<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/NCT04424030</url>
  </required_header>
  <id_info>
    <org_study_id>56001</org_study_id>
    <secondary_id>SSU00099737</secondary_id>
    <nct_id>NCT04424030</nct_id>
  </id_info>
  <brief_title>International Consortium for Multimodality Phenotyping in Adults With Non-compaction</brief_title>
  <acronym>NONCOMPACT</acronym>
  <official_title>International Consortium for Multimodality Phenotyping in Adults With Non-compaction</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Stanford University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>The Cleveland Clinic</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>University of Pennsylvania</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Erasmus Medical Center</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Seoul National University Hospital</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Stanford University</source>
  <oversight_info>
    <has_dmc>No</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>
      Non-compaction cardiomyopathy (NCCM) is a heterogeneous, poorly understood disorder&#xD;
      characterized by a prominent inner layer of loose myocardial tissue, and associated with&#xD;
      heart failure, stroke, severe rhythm irregularities and death. For a growing population&#xD;
      diagnosed with NCCM there is a need for better risk stratification to appropriately allocate&#xD;
      (or safely withhold) these impactful preventive measures. The goal of this international&#xD;
      consortium is to improve care of patients with non-compaction cardiomyopathy. We hypothesize&#xD;
      that comprehensive analysis of clinical, genetic, structural and functional information will&#xD;
      improve risk stratification. In addition, we hypothesize that detailed structural analysis&#xD;
      will allow for differentiation of pathological and benign patterns of non-compaction. In a&#xD;
      large cohort of adult patients with suspected NCCM we will perform in-depth phenotyping,&#xD;
      including clinical information, pedigree data, genetics, echocardiography and MRI, and follow&#xD;
      patients for up to 3 years. We will apply machine-learning based analytics to develop&#xD;
      predictive models and compare their performance to currently used models and treatment&#xD;
      criteria. Secondly, in a subset of patients we will perform high-resolution cardiac CT for&#xD;
      detailed structural characterization of the myocardial wall. We will investigate associations&#xD;
      between myocardial structure and regional contractile function, as assessed by echo and MRI.&#xD;
      The aim of this proposal is to identify a structural signature associated with pathological&#xD;
      non-compaction and improve developed risk prediction models. Discovery of pathological&#xD;
      structural signatures through innovative imaging techniques, in relation to myocardial&#xD;
      contractility, will advance our understanding of NCCM.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">January 1, 2021</start_date>
  <completion_date type="Anticipated">April 1, 2025</completion_date>
  <primary_completion_date type="Anticipated">April 1, 2025</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>
  <primary_outcome>
    <measure>Incidence of hard embolic adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>Clinical neuro/systemic embolic event by autopsy, imaging or specialist evaluation</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Incidence of hard arrhythmic adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>Sudden death (aborted), appropriate ICD discharge or VT/VF on ECG or rhythm/device monitoring</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Incidence of hard heart failure related adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>Heart failure death, cardiac transplantation or mechanical circulatory support</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of composite of hard adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>Hard embolic, arrhythmic and heart failure related adverse events (as described above)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of all embolic adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>Hard embolic adverse events or transient neurologic event without objective infarction</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of all arrhythmic adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>Hard arrhythmic adverse events or syncope without recorded arrhythmia</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of all heart failure related adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>Hard heart failure related adverse events or resynchronization therapy, heart failure hospital admission</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of composite of adverse events</measure>
    <time_frame>Up to 4 years after enrollment</time_frame>
    <description>All embolic, arrhythmic or heart failure related adverse events (as described above)</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">600</enrollment>
  <condition>Non-Compaction Cardiomyopathy</condition>
  <arm_group>
    <arm_group_label>Multimodality imaging</arm_group_label>
    <description>Patients who have undergone echocardiography and cardiac MRI as part of their clinical management A research cardiac CT scan will be performed in eligible participants</description>
  </arm_group>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>Echocardiography</intervention_name>
    <description>Standard echocardiography exam performed as part of clinical management.</description>
    <arm_group_label>Multimodality imaging</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>Magnetic resonance imaging (MRI)</intervention_name>
    <description>Standard comprehensive cardiac MRI exam of the heart performed as part of clinical management.</description>
    <arm_group_label>Multimodality imaging</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>Computed tomography (CT)</intervention_name>
    <description>ECG-triggered, contrast-enhanced CT scan of the heart performed for research purposes in eligible study participants.</description>
    <arm_group_label>Multimodality imaging</arm_group_label>
  </intervention>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      After enrollment, blood will be drawn via venipuncture. The blood samples (35cc in total)&#xD;
      will be centrifuged (15 minutes at 3500 RPM), after which the plasma, serum, red cells and&#xD;
      buffy coat will be aliquoted from the vacutainers into color-coded vials. In addition to&#xD;
      standard EDTA tubes we may include a CPT or PAX tube for more extensive analytics, or an&#xD;
      Oragene tube for remote saliva collection. The aliquots and tubes will be stored at -70⁰C.&#xD;
      The samples will remain at the site until analysis.&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with hypertrabeculation of the left ventricle fulfilling echo-based criteria of&#xD;
