xml: <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/NCT05981092</url>
    </required_header>
    <id_info>
      <org_study_id>C4981001</org_study_id>
      <secondary_id>2022-000398-20</secondary_id>
      <nct_id>NCT05981092</nct_id>
    </id_info>
    <brief_title>A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease)</brief_title>
    <acronym>BAG3 DCM</acronym>
    <official_title>A Prospective Multinational Study of the Natural History of Participants With BAG3 Mutation Associated Dilated Cardiomyopathy</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Pfizer</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Pfizer</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>
        The purpose of this study is to learn about the natural progression of DCM (dilated&#xD;
        cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is&#xD;
        weakened and the heart becomes enlarged. This makes it hard for the heart to pump enough&#xD;
        blood for the body.&#xD;
  &#xD;
        The study is seeking up to about 35 participants who have:&#xD;
  &#xD;
          -  BAG3 mutation (change in the gene) that causes or is likely to cause dilated&#xD;
             cardiomyopathy&#xD;
  &#xD;
          -  NYHA (New York Heart Association) Class I-IV at screening (Stage B-D)&#xD;
  &#xD;
          -  Left Ventricular Ejection Fraction less than or equal to 50% (meaning reduced heart&#xD;
             function)&#xD;
  &#xD;
        All participants in this study will receive their usual treatment. The investigators will&#xD;
        observe the natural progression of people who have BAG3 DCM. This will help the investigators&#xD;
        better understand the disease and aid in future research.&#xD;
  &#xD;
        Participants will take part in this study for one year. During this time, participants will&#xD;
        visit the site at least 4 times (about every 3 months). Participants will undergo study&#xD;
        procedures and give information about their health. These procedures will include a physical&#xD;
        exam, cardiac magnetic resonance imaging, echocardiography, ECG monitoring, activity&#xD;
        monitoring, cardiopulmonary exercise testing, and blood tests. Participants will answer&#xD;
        questions about health and quality of life. The study team will also call participants about&#xD;
        1 time over the phone.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">October 14, 2022</start_date>
    <completion_date type="Anticipated">April 19, 2025</completion_date>
    <primary_completion_date type="Anticipated">April 19, 2025</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <primary_outcome>
      <measure>Determine baseline of cardiac structure and function in BAG3 associated DCM.</measure>
      <time_frame>Baseline</time_frame>
      <description>Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Determine changes over time in cardiac structure and function in BAG3 associated DCM.</measure>
      <time_frame>1 year</time_frame>
      <description>Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of one year follow up time.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Changes in high sensitivity troponin I (hsTNI) levels</measure>
      <time_frame>Baseline to 1 year</time_frame>
      <description>HsTNI will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNI to severity of clinical disease progression.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Changes in high sensitivity troponin T (hsTNT) levels</measure>
      <time_frame>Baseline to 1 year</time_frame>
      <description>HsTNT will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNT to severity of clinical disease progression.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Changes in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels</measure>
      <time_frame>Baseline to 1 year</time_frame>
      <description>NT-proBNP will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of NT-proBNP to severity of clinical disease progression.</description>
    </secondary_outcome>
    <number_of_groups>1</number_of_groups>
    <enrollment type="Anticipated">35</enrollment>
    <condition>Cardiomyopathy, Dilated</condition>
    <condition>Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)</condition>
    <arm_group>
      <arm_group_label>BAG3 DCM</arm_group_label>
      <description>A single arm observational trial where all participants will undergo the same schedule of assessments.</description>
    </arm_group>
    <eligibility>
      <study_pop>
        <textblock>
          primary care clinic and referrals from primary care clinic to investigational sites&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Key Inclusion Criteria:&#xD;
  &#xD;
            -  Documented BAG3 mutation that causes or is likely to cause dilated cardiomyopathy&#xD;
  &#xD;
            -  New York Heart Association (NYHA) Class I-IV at screening (Stage B-D)&#xD;
  &#xD;
            -  Left Ventricular Ejection Fraction ≤50% (i.e., Reduced Heart Function)&#xD;
  &#xD;
          Key Exclusion Criteria:&#xD;
  &#xD;
            -  Acute decompensated heart failure within 1 month prior to enrollment.(such as&#xD;
               hospitalization)&#xD;
  &#xD;
            -  Any of the following within 3 months prior to screening: myocardial infarction (MI),&#xD;
               cardiac surgical procedures (other than for pacemaker/ICD/CRT-defibrillator [CRT-D]&#xD;
               implantation), acute coronary syndrome, or hospitalization for cardiac arrhythmia.&#xD;
  &#xD;
            -  History of heart transplantation&#xD;
  &#xD;
