
      Objectives&#xD;
&#xD;
      Aim 1: Clinical Research&#xD;
&#xD;
        1. To provide a mechanism to store information about newborns, infants and children with&#xD;
           PH;&#xD;
&#xD;
        2. To determine the incidence and natural history of the various etiologies of pediatric&#xD;
           PH;&#xD;
&#xD;
        3. To define the investigator current diagnostic and therapeutic approaches to the diverse&#xD;
           conditions associated with pediatric PH;&#xD;
&#xD;
        4. To determine the response of children with PH to chronic therapies.&#xD;
&#xD;
      Aim 2: Research Infrastructure To create a robust scalable data architecture, to combine&#xD;
      traditional registry data, electronic Health Record (EHR), and PRO (Patient Reported Outcome)&#xD;
      data in a single resource.&#xD;
&#xD;
      Aim 3: Informatics Address three classes of unanswered questions crucial for the&#xD;
      characterization and management of PH, comparing the information value of registry vs. EHR&#xD;
      vs. fused data across registry/EHR/PROs, in the domains of spectrum of PH comorbidities, PH&#xD;
      indicators and endpoints of morbidity and mortality, and response to therapies in PH.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date>October 2014</start_date>
  <completion_date type="Anticipated">December 2031</completion_date>
  <primary_completion_date type="Anticipated">December 2030</primary_completion_date>
  <study_type>Observational [Patient Registry]</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>
  <target_duration>5 Years</target_duration>
  <primary_outcome>
    <measure>Time to clinical worsening</measure>
    <time_frame>36 months</time_frame>
    <description>Death</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Escalation of Pulmonary Hypertension Therapy</measure>
    <time_frame>36 months</time_frame>
    <description>The addition of patients baseline medication therapy, this can include going from mono therapy to dual therapy, or dual therapy to triple therapy</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Right Heart Failure</measure>
    <time_frame>36 months</time_frame>
    <description>Elevated Right atrial pressure greater than 10 by right heart catheterization</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">1000</enrollment>
  <condition>Pulmonary Vascular Disease</condition>
  <condition>Pulmonary Arterial Hypertension</condition>
  <arm_group>
    <arm_group_label>Pulmonary Arterial Hypertension</arm_group_label>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Pulmonary Hypertension (PH) is a syndrome characterized by vasoconstriction and abnormal&#xD;
        growth and function of endothelial and smooth muscle cells and other components within the&#xD;
        pulmonary vessels, which leads to elevation of the pulmonary artery pressure. PH may be&#xD;
        idiopathic (primary) without any known cause. Some cases of PH are familial. PH may also be&#xD;
        secondary to a specific disease process such as portal hypertension, congenital heart&#xD;
        disease, chronic lung disease, thromboembolic disease, connective tissue disease, HIV, and&#xD;
        use of anorexigens.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  The subject's age of onset of pulmonary hypertension must be prior to age 18 years&#xD;
&#xD;
          -  The person providing consent must be able to read either Spanish or English.&#xD;
&#xD;
          -  The subject (and/or parent/legal guardian) must be able to provide informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosed with pulmonary hypertension after age 18&#xD;
&#xD;
          -  Refusal to sign informed consent&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Day</minimum_age>
    <maximum_age>21 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Steven H Abman, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Colorado</affiliation>
  </overall_official>
  <overall_official>
    <last_name>David D Ivy, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Colorado</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Kenneth D Mandl, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Boston Children's Hospital, Harvard School of Medicine</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Jeffrey Fineman, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University California San Francisco</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Jeffrey Feinstein, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Stanford University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Ian Adatia, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Alberta Edmonton</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Catherine Avitabile, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital of Philadelphia</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Mary Mullen, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Boston Children's Hospital</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Eric Austin, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Vanderbilt University Medical Center</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Marc Natter, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Boston Children's Hospital</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Erika Rosenzweig, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Columbia University</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Steven Abman, MD</last_name>
    <phone>303-881-9765</phone>
    <email>steven.abman@uanschutz.org</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Robin Mascotti</last_name>
    <phone>303-724-6513</phone>
    <email>robin.mascotti@childrenscolorado.org</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Stanford University Medical center</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jeffrey Feinstein, MD</last_name>
      <email>jeff.feinstein@stanford.edu</email>
    </contact>
    <investigator>
      <last_name>Jeffrey Feinstein, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University California San Francisco</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jeffrey Fineman, MD</last_name>
      <email>jeff.fineman@ucsf.edu</email>
    </contact>
    <investigator>
      <last_name>Jeffrey Fineman, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </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>
    <status>Completed</status>
  </location>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Mary Mullen, MD</last_name>
      <email>Mary.Mullen@cardio.chboston.org</email>
    </contact>
    <investigator>
      <last_name>Mary Mullen, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Columbia University Medical Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Erika Rosenzweig, MD</last_name>
      <email>esb14@columbia.edu</email>
    </contact>
    <investigator>
      <last_name>Erika Rosenzweig, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Catherine Avitabile, MD</last_name>
      <email>AvitabileC@email.CHOP.EDU</email>
    </contact>
    <investigator>
      <last_name>Brian Hanna, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Vanderbilt University Medical Center</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Eric Austin, MD</last_name>
      <email>eric.austin@Vanderbilt.edu</email>
    </contact>
    <investigator>
      <last_name>Eric Austin, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Alberta Edmonton</name>
      <address>
        <city>Edmonton</city>
        <state>Alberta</state>
        <country>Canada</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Ian Adatia, MD</last_name>
      <email>iadatia@ualberta.ca</email>
    </contact>
    <investigator>
      <last_name>Ian Adatia, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2022</verification_date>
  <study_first_submitted>September 10, 2014</study_first_submitted>
  <study_first_submitted_qc>September 23, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 26, 2014</study_first_posted>
  <last_update_submitted>August 10, 2022</last_update_submitted>
  <last_update_submitted_qc>August 10, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">August 12, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Pulmonary Arterial Hypertension (PAH)</keyword>
  <keyword>Pulmonary Vascular Disease (PVD)</keyword>
  <keyword>Registry</keyword>
  <keyword>Pediatric</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hypertension, Pulmonary</mesh_term>
    <mesh_term>Pulmonary Arterial Hypertension</mesh_term>
    <mesh_term>Hypertension</mesh_term>
    <mesh_term>Vascular Diseases</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/NCT02249923
org study id: 14-0018
secondary id: U01HL121518
nct id: NCT02249923
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
Patients are being asked to be in this research study because medical researchers hope that
      by gathering information about a large number of children with pulmonary hypertension over
      time, their understanding of the disease process will increase and lead to better treatment.
      Investigators believe that pulmonary hypertension in children is different than pulmonary
      hypertension in adults and this study will help us understand those differences.
Pulmonary Hypertension (PH) is a syndrome characterized by vasoconstriction and abnormal
      growth and function of endothelial and smooth muscle cells and other components within the
      pulmonary vessels, which leads to elevation of the pulmonary artery pressure. PH may be
      idiopathic (primary) without any known cause. Some cases of PH are familial. PH may also be
      secondary to a specific disease process such as portal hypertension, congenital heart
      disease, chronic lung disease, thromboembolic disease, connective tissue disease, human
      immunodeficiency virus (HIV), and use of anorexigens. Left untreated, PH is often progressive
      and fatal. There is no cure for PH. Therapy focuses upon treatment of secondary causes if
      present, and reduction of the pulmonary artery pressure through medical therapy. There have
      been many new developments within the past few years in the management of patients with PH.
      While there is no cure for PH early detection and treatment are important for survival of
      patients. Limited data is available that describes the etiologies, clinical course and
      prognosis of pediatric pulmonary hypertension.