        non-compaction cardiomyopathy&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  ≥18 years old&#xD;
&#xD;
          -  Hypertrabeculation of the left ventricle fulfilling the echo-based Jenni criteria of&#xD;
             NCCM&#xD;
&#xD;
          -  Clinical cardiac MRI examination performed or planned&#xD;
&#xD;
        Exclusion Criteria (general cohort):&#xD;
&#xD;
          -  Complex congenital disease (including transposition great arteries, tetralogy of&#xD;
             Fallot, tricuspid atresia, truncus arteriosis, single ventricle, hypoplastic left&#xD;
             heart, pulmonary atresia, double-outlet RV), neuromuscular disorders or isolated RV&#xD;
             non-compaction&#xD;
&#xD;
          -  Inability to provide informed consent&#xD;
&#xD;
          -  Contra-indications to MRI, which apply if the clinical cardiac MRI has not yet been&#xD;
             performed at the time of study enrollment: permanent pacemakers/ICDs, MRI contrast&#xD;
             medium allergy, significant arrhythmia with highly irregular RR intervals, severe&#xD;
             dyspnea with inability to lay flat/breath hold, inability to communicate with the MRI&#xD;
             technician or follow commands for any reason (psychosis, agitation, etc.), other&#xD;
             site-specific contra-indications to clinical MRI of the heart.&#xD;
&#xD;
        Exclusion Criteria (cardiac CT examination):&#xD;
&#xD;
          -  Age &lt;21 years&#xD;
&#xD;
          -  Decompensated heart failure, or otherwise clinically unstable&#xD;
&#xD;
          -  BMI&gt;40 kg/m2&#xD;
&#xD;
          -  Pregnancy (or cannot be ruled out)&#xD;
&#xD;
          -  Known iodine contrast medium allergy&#xD;
&#xD;
          -  Kidney dysfunction: eGFR&lt;45 ml/min&#xD;
&#xD;
          -  Thyroid disease: toxic multinodular goiter, Graves' disease, Hashimoto's thyroiditis&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Koen Nieman, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Stanford University</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Koen Nieman, MD, PhD</last_name>
    <phone>6507237476</phone>
    <email>knieman@stanford.edu</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Katie DeSutter, BS</last_name>
    <phone>6507254151</phone>
    <email>ksutter@stanford.edu</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Stanford University</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Matthew Wheeler, MD</last_name>
      <phone>650-725-7622</phone>
      <email>wheelerm@stanford.edu</email>
    </contact>
    <contact_backup>
      <last_name>Koen Nieman, MD</last_name>
      <phone>6507237476</phone>
      <email>knieman@stanford.edu</email>
    </contact_backup>
  </location>
  <location>
    <facility>
      <name>Cleveland Clinic Foundation</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Wilson Tang, MD PhD</last_name>
    </contact>
    <investigator>
      <last_name>Deborah Kwon, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Pennsylvania</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Anjali Owens, MD</last_name>
    </contact>
    <investigator>
      <last_name>Harold Litt, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Seoul National University Hospital</name>
      <address>
        <city>Seoul</city>
        <zip>03080</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Seung-Pyo Lee, MD</last_name>
    </contact>
    <investigator>
      <last_name>Whal Lee, MD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Erasmus Medical Center</name>
      <address>
        <city>Rotterdam</city>
        <zip>3015GD</zip>
        <country>Netherlands</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Kadir Caliskan, MD PhD</last_name>
    </contact>
    <investigator>
      <last_name>Ricardo Budde, MD, PhD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
    <investigator>
      <last_name>Alexander Hirsch, MD, PhD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Korea, Republic of</country>
    <country>Netherlands</country>
    <country>United States</country>
  </location_countries>
  <verification_date>April 2023</verification_date>
  <study_first_submitted>June 5, 2020</study_first_submitted>
  <study_first_submitted_qc>June 5, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 9, 2020</study_first_posted>
  <last_update_submitted>April 11, 2023</last_update_submitted>
  <last_update_submitted_qc>April 11, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">April 13, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Stanford University</investigator_affiliation>
    <investigator_full_name>Koen Nieman</investigator_full_name>
    <investigator_title>Associate Professor of Medicine</investigator_title>
  </responsible_party>
  <keyword>Echocardiography</keyword>
  <keyword>Magnetic resonance imaging</keyword>
  <keyword>Computed tomography</keyword>
  <keyword>Prognosis</keyword>
  <keyword>Genetics</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cardiomyopathies</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>De-identified datasets will be created and maintained on a public website in a password-protected area, where the frequency of use of the datasets can be monitored by Stanford and NHLBI.</ipd_description>
  </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/NCT04424030
org study id: 56001
secondary id: SSU00099737
nct id: NCT04424030
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Non-compaction cardiomyopathy (NCCM) is a heterogeneous, poorly understood disorder
      characterized by a prominent inner layer of loose myocardial tissue, and associated with
      heart failure, stroke, severe rhythm irregularities and death. For a growing population
      diagnosed with NCCM there is a need for better risk stratification to appropriately allocate
      (or safely withhold) these impactful preventive measures. The goal of this international
      consortium is to improve care of patients with non-compaction cardiomyopathy. We hypothesize
      that comprehensive analysis of clinical, genetic, structural and functional information will
      improve risk stratification. In addition, we hypothesize that detailed structural analysis
      will allow for differentiation of pathological and benign patterns of non-compaction. In a
      large cohort of adult patients with suspected NCCM we will perform in-depth phenotyping,
      including clinical information, pedigree data, genetics, echocardiography and MRI, and follow
      patients for up to 3 years. We will apply machine-learning based analytics to develop
      predictive models and compare their performance to currently used models and treatment
      criteria. Secondly, in a subset of patients we will perform high-resolution cardiac CT for
      detailed structural characterization of the myocardial wall. We will investigate associations
      between myocardial structure and regional contractile function, as assessed by echo and MRI.
