<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/NCT04476901</url>
  </required_header>
  <id_info>
    <org_study_id>20200566</org_study_id>
    <secondary_id>CDMRP-PR191597</secondary_id>
    <secondary_id>20-02-134</secondary_id>
    <nct_id>NCT04476901</nct_id>
  </id_info>
  <brief_title>Transendocardial Injection of Allogeneic-MSC in Patients With Non-Ischemic Dilated Cardiomyopathy</brief_title>
  <acronym>DCMII</acronym>
  <official_title>A Phase IIB Randomized, Placebo-Controlled, Multicenter Study of the Comparative Efficacy and Safety of Transendocardial Injection of Allogeneic-MSC Versus Placebo in Patients With Non- Ischemic Dilated Cardiomyopathy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Joshua M Hare</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>United States Department of Defense</agency>
      <agency_class>U.S. Fed</agency_class>
    </collaborator>
    <collaborator>
      <agency>The University of Texas Health Science Center, Houston</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>University of Miami</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</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 evaluate the safety and effectiveness of an experimental drug&#xD;
      called human allogeneic mesenchymal stem cell therapy.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">May 7, 2021</start_date>
  <completion_date type="Anticipated">December 2024</completion_date>
  <primary_completion_date type="Anticipated">December 2024</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in LVEF</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in Left Ventricular Ejection Fraction (LVEF) as assessed via cardiac Magnetic Resonance Imaging (MRI)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in global ventricular strain</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in global ventricular strain as assessed via cardiac Harmonic Phase (HARP) MRI</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in left regional strain</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in regional ventricular strain as assessed via cardiac HARP MRI</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Left ventricular function concordance</measure>
    <time_frame>12 months</time_frame>
    <description>The left ventricular function concordance will be measured as the Number of individuals who experienced an increase in left ventricular ejection fraction (LVEF) and a simultaneous decrease in both left ventricular end systolic volume index (LVESVI) and left ventricular end diastolic volume index (LVEDVI)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in LVEDVI</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in left ventricular end diastolic index (LVEDVI) as assessed via cardiac MRI</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in LVESVI</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in left ventricular end systolic index (LVESVI) as assessed via cardiac MRI</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Maximal oxygen consumption (peak VO2)</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in maximal oxygen consumption (peak VO2) as assessed via treadmill</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Exercise tolerance</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in exercise tolerance as assessed as the distance covered via the six-minute walk test</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Minnesota Living with Heart Failure Questionnaire (MLHFQ) Score</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Minnesota Living with Heart Failure Questionnaire (MLHFQ) is a 21-item questionnaire with a total score ranging from 0 to 105 with lower scores indicative of better outcome.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in New York Heart Association (NYHA) Class</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>NYHA Classifications of heart failure are as follows: Class I (no limitations); Class II (mild symptoms); Class III (marked limitations); Class IV (Severe limitations)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percent change in flow mediated diameter</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in endothelial function will be reported as the percent change in flow mediated diameter assessed via flow mediated dilation (FMD).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in EPC-CFU</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in endothelial function will be reported as the change in Endothelial Progenitor Cell Colony Forming Unit (EPC-CFU) assessed via blood sample assay</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in NT-proBNP as assessed via blooddraw</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in cytokines</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Change in NT-proBNP as assessed via blooddraw</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of MACE</measure>
    <time_frame>12 months</time_frame>
    <description>Safety will be reported as the incidence of Major Adverse Cardiac Events (MACE) assessed by treating physician</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of TE-SAEs</measure>
    <time_frame>Day 30</time_frame>
