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Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells

This study is currently recruiting participants.
Verified September 2016 by Timothy J. Nelson, Mayo Clinic
Sponsor:
ClinicalTrials.gov Identifier:
NCT02549625
First Posted: September 15, 2015
Last Update Posted: September 26, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Timothy J. Nelson, Mayo Clinic
  Purpose
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) and intracoronary delivery for individuals with declining ventricle systemic pumps. This procedure has the potential to foster a new strategy for congenital heart patients. This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) related serious adverse events and 2) monitor changes in cardiac structure and function.

Condition Intervention Phase
Congenital Heart Disease Biological: Autologous Bone Marrow-derived Mononuclear Cells Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells for Systemic, Single Right Ventricular Failure Due to Congenital Heart Disease

Resource links provided by NLM:


Further study details as provided by Timothy J. Nelson, Mayo Clinic:

Primary Outcome Measures:
  • Number of related serious adverse events [ Time Frame: 6 months post cell delivery ]

Secondary Outcome Measures:
  • Number of subjects who died [ Time Frame: 6 months post cell delivery ]
  • Number of subjects with sustained or symptomatic ventricular arrhythmias [ Time Frame: 6 months post cell delivery ]
  • Number of subjects with acute decompensated heart failure [ Time Frame: 6 months post cell delivery ]
  • Number of subjects with myocardial infarction [ Time Frame: 6 months post cell delivery ]
  • Number of subjects with cardiac infection [ Time Frame: 6 months post cell delivery ]
  • Number of subjects with unexpected cardiovascular procedures within 6 months following cellular transplantation [ Time Frame: 6 months post cell delivery ]
  • Percentage of subjects that have cells delivered [ Time Frame: 6 months post cell delivery ]
  • Percentage of subjects completing the 6-month follow-up [ Time Frame: 6 months post cell delivery ]
  • Change in CT/MRI derived right ventricle ejection fraction [ Time Frame: Baseline, 6 months post cell delivery ]
  • Change in diastolic ventricular volume [ Time Frame: Baseline, 6 months post cell delivery ]
  • Change in systolic ventricular volume [ Time Frame: Baseline, 6 months post cell delivery ]
  • Change in echocardiography derived right ventricle ejection fraction [ Time Frame: Baseline, 6 months post cell delivery ]

Estimated Enrollment: 10
Study Start Date: August 2015
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Single-arm
Intracoronary delivery of autologous bone marrow-derived mononuclear cells using catheterization procedure
Biological: Autologous Bone Marrow-derived Mononuclear Cells
Other Name: MNC

Detailed Description:
This study is an open label Phase I trial to determine the safety and feasibility of bone marrow-derived mononuclear cells delivered into the coronary circulation of subjects with Fontan circulation and right ventricular dysfunction. Subjects will be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will be consented and offered participation in this trial. Once informed consent has been obtained; preoperative values will be established and a selection committee will review subjects within three days prior to planned procedure to confirm inclusion and exclusion criteria. This will require a non-study cardiologist to review the clinical case and confirm the baseline cardiac function with evidence of disease progression with right ventricle dysfunction. However, individuals with decreased cardiac function requiring inotropic support and immediate listing for cardiac transplantation will NOT be included such that the risk of this research procedure may not be acceptable. Following cell-based product delivery, subjects will be followed for 6-months according to a pre-determined schedule that includes imaging studies along with questionnaires, electrophysiology and laboratory studies. Subjects will be contacted for an additional 18-months for surveillance by phone.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Individuals with Fontan circulation with right ventricular dysfunction
  • Ejection fraction equal to or less than 40% despite optimal outpatient medical management for at least 3 months
  • Able to undergo bone marrow aspirate according to clinical consultation with hematology (INR must be maintained at or below 1.5)
  • Able to undergo an MRI or CT examination

Exclusion Criteria:

  • Individuals or parents of minors unwilling to consent to participation
  • Individuals with severe chronic diseases, extra-cardiac syndromes, or cancer
  • Females 10 years and older with positive pregnancy test or lack of effective birth control method
  • Individuals currently requiring IV inotropes
  • Individuals with bleeding disorders or history of thrombosis
  • Individuals not eligible for MRI and have elevated serum creatinine level (eGFR less that 45 mL/min) and therefore cannot have a CT examination
  • Individuals not eligible for MRI and have a prior significant reaction to intravenous contrast required for CT examination
  • Individuals with the following conditions within 60 days prior to procedure:

    • Cardiogenic shock or extracorporeal circulation;
    • New arrhythmia that required medication for control;
    • Documented infection requiring treatment with antibiotics, and/or current infection being treated with antibiotics;
    • Cardiac condition requiring emergency procedure;
    • Cardiovascular surgery;
    • Seizures or history of significant neurological injury;
    • Multi-system organ failure including acute or chronic renal failure
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02549625


Contacts
Contact: Karen M Cavanaugh, CCRP 507-538-8425 cavanaugh.karen@mayo.edu
Contact: Karen S Miller, CCRP 507-266-5510 miller.karen1@mayo.edu

Locations
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Karen M Cavanaugh, CCRP    507-538-8425    cavanaugh.karen@mayo.edu   
Contact: Lori A Riess, CCRP    507-538-7730    riess.lori@mayo.edu   
Principal Investigator: Muhammad Y Qureshi, MBBS         
Sponsors and Collaborators
Timothy J. Nelson
Investigators
Study Director: Timothy J Nelson, M.D., Ph.D. Mayo Clinic
Principal Investigator: Muhammad Y Qureshi, MBBS Mayo Clinic
  More Information

Responsible Party: Timothy J. Nelson, Program Director, Mayo Clinic
ClinicalTrials.gov Identifier: NCT02549625     History of Changes
Other Study ID Numbers: 13-006427
First Submitted: September 10, 2015
First Posted: September 15, 2015
Last Update Posted: September 26, 2016
Last Verified: September 2016

Keywords provided by Timothy J. Nelson, Mayo Clinic:
Single right ventricular failure

Additional relevant MeSH terms:
Heart Diseases
Heart Defects, Congenital
Cardiovascular Diseases
Cardiovascular Abnormalities
Congenital Abnormalities