      The aim of this proposal is to identify a structural signature associated with pathological
      non-compaction and improve developed risk prediction models. Discovery of pathological
      structural signatures through innovative imaging techniques, in relation to myocardial
      contractility, will advance our understanding of NCCM.
observational model: Cohort
time perspective: Prospective
measure: Incidence of hard embolic adverse events
time frame: Up to 4 years after enrollment
description: Clinical neuro/systemic embolic event by autopsy, imaging or specialist evaluation
measure: Incidence of hard arrhythmic adverse events
time frame: Up to 4 years after enrollment
description: Sudden death (aborted), appropriate ICD discharge or VT/VF on ECG or rhythm/device monitoring
measure: Incidence of hard heart failure related adverse events
time frame: Up to 4 years after enrollment
description: Heart failure death, cardiac transplantation or mechanical circulatory support
measure: Incidence of composite of hard adverse events
time frame: Up to 4 years after enrollment
description: Hard embolic, arrhythmic and heart failure related adverse events (as described above)
measure: Incidence of all embolic adverse events
time frame: Up to 4 years after enrollment
description: Hard embolic adverse events or transient neurologic event without objective infarction
measure: Incidence of all arrhythmic adverse events
time frame: Up to 4 years after enrollment
description: Hard arrhythmic adverse events or syncope without recorded arrhythmia
measure: Incidence of all heart failure related adverse events
time frame: Up to 4 years after enrollment
description: Hard heart failure related adverse events or resynchronization therapy, heart failure hospital admission
measure: Incidence of composite of adverse events
time frame: Up to 4 years after enrollment
description: All embolic, arrhythmic or heart failure related adverse events (as described above)
arm group label: Multimodality imaging
description: Patients who have undergone echocardiography and cardiac MRI as part of their clinical management A research cardiac CT scan will be performed in eligible participants
intervention type: Diagnostic Test
intervention name: Echocardiography
description: Standard echocardiography exam performed as part of clinical management.
arm group label: Multimodality imaging
intervention type: Diagnostic Test
intervention name: Magnetic resonance imaging (MRI)
description: Standard comprehensive cardiac MRI exam of the heart performed as part of clinical management.
arm group label: Multimodality imaging
intervention type: Diagnostic Test
intervention name: Computed tomography (CT)
description: ECG-triggered, contrast-enhanced CT scan of the heart performed for research purposes in eligible study participants.
arm group label: Multimodality imaging
After enrollment, blood will be drawn via venipuncture. The blood samples (35cc in total)
      will be centrifuged (15 minutes at 3500 RPM), after which the plasma, serum, red cells and
      buffy coat will be aliquoted from the vacutainers into color-coded vials. In addition to
      standard EDTA tubes we may include a CPT or PAX tube for more extensive analytics, or an
      Oragene tube for remote saliva collection. The aliquots and tubes will be stored at -70⁰C.
      The samples will remain at the site until analysis.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Koen Nieman, MD, PhD
role: Principal Investigator
affiliation: Stanford University
last name: Koen Nieman, MD, PhD
phone: 6507237476
email: knieman@stanford.edu
last name: Katie DeSutter, BS
phone: 6507254151
email: ksutter@stanford.edu
facility: 
status: Recruiting
contact: 
contact backup: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
investigator: 
country: Korea, Republic of
country: Netherlands
country: United States
responsible party type: Principal Investigator
investigator affiliation: Stanford University
investigator full name: Koen Nieman
investigator title: Associate Professor of Medicine
mesh term: Cardiomyopathies
sharing ipd: Yes
ipd description: De-identified datasets will be created and maintained on a public website in a password-protected area, where the frequency of use of the datasets can be monitored by Stanford and NHLBI.