    <description>Safety will be reported as the incidence of Treatment Emergent Serious Adverse Events (TE-SAEs) assessed by treating physician</description>
  </secondary_outcome>
  <number_of_arms>6</number_of_arms>
  <enrollment type="Anticipated">136</enrollment>
  <condition>Non-ischemic Dilated Cardiomyopathy</condition>
  <arm_group>
    <arm_group_label>Genotype A administered with placebo Group</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Participants whose blood genotyping resulted with Genotype A (absence of any variant/ presence of benign variant) and randomized to the placebo group will receive the placebo intervention.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Genotype A administered with hMSC Group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants whose blood genotyping resulted with Genotype A (absence of any variant/ presence of benign variant) and randomized to the treatment group will receive the hMSC intervention.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Genotype B administered with placebo Group</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the placebo group will receive the placebo intervention.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Genotype B administered with hMSC Group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the treatment group will receive the hMSC intervention.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Genotype C administered with placebo Group</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the placebo group will receive the placebo intervention.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Genotype C administered with hMSC Group</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the treatment group will receive the hMSC intervention.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>allogeneic human mesenchymal stem cells (hMSCs)</intervention_name>
    <description>allo-hMSCs, 16-20 million cells/ml delivered at a dose of 0.5 ml/ injection x 10 injections for a total of 80-100 million allo-hMSCs</description>
    <arm_group_label>Genotype A administered with hMSC Group</arm_group_label>
    <arm_group_label>Genotype B administered with hMSC Group</arm_group_label>
    <arm_group_label>Genotype C administered with hMSC Group</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo will be administered as injections of plasmalyte A supplemented with 1% of 25% human serum albumin (HSA). 0.5 ml/ injection x 10 injections</description>
    <arm_group_label>Genotype A administered with placebo Group</arm_group_label>
    <arm_group_label>Genotype B administered with placebo Group</arm_group_label>
    <arm_group_label>Genotype C administered with placebo Group</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        In order to be eligible to participate in this study, an individual must meet all of the&#xD;
        following criteria:&#xD;
&#xD;
          1. Men and women aged 18 to 80 years (inclusive) at the time of signing the informed&#xD;
             consent form.&#xD;
&#xD;
          2. Diagnosis of NIDCM with left ventricular ejection fraction ≤45%.&#xD;
&#xD;
          3. Appropriate guideline-directed optimal medical therapy for non-ischemic&#xD;
             cardiomyopathy. At a minimum, subjects must be on beta blockers and&#xD;
             angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs)&#xD;
             or Angiotensin Receptor Neprilysin Inhibitors (ARNI) or have appropriate medical&#xD;
             indication precluding use of one or both of these agents. Subjects must be on a stable&#xD;
             regimen for at least 30 days prior to the procedure. Dose titration is allowed.&#xD;
&#xD;
          4. Be a candidate for cardiac catheterization.&#xD;
&#xD;
          5. Be willing to undergo DNA test.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        An individual who meets any of the following criteria will be excluded from participation&#xD;
        in this study:&#xD;
&#xD;
          1. Be eligible for or require standard-of-care surgical or percutaneous intervention for&#xD;
             the treatment of non-ischemic dilated cardiomyopathy&#xD;
&#xD;
          2. Clinical manifestation of coronary artery disease (CAD) (e.g., chest pain and&#xD;
             concomitant clinical findings such as electrocardiogram changes suggestive of coronary&#xD;
             ischemia, myocardial infarction) or evidence of endocardial or transmural scar on&#xD;
             cardiac MRI suggestive of undiagnosed CAD or history of percutaneous coronary&#xD;
             intervention (PCI) or coronary artery bypass surgery (CABG). Be indicated for or&#xD;
             require coronary artery revascularization&#xD;
&#xD;
          3. Documented presence of epicardial stenosis of 70% or greater in one or more major&#xD;
             epicardial coronary arteries&#xD;
&#xD;
          4. Valvular heart disease including 1) aortic valve prosthesis, mechanical mitral valve,&#xD;
             and mitral valve clip; 2) severe aortic valve insufficiency/regurgitation within 12&#xD;
             months of consent&#xD;
&#xD;
          5. Aortic stenosis with valve area ≤ 1.5cm2&#xD;
&#xD;
