<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/NCT03230591</url>
  </required_header>
  <id_info>
    <org_study_id>4-2017-0471</org_study_id>
    <nct_id>NCT03230591</nct_id>
  </id_info>
  <brief_title>Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy</brief_title>
  <official_title>Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy (ACTION-Fabry Trial)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Yonsei University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Yonsei University</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Study Design: This is an observational study. No treatment or intervention will be assigned&#xD;
      to the subjects. All patients will receive full standard of care concomitant medication for&#xD;
      the treatment of their cardiac condition. 25 patients with genetically confirmed&#xD;
      Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D&#xD;
      strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and&#xD;
      after 1 year of treatment with ERT with Agalsidase Alfa for follow-up.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      1. Objectives - The purpose of this study is to evaluate the impact of ERT with Agalsidase&#xD;
           Alfa on LV diastolic function and flow in patients with Fabry's cardiomyopathy using LV&#xD;
           2D strain, diastolic stress echocardiography, LV vortex flow and CMR.&#xD;
&#xD;
        2. Primary / Secondary Endpoint 1) Primary endpoint:&#xD;
&#xD;
             -  Change from baseline in peak exercise E/E' by diastolic stress echocardiography,&#xD;
                global longitudinal strain and LV vortex flow parameters at 1 year 2) Secondary&#xD;
                endpoints:&#xD;
&#xD;
             -  Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow&#xD;
                up&#xD;
&#xD;
                  -  Changes from baseline in evaluation of the degree of the resting LV diastolic&#xD;
                     function&#xD;
&#xD;
                       -  Changes from baseline in other echo-parameters; LV mass index, reduction&#xD;
                          of peak exercise E/E prime at 1 year follow up&#xD;
&#xD;
                            -  Changes from baseline in quality of life using questionnaire ⑤&#xD;
                               Change from baseline in peak VO2, exercise time, AT by diastolic&#xD;
                               stress echocardiography at 1 year follow up ⑥ Change in T1 baseline&#xD;
                               (myo, ms) &amp; T1 baseline (blood, ms), T1 postcontrast (myo, ms) &amp; T1&#xD;
                               baseline (blood, ms) by CMR&#xD;
&#xD;
        3. Study Methods 1) Study Design: This is an observational study. No treatment or&#xD;
           intervention will be assigned to the subjects. All patients will receive full standard&#xD;
           of care concomitant medication for the treatment of their cardiac condition. 25 patients&#xD;
           with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with&#xD;
           Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex&#xD;
           flow analysis, and CMR at baseline and after 1 year of treatment with ERT with&#xD;
           Agalsidase Alfa for follow-up.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">July 12, 2017</start_date>
  <completion_date type="Anticipated">January 2025</completion_date>
  <primary_completion_date type="Anticipated">January 2025</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>peak exercise E/E' by diastolic stress echocardiography</measure>
    <time_frame>1 year</time_frame>
    <description>Change from baseline in peak exercise E/E' by diastolic stress echocardiography</description>
  </primary_outcome>
  <primary_outcome>
    <measure>global longitudinal strain</measure>
    <time_frame>1 year</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>extracellular volume by CMR</measure>
    <time_frame>1 year</time_frame>
    <description>Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow up</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>evaluation of the degree of the resting LV diastolic function</measure>
    <time_frame>1 year</time_frame>
    <description>Changes from baseline in evaluation of the degree of the resting LV diastolic function</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>quality of life using questionnaire</measure>
    <time_frame>1 year</time_frame>
    <description>Changes from baseline in quality of life using questionnaire</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">25</enrollment>
  <condition>Fabry Disease</condition>
  <arm_group>
    <arm_group_label>Fabry's disease</arm_group_label>
    <description>Fabry's disease patients who were confirmed by enzyme assay and gene study</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Echocardiography</intervention_name>
    <description>LV vortex flow in Echocardiography</description>
    <arm_group_label>Fabry's disease</arm_group_label>
  </intervention>
  <biospec_retention>Samples Without DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      10cc blood&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients aged 16 ~ 75 years with Fabry disease confirmed by enzyme assay and gene test&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients aged 16~ 75 years with Fabry disease confirmed by enzyme assay and gene test&#xD;
&#xD;
          -  Patients who have LVH in 2D echocardiography (end diastolic septum and posterior wall&#xD;
             thickness ≥ 12mm) or Patients who present with cardiac changes (indicative of disease&#xD;
             progression such as decreased global longitudinal strain on 2D strain echocardiography&#xD;
             or low native T1 mapping on CMR)&#xD;
&#xD;
          -  Patients provided written informed consent to participate in this study&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Contraindication for enzyme replacement treatment with Agalsidase Alfa&#xD;
&#xD;
          -  Patients who cannot receive supine bicycle stress echocardiography, contrast&#xD;
             echocardiography or CMR&#xD;
&#xD;
          -  Patients with hemodynamically significant valvular heart disease or arrhythmias&#xD;
&#xD;
          -  Patients who have history of acute myocardial infarction or congestive heart failure&#xD;
             with reduced LV ejection fraction of less than 35%&#xD;
&#xD;
          -  Patients who had any cerebrovascular accident in the prior 6 months&#xD;
&#xD;
          -  Scheduled or planned surgery in the next 6 months&#xD;
&#xD;
          -  Patients with chronic liver cirrhosis&#xD;
&#xD;
          -  Patients who are allergic to contrast agent (e.g. Definity�, Lantheus Medical Imaging,&#xD;
             North Billerica, MA, USA)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>16 Years</minimum_age>
    <maximum_age>75 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Geu-Ru Hong, MD, Ph.D</last_name>
    <phone>82-2-2228-8443</phone>
    <email>grhong@yuhs.ac</email>
  </overall_contact>
  <location>
    <facility>
      <name>Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine</name>
      <address>
        <city>Seoul</city>
        <zip>03722</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Geu-Ru Hong, MD, PhD</last_name>
      <phone>82-2-2228-8443</phone>
      <email>GRHONG@yuhs.ac</email>
    </contact>
  </location>
  <location_countries>
    <country>Korea, Republic of</country>
  </location_countries>
  <verification_date>June 2021</verification_date>
  <study_first_submitted>July 21, 2017</study_first_submitted>
  <study_first_submitted_qc>July 23, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">July 26, 2017</study_first_posted>
  <last_update_submitted>June 23, 2021</last_update_submitted>
  <last_update_submitted_qc>June 23, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">June 24, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Fabry's disease</keyword>
  <keyword>Agalsidase Alfa</keyword>
  <keyword>Cardiomyopathy</keyword>
  <keyword>Enzyme replacement therapy</keyword>
  <keyword>Diastolic stress echocardiography</keyword>
  <keyword>LV vortex flow</keyword>
  <keyword>Cardiac MRI</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fabry Disease</mesh_term>
    <mesh_term>Cardiomyopathies</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</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/NCT03230591
org study id: 4-2017-0471
nct id: NCT03230591
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
Study Design: This is an observational study. No treatment or intervention will be assigned
      to the subjects. All patients will receive full standard of care concomitant medication for
      the treatment of their cardiac condition. 25 patients with genetically confirmed
      Anderson-Fabry disease who have a plan to start ERT with Agalsidase Alfa will undergo 2D
      strain, diastolic stress echocardiography, LV vortex flow analysis, and CMR at baseline and
      after 1 year of treatment with ERT with Agalsidase Alfa for follow-up.
1. Objectives - The purpose of this study is to evaluate the impact of ERT with Agalsidase
           Alfa on LV diastolic function and flow in patients with Fabry's cardiomyopathy using LV
           2D strain, diastolic stress echocardiography, LV vortex flow and CMR.