            -  eGFR &lt;30 mL/min/1.73 m2 (significantly impaired kidney function)&#xD;
  &#xD;
            -  Noncardiac condition that limits lifespan to &lt;1 year.&#xD;
  &#xD;
            -  Presence of other form(s) of cardiomyopathy contributing to heart failure&#xD;
  &#xD;
            -  Previous administration with an investigational drug within 30 days (or as determined&#xD;
               by the local requirement).&#xD;
  &#xD;
            -  No more than 3 first-degree members of the same family who are already participating&#xD;
               in the study&#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>Pfizer CT.gov Call Center</last_name>
      <role>Study Director</role>
      <affiliation>Pfizer</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Pfizer CT.gov Call Center</last_name>
      <phone>1-800-718-1021</phone>
      <email>ClinicalTrials.gov_Inquiries@pfizer.com</email>
    </overall_contact>
    <location>
      <facility>
        <name>Brigham &amp; Women's Hospital</name>
        <address>
          <city>Boston</city>
          <state>Massachusetts</state>
          <zip>02115</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Brigham and Women's Hospital</name>
        <address>
          <city>Boston</city>
          <state>Massachusetts</state>
          <zip>02115</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Montefiore Medical Center(Hutchinson Metro Center)</name>
        <address>
          <city>Bronx</city>
          <state>New York</state>
          <zip>10461</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Montefiore Medical Center(Medical Arts Pavilion)</name>
        <address>
          <city>Bronx</city>
          <state>New York</state>
          <zip>10467</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Montefiore Medical Center</name>
        <address>
          <city>Bronx</city>
          <state>New York</state>
          <zip>10467</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Division of Heart Failure and Transplantation(Hospital of the University of Pennsylvania)</name>
        <address>
          <city>Philadelphia</city>
          <state>Pennsylvania</state>
          <zip>19104</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
    </location>
    <location>
      <facility>
        <name>Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy</name>
        <address>
          <city>Warszawa</city>
          <zip>04-628</zip>
          <country>Poland</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Hospital Universitario Puerta de Hierro Majadahonda</name>
        <address>
          <city>Majadahonda</city>
          <state>Madrid</state>
          <zip>28222</zip>
          <country>Spain</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Hospital Clinico Universitario Virgen de La Arrixaca</name>
        <address>
          <city>El Palmar</city>
          <state>Murcia</state>
          <zip>30120</zip>
          <country>Spain</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>CHUAC-Complejo Hospitalario Universitario A Coruña</name>
        <address>
          <city>A Coruna</city>
          <zip>15006</zip>
          <country>Spain</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location>
      <facility>
        <name>Hospital Universitario 12 de Octubre</name>
        <address>
          <city>Madrid</city>
          <zip>28041</zip>
          <country>Spain</country>
        </address>
      </facility>
      <status>Not yet recruiting</status>
    </location>
    <location>
      <facility>
        <name>The Royal Brompton and Harefield Hospitals</name>
        <address>
          <city>Harefield</city>
          <state>London</state>
          <zip>UB9 6JH</zip>
          <country>United Kingdom</country>
        </address>
      </facility>
      <status>Recruiting</status>
    </location>
    <location_countries>
      <country>Poland</country>
      <country>Spain</country>
      <country>United Kingdom</country>
      <country>United States</country>
    </location_countries>
    <link>
      <url>https://pmiform.com/clinical-trial-info-request?StudyID=C4981001</url>
      <description>To obtain contact information for a study center near you, click here.</description>
    </link>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>June 14, 2023</study_first_submitted>
    <study_first_submitted_qc>July 31, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 8, 2023</study_first_posted>
    <last_update_submitted>August 29, 2023</last_update_submitted>
    <last_update_submitted_qc>August 29, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 31, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>BAG3</keyword>
    <keyword>DCM</keyword>
    <keyword>cardiomyopathy</keyword>
    <keyword>dilated cardiomyopathy</keyword>
    <keyword>heritable heart disease</keyword>
    <keyword>heart disease</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Heart Diseases</mesh_term>
      <mesh_term>Cardiomyopathies</mesh_term>
      <mesh_term>Cardiomyopathy, Dilated</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
      <ipd_description>Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.</ipd_description>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05981092
  org study id: C4981001
  secondary id: 2022-000398-20
  nct id: NCT05981092
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  The purpose of this study is to learn about the natural progression of DCM (dilated
        cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is
        weakened and the heart becomes enlarged. This makes it hard for the heart to pump enough
        blood for the body.
  