          6. Cardiomyopathy due to acute Post-partum (within 6 months), Non-compaction, or&#xD;
             Hypertrophic cardiomyopathy&#xD;
&#xD;
          7. Cardiomyopathy due to known toxin (e.g amyloid) Note: anthracycline induced&#xD;
             cardiomyopathy will be allowed&#xD;
&#xD;
          8. QTc interval &gt; 550 ms on baseline electrocardiogram (ECG) (note: QTc interval is the&#xD;
             interval between the start of the Q wave and the end of the T wave in the heart's&#xD;
             electrical cycle)&#xD;
&#xD;
          9. Automated Implantable Cardioverter Defibrillator (AICD) appropriate firing or anti&#xD;
             tachycardia pacing for ventricular tachycardia or ventricular fibrillation within 30&#xD;
             days prior to consent&#xD;
&#xD;
         10. Have an estimated baseline glomerular filtration rate below the clinical site's&#xD;
             institutional cutoff&#xD;
&#xD;
         11. A hematologic abnormality during baseline testing as evidenced by hemoglobin &lt; 9 g/dl;&#xD;
             hematocrit &lt; 30%; absolute neutrophil count &lt; 2,000 or total WBC count more than 2&#xD;
             times upper limit of normal; or platelet values &lt; 100,000/ul&#xD;
&#xD;
         12. Have liver dysfunction, as evidenced by enzymes Aspartate Transaminase Enzyme (AST)&#xD;
             and Alanine Aminotransferase Enzyme (ALT) greater than three times the ULN&#xD;
&#xD;
         13. Have a bleeding diathesis or coagulopathy (International Normalised Ratio (INR) &gt;&#xD;
             1.5), cannot be withdrawn from anticoagulation therapy, or will refuse blood&#xD;
             transfusions&#xD;
&#xD;
         14. Be a solid organ transplant recipient. This does not include prior cell based therapy&#xD;
             (&gt;12 months prior to enrollment), bone, skin, ligament, tendon or corneal grafting.&#xD;
&#xD;
         15. Have a history of organ or cell transplant rejection&#xD;
&#xD;
         16. Have a clinical history of malignancy within the past 12 months (i.e., subjects with&#xD;
             prior malignancy must be disease free for 12 months), except curatively treated basal&#xD;
             cell or squamous cell carcinoma or cervical carcinoma&#xD;
&#xD;
         17. Drug and/or alcohol abuse or dependence within the past 9 months&#xD;
&#xD;
         18. Be serum positive for HIV, hepatitis B surface antigen, or viremic hepatitis C&#xD;
&#xD;
         19. Documented presence of a known Left Ventricular (LV) thrombus, aortic dissection, or&#xD;
             aortic aneurysm. (Refer to &quot;Guidance to the PI&quot; section with regards to LV thrombus,&#xD;
             below)&#xD;
&#xD;
         20. Blood glucose levels (HbA1c) &gt;10%&#xD;
&#xD;
         21. Severe radiographic contrast allergy&#xD;
&#xD;
         22. Known history of anaphylactic reaction to penicillin or streptomycin&#xD;
&#xD;
         23. Hypersensitivity to dimethyl sulfoxide (DMSO)&#xD;
&#xD;
         24. Non-cardiac condition with life expectancy &lt; 1 year&#xD;
&#xD;
         25. Acute stroke or transient ischemic attack within 3 months of enrollment&#xD;
&#xD;
         26. Be pregnant, nursing, or of childbearing potential while not practicing effective&#xD;
             contraceptive methods&#xD;
&#xD;
         27. Pacemaker-dependence with an Implantable Cardioverter Defibrillator (ICD) (Note:&#xD;
             pacemaker-dependent candidates without an ICD are not excluded)&#xD;
&#xD;
         28. Presence of a pacemaker and/or ICD generator with any of the following&#xD;
             limitations/conditions:&#xD;
&#xD;
               -  manufactured before the year 2000&#xD;
&#xD;
               -  leads implanted &lt; 6 weeks prior to consent&#xD;
&#xD;
               -  non-transvenous epicardial or abandoned leads&#xD;
&#xD;
               -  subcutaneous ICDs&#xD;
&#xD;
               -  leadless pacemakers&#xD;
&#xD;
         29. A cardiac resynchronization therapy (CRT) device implanted less than 3 months prior to&#xD;
             consent&#xD;
&#xD;
         30. Other MRI contraindications (e.g. subject body habitus incompatible with MRI)&#xD;
&#xD;
         31. Need for advanced heart failure therapy (e.g. IV inotropes)&#xD;
&#xD;
         32. Be currently participating (or participated within the previous 30 days) in an&#xD;
             investigational therapeutic or device trial&#xD;
&#xD;
         33. Any other condition that in the judgment of the Investigator would be a&#xD;
             contraindication to enrollment or follow-up&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Joshua Hare, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Miami</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Shelly L Sayre, MPH</last_name>
    <phone>713-500-9529</phone>
    <email>Shelly.L.Sayre@uth.tmc.edu</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Lina Caceres</last_name>
    <phone>305-243-5399</phone>
    <email>lvc25@med.miami.edu</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Stanford University</name>
      <address>
        <city>Stanford</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Fouzia Khan, MBBS</last_name>
      <phone>650-736-1410</phone>
      <email>fouziak@stanford.edu</email>
    </contact>