        2. Primary / Secondary Endpoint 1) Primary endpoint:

             -  Change from baseline in peak exercise E/E' by diastolic stress echocardiography,
                global longitudinal strain and LV vortex flow parameters at 1 year 2) Secondary
                endpoints:

             -  Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow
                up

                  -  Changes from baseline in evaluation of the degree of the resting LV diastolic
                     function

                       -  Changes from baseline in other echo-parameters; LV mass index, reduction
                          of peak exercise E/E prime at 1 year follow up

                            -  Changes from baseline in quality of life using questionnaire ⑤
                               Change from baseline in peak VO2, exercise time, AT by diastolic
                               stress echocardiography at 1 year follow up ⑥ Change in T1 baseline
                               (myo, ms) & T1 baseline (blood, ms), T1 postcontrast (myo, ms) & T1
                               baseline (blood, ms) by CMR

        3. Study Methods 1) Study Design: This is an observational study. No treatment or
           intervention will be assigned to the subjects. All patients will receive full standard
           of care concomitant medication for the treatment of their cardiac condition. 25 patients
           with genetically confirmed Anderson-Fabry disease who have a plan to start ERT with
           Agalsidase Alfa will undergo 2D strain, diastolic stress echocardiography, LV vortex
           flow analysis, and CMR at baseline and after 1 year of treatment with ERT with
           Agalsidase Alfa for follow-up.
observational model: Case-Only
time perspective: Prospective
measure: peak exercise E/E' by diastolic stress echocardiography
time frame: 1 year
description: Change from baseline in peak exercise E/E' by diastolic stress echocardiography
measure: global longitudinal strain
time frame: 1 year
measure: extracellular volume by CMR
time frame: 1 year
description: Changes from baseline in extracellular volume by CMR (T1 mapping) at 1 year follow up
measure: evaluation of the degree of the resting LV diastolic function
time frame: 1 year
description: Changes from baseline in evaluation of the degree of the resting LV diastolic function
measure: quality of life using questionnaire
time frame: 1 year
description: Changes from baseline in quality of life using questionnaire
arm group label: Fabry's disease
description: Fabry's disease patients who were confirmed by enzyme assay and gene study
intervention type: Other
intervention name: Echocardiography
description: LV vortex flow in Echocardiography
arm group label: Fabry's disease
10cc blood
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 16 Years
maximum age: 75 Years
healthy volunteers: No
last name: Geu-Ru Hong, MD, Ph.D
phone: 82-2-2228-8443
email: grhong@yuhs.ac
facility: 
status: Recruiting
contact: 
country: Korea, Republic of
responsible party type: Sponsor
mesh term: Fabry Disease
mesh term: Cardiomyopathies
sharing ipd: Undecided