Safety of BM-MNC Intramyocardial Delivery in Ebstein RV

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 evaluate the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) delivered into the myocardium of the right ventricle of subjects with Ebstein anomaly undergoing surgical Ebstein repair. Additionally, the potential cardiovascular benefits will also be evaluated. This add-on procedure is anticipated to pose little risk to the subject and has the potential to foster a new strategy that leverages the regenerative capacity of individuals with congenital heart disease during the surgically mandated Ebstein repair.

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

  • Age 6 months to 30 years
  • Clinically planned, 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 Ebstein repair; planned preoperatively or required intraoperatively
  • Individuals with, or reasonably expected to have, complications during surgical Ebstein repair or during post-operative recovery
  • Individual has not completed or will not be completing all pre-procedure work-up within 30 days of surgical Ebstein repair as listed in section 6 of this protocol AND lack of pre-procedure work-up documented as a safety concern by a site investigator
  • Other clinical concerns as documented by a site investigator that could reasonably increase the risk of complications during or after surgical Ebstein repair
  • Individual whose cells have been determined, by the sponsor, to not be acceptable for release to the investigational site or individual whose cells have been compromised after cells released to investigational site (cell treatment group only)
  • Individuals who require surgery on pulmonary, mitral, or aortic valve
  • Individuals with pulmonary atresia or atrioventricular discordance with ventriculoarterial discordance

 

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