    <contact_backup>
      <last_name>Ashwini Narayana</last_name>
      <email>ashwinil@stanford.edu</email>
    </contact_backup>
    <investigator>
      <last_name>Phil Yang, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Miami Miller School of Medicine</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33136</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Lina Caceres</last_name>
      <phone>305-243-5399</phone>
      <email>lvc25@med.miami.edu</email>
    </contact>
    <contact_backup>
      <last_name>Jairo Tovar</last_name>
      <phone>305-243-5399</phone>
      <email>jat243@med.miami.edu</email>
    </contact_backup>
    <investigator>
      <last_name>Josh Hare, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Louisville</name>
      <address>
        <city>Louisville</city>
        <state>Kentucky</state>
        <zip>40202</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Heidi Wilson</last_name>
      <phone>502-540-3721</phone>
      <email>heidi.wilson@louisville.edu</email>
    </contact>
    <contact_backup>
      <last_name>Julie Caswell</last_name>
      <phone>502-587-4177</phone>
      <email>julie.caswell@louisville.edu</email>
    </contact_backup>
    <investigator>
      <last_name>Roberto Bolli, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Texas Heart Institute</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Nichole Piece</last_name>
      <phone>832-355-9173</phone>
      <email>npiece@texasheart.org</email>
    </contact>
    <contact_backup>
      <last_name>Sylvia Carranza</last_name>
      <phone>832-355-8524</phone>
      <email>SCarranza@texasheart.org</email>
    </contact_backup>
    <investigator>
      <last_name>Emerson Perin, MD, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.cctrn.org</url>
    <description>Cardiovascular Cell Therapy Research Network</description>
  </link>
  <link>
    <url>https://www.nih.gov/about-nih/what-we-do/nih-turning-discovery-into-health/stem-cells</url>
    <description>Information on stem cells from the National Institutes of Health</description>
  </link>
  <link>
    <url>http://www.dcmii.org</url>
    <description>DCM II Trial</description>
  </link>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>July 15, 2020</study_first_submitted>
  <study_first_submitted_qc>July 15, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 20, 2020</study_first_posted>
  <last_update_submitted>May 11, 2023</last_update_submitted>
  <last_update_submitted_qc>May 11, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 12, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>University of Miami</investigator_affiliation>
    <investigator_full_name>Joshua M Hare</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <keyword>Allogeneic mesenchymal stem cells</keyword>
  <keyword>Bone marrow-derived mesenchymal stem cells</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cardiomyopathies</mesh_term>
    <mesh_term>Cardiomyopathy, Dilated</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/NCT04476901
org study id: 20200566
secondary id: CDMRP-PR191597
secondary id: 20-02-134
nct id: NCT04476901
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to evaluate the safety and effectiveness of an experimental drug
      called human allogeneic mesenchymal stem cell therapy.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Change in LVEF
time frame: Baseline, 12 months
description: Change in Left Ventricular Ejection Fraction (LVEF) as assessed via cardiac Magnetic Resonance Imaging (MRI)
measure: Change in global ventricular strain
time frame: Baseline, 12 months
description: Change in global ventricular strain as assessed via cardiac Harmonic Phase (HARP) MRI
measure: Change in left regional strain
time frame: Baseline, 12 months
description: Change in regional ventricular strain as assessed via cardiac HARP MRI
measure: Left ventricular function concordance
time frame: 12 months
description: The left ventricular function concordance will be measured as the Number of individuals who experienced an increase in left ventricular ejection fraction (LVEF) and a simultaneous decrease in both left ventricular end systolic volume index (LVESVI) and left ventricular end diastolic volume index (LVEDVI)
measure: Change in LVEDVI
time frame: Baseline, 12 months
description: Change in left ventricular end diastolic index (LVEDVI) as assessed via cardiac MRI
measure: Change in LVESVI
time frame: Baseline, 12 months
description: Change in left ventricular end systolic index (LVESVI) as assessed via cardiac MRI
measure: Change in Maximal oxygen consumption (peak VO2)
time frame: Baseline, 12 months
description: Change in maximal oxygen consumption (peak VO2) as assessed via treadmill
measure: Change in Exercise tolerance
time frame: Baseline, 12 months
description: Change in exercise tolerance as assessed as the distance covered via the six-minute walk test
measure: Change in Minnesota Living with Heart Failure Questionnaire (MLHFQ) Score
time frame: Baseline, 12 months
description: Minnesota Living with Heart Failure Questionnaire (MLHFQ) is a 21-item questionnaire with a total score ranging from 0 to 105 with lower scores indicative of better outcome.