        The study is seeking up to about 35 participants who have:
  
          -  BAG3 mutation (change in the gene) that causes or is likely to cause dilated
             cardiomyopathy
  
          -  NYHA (New York Heart Association) Class I-IV at screening (Stage B-D)
  
          -  Left Ventricular Ejection Fraction less than or equal to 50% (meaning reduced heart
             function)
  
        All participants in this study will receive their usual treatment. The investigators will
        observe the natural progression of people who have BAG3 DCM. This will help the investigators
        better understand the disease and aid in future research.
  
        Participants will take part in this study for one year. During this time, participants will
        visit the site at least 4 times (about every 3 months). Participants will undergo study
        procedures and give information about their health. These procedures will include a physical
        exam, cardiac magnetic resonance imaging, echocardiography, ECG monitoring, activity
        monitoring, cardiopulmonary exercise testing, and blood tests. Participants will answer
        questions about health and quality of life. The study team will also call participants about
        1 time over the phone.
  measure: Determine baseline of cardiac structure and function in BAG3 associated DCM.
  time frame: Baseline
  description: Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.
  measure: Determine changes over time in cardiac structure and function in BAG3 associated DCM.
  time frame: 1 year
  description: Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of one year follow up time.
  measure: Changes in high sensitivity troponin I (hsTNI) levels
  time frame: Baseline to 1 year
  description: HsTNI will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNI to severity of clinical disease progression.
  measure: Changes in high sensitivity troponin T (hsTNT) levels
  time frame: Baseline to 1 year
  description: HsTNT will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNT to severity of clinical disease progression.
  measure: Changes in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels
  time frame: Baseline to 1 year
  description: NT-proBNP will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of NT-proBNP to severity of clinical disease progression.
  arm group label: BAG3 DCM
  description: A single arm observational trial where all participants will undergo the same schedule of assessments.
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Pfizer CT.gov Call Center
  role: Study Director
  affiliation: Pfizer
  last name: Pfizer CT.gov Call Center
  phone: 1-800-718-1021
  email: ClinicalTrials.gov_Inquiries@pfizer.com
  facility: 
  status: Recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Not yet recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Recruiting
  facility: 
  status: Not yet recruiting
  facility: 
  status: Recruiting
  country: Poland
  country: Spain
  country: United Kingdom
  country: United States
  url: https://pmiform.com/clinical-trial-info-request?StudyID=C4981001
  description: To obtain contact information for a study center near you, click here.
  responsible party type: Sponsor
  mesh term: Heart Diseases
  mesh term: Cardiomyopathies
  mesh term: Cardiomyopathy, Dilated
  sharing ipd: No
  ipd description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.