Phase I Study of Bone Marrow Derived Mononuclear Cells to Right Ventricle of Patients With Ebstein Anomaly

Overview

  • Study type

    Interventional
  • Study phase

    I
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 15-007580
    NCT ID: NCT02914171
    Sponsor Protocol Number: 15-007580

About this study

The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNS) collected from bone marrow (BM) and using an add-on intramyocardial delivery for individuals with Ebstein anomaly undergoing surgical intervention compared to the control group undergoing the same surgical procedure without cell delivery. This add-on procedure has the potential to foster a new strategy for individuals with congenital heart disease.This is an open-label study of autologous MNC derived from bone marrow with a 2-year follow-up to document 1) incidence and severity of adverse event and 2) monitor changes in cardiac structure and function.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Individuals clinically planned for elective surgical Ebstein repair
  • Individuals able to undergo bone marrow aspirate according to clinical consultation with Hematology (cell treatment group only)
  • Individuals able to undergo preoperative MRI or CT examination
  • Individual and/or parent willing and able to give informed consent and willing to commit to completion of follow-up

Exclusion Criteria:

  • Individuals requiring cavopulmonary shunt at the time of surgical intervention; planned preoperatively or required intraoperatively (pre-operative cardiothoracic ratio >0.65, right ventricular end-diastolic volume >250mL/m², or right ventricular ejection fraction ≤25% will be excluded due to increased likelihood of requiring cavopulmonary shunt)
  • Individuals who require tricuspid valve replacement
  • Individuals who require surgery on pulmonary, mitral, or aortic valve
  • Individuals with pulmonary atresia or atrioventricular discordance with ventriculoarterial discordance
  • Individuals with history of ventricular arrhythmia
  • Individuals who have undergone previous cardiac operations
  • Individuals with preoperative ventricular arrhythmia requiring medical management
  • Individuals with severe chronic diseases, extra-cardiac syndromes, or history of any cancer
  • Individuals with current IV inotrope requirements
  • Individuals with bleeding disorders or history of thrombosis
  • Subjects not eligible for MRI or CT examination due to either 1) elevated serum creatinine level (eGFR <45mL/min), 2) prior significant reaction to intravenous contrast required for MRI or CT examination
  • Individuals with the following conditions within 60 days prior to surgery:
    • Cardiogenic shock or extracorporeal circulation
    • Documented infection requiring treatment with intravenous and/or oral antibiotics
    • Cardiac condition requiring emergency procedure
    • Seizures or history of significant neurological injury
    • Multi-system organ failure including acute or chronic renal failure
  • Female subjects 10 years and older with positive pregnancy test or lack of effective birth control method during the 30 days prior to procedure
  • Individuals who weigh more than 90 kg secondary to cell-product dosing restrictions

The cell-product will not be delivered in subjects in the cell-treatment group after confirmation of cell-product not meeting release criteria by the Human Cellular Therapy Laboratory Manufacturing team.

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

M. Yasir Qureshi, M.B.B.S.

Open for enrollment

Contact information:

Karen Cavanaugh CCRP

(507)538-8425

Cavanaugh.Karen@mayo.edu