measure: Change in New York Heart Association (NYHA) Class
time frame: Baseline, 12 months
description: NYHA Classifications of heart failure are as follows: Class I (no limitations); Class II (mild symptoms); Class III (marked limitations); Class IV (Severe limitations)
measure: Percent change in flow mediated diameter
time frame: Baseline, 12 months
description: Change in endothelial function will be reported as the percent change in flow mediated diameter assessed via flow mediated dilation (FMD).
measure: Change in EPC-CFU
time frame: Baseline, 12 months
description: Change in endothelial function will be reported as the change in Endothelial Progenitor Cell Colony Forming Unit (EPC-CFU) assessed via blood sample assay
measure: Change in N-Terminal Pro-Brain Natriuretic Peptide (NT-proBNP)
time frame: Baseline, 12 months
description: Change in NT-proBNP as assessed via blooddraw
measure: Change in cytokines
time frame: Baseline, 12 months
description: Change in NT-proBNP as assessed via blooddraw
measure: Incidence of MACE
time frame: 12 months
description: Safety will be reported as the incidence of Major Adverse Cardiac Events (MACE) assessed by treating physician
measure: Incidence of TE-SAEs
time frame: Day 30
description: Safety will be reported as the incidence of Treatment Emergent Serious Adverse Events (TE-SAEs) assessed by treating physician
arm group label: Genotype A administered with placebo Group
arm group type: Placebo Comparator
description: Participants whose blood genotyping resulted with Genotype A (absence of any variant/ presence of benign variant) and randomized to the placebo group will receive the placebo intervention.
arm group label: Genotype A administered with hMSC Group
arm group type: Experimental
description: Participants whose blood genotyping resulted with Genotype A (absence of any variant/ presence of benign variant) and randomized to the treatment group will receive the hMSC intervention.
arm group label: Genotype B administered with placebo Group
arm group type: Placebo Comparator
description: Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the placebo group will receive the placebo intervention.
arm group label: Genotype B administered with hMSC Group
arm group type: Experimental
description: Participants whose blood genotyping resulted with Genotype B (variants of uncertain significance) and randomized to the treatment group will receive the hMSC intervention.
arm group label: Genotype C administered with placebo Group
arm group type: Placebo Comparator
description: Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the placebo group will receive the placebo intervention.
arm group label: Genotype C administered with hMSC Group
arm group type: Experimental
description: Participants whose blood genotyping resulted with Genotype C (pathogenic or likely pathogenic variants) and randomized to the treatment group will receive the hMSC intervention.
intervention type: Biological
intervention name: allogeneic human mesenchymal stem cells (hMSCs)
description: allo-hMSCs, 16-20 million cells/ml delivered at a dose of 0.5 ml/ injection x 10 injections for a total of 80-100 million allo-hMSCs
arm group label: Genotype A administered with hMSC Group
arm group label: Genotype B administered with hMSC Group
arm group label: Genotype C administered with hMSC Group
intervention type: Other
intervention name: Placebo
description: Placebo will be administered as injections of plasmalyte A supplemented with 1% of 25% human serum albumin (HSA). 0.5 ml/ injection x 10 injections
arm group label: Genotype A administered with placebo Group
arm group label: Genotype B administered with placebo Group
arm group label: Genotype C administered with placebo Group
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: No
last name: Joshua Hare, MD
role: Principal Investigator
affiliation: University of Miami
last name: Shelly L Sayre, MPH
phone: 713-500-9529
email: Shelly.L.Sayre@uth.tmc.edu
last name: Lina Caceres
phone: 305-243-5399
email: lvc25@med.miami.edu
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
country: United States
url: https://www.cctrn.org
description: Cardiovascular Cell Therapy Research Network
url: https://www.nih.gov/about-nih/what-we-do/nih-turning-discovery-into-health/stem-cells
description: Information on stem cells from the National Institutes of Health
url: http://www.dcmii.org
description: DCM II Trial
responsible party type: Sponsor-Investigator
investigator affiliation: University of Miami
investigator full name: Joshua M Hare
investigator title: Principal Investigator
mesh term: Cardiomyopathies
mesh term: Cardiomyopathy, Dilated
sharing ipd: No