      Objectives

      Aim 1: Clinical Research

        1. To provide a mechanism to store information about newborns, infants and children with
           PH;

        2. To determine the incidence and natural history of the various etiologies of pediatric
           PH;

        3. To define the investigator current diagnostic and therapeutic approaches to the diverse
           conditions associated with pediatric PH;

        4. To determine the response of children with PH to chronic therapies.

      Aim 2: Research Infrastructure To create a robust scalable data architecture, to combine
      traditional registry data, electronic Health Record (EHR), and PRO (Patient Reported Outcome)
      data in a single resource.

      Aim 3: Informatics Address three classes of unanswered questions crucial for the
      characterization and management of PH, comparing the information value of registry vs. EHR
      vs. fused data across registry/EHR/PROs, in the domains of spectrum of PH comorbidities, PH
      indicators and endpoints of morbidity and mortality, and response to therapies in PH.
observational model: Cohort
time perspective: Prospective
measure: Time to clinical worsening
time frame: 36 months
description: Death
measure: Escalation of Pulmonary Hypertension Therapy
time frame: 36 months
description: The addition of patients baseline medication therapy, this can include going from mono therapy to dual therapy, or dual therapy to triple therapy
measure: Right Heart Failure
time frame: 36 months
description: Elevated Right atrial pressure greater than 10 by right heart catheterization
arm group label: Pulmonary Arterial Hypertension
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 1 Day
maximum age: 21 Years
healthy volunteers: No
last name: Steven H Abman, MD
role: Principal Investigator
affiliation: Children's Hospital Colorado
last name: David D Ivy, MD
role: Principal Investigator
affiliation: Children's Hospital Colorado
last name: Kenneth D Mandl, MD
role: Principal Investigator
affiliation: Boston Children's Hospital, Harvard School of Medicine
last name: Jeffrey Fineman, MD
role: Principal Investigator
affiliation: University California San Francisco
last name: Jeffrey Feinstein, MD
role: Principal Investigator
affiliation: Stanford University
last name: Ian Adatia, MD
role: Principal Investigator
affiliation: University of Alberta Edmonton
last name: Catherine Avitabile, MD
role: Principal Investigator
affiliation: Children's Hospital of Philadelphia
last name: Mary Mullen, MD
role: Principal Investigator
affiliation: Boston Children's Hospital
last name: Eric Austin, MD
role: Principal Investigator
affiliation: Vanderbilt University Medical Center
last name: Marc Natter, MD
role: Principal Investigator
affiliation: Boston Children's Hospital
last name: Erika Rosenzweig, MD
role: Principal Investigator
affiliation: Columbia University
last name: Steven Abman, MD
phone: 303-881-9765
email: steven.abman@uanschutz.org
last name: Robin Mascotti
phone: 303-724-6513
email: robin.mascotti@childrenscolorado.org
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Completed
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
country: Canada
country: United States
responsible party type: Sponsor
mesh term: Hypertension, Pulmonary
mesh term: Pulmonary Arterial Hypertension
mesh term: Hypertension
mesh term: Vascular Diseases
sharing ipd: No