Study of Autologous Bone Marrow Derived Mononuclear Cells for Treatment of Ebstein Anomaly
Condition: Ebstein Anomaly
MeSH terms: Ebstein Anomaly; Congenital Abnormalities
Study type: Interventional
Phase: Phase 1

Summary: 
      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.
    

Detailed description: 
      This study is an open label Phase I trial to determine the safety and feasibility of bone
      marrow-derived mononuclear cells to the right ventricle of subjects with Ebstein anomaly at
      the time of a planned surgical Ebstein repair compared to the control group undergoing the
      same planned surgical intervention without the cell delivery add-on procedure. Subjects will
      be screened at outpatient clinic visits at Mayo Clinic and interested qualified subjects will
      be consented and offered participation in this trial. Subject/Family will decide to
      participate in cell delivery or control arm. Once informed consent has been obtained,
      subjects will undergo identical evaluation (other than Hematology consultation and BM harvest
      occurring only in the cell-treatment group),preoperative values will be established/confirmed
      and a selection committee will review subjects within three days prior to planned procedure
      to confirm inclusion and exclusion criteria.However, individuals at high-risk, primarily
      neonates and those with preoperative advanced right heart failure will NOT be included such
      that the risk of this add-on procedure may not be acceptable. Following surgical Ebstein
      repair for the control group and following cell-based product delivery for the treatment
      group, all subjects will be followed for 24-months according to a pre-determined schedule
      that includes imaging studies along with a questionnaire, electrophysiology and laboratory
      studies at 1, 6, and 24 months post procedure. All subjects will be contacted for
      surveillance by phone at 3, 12, and 18 month post procedure.
    

Intervention name(s): Autologous Bone Marrow-derived Mononuclear Cells; Insertable cardiac monitor
Intervention type(s): Biological; Device
Intervention description(s): Following surgical Ebstein repair a Medtronic Reveal LINQ Insertable Cardiac Monitor (ICM) will be placed subcutaneously in the anterior chest to continuously monitor the subject's heart rhythm and to record cardiac information through automatic detection of arrhythmias.
Other intervention name(s): MNC; ICM; ILR
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Myopathic right ventricle