Safety Study of Autologous Umbilical Cord Blood Cells for Treatment of Hypoplastic Left Heart Syndrome

  • Study type:

    Interventional What is this?

    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.
  • Study phase:

    I What is this?

    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.

Study IDs

  • Site IRB:

    • Rochester, Minnesota: 12-008521
  • NCT ID:

    NCT01883076
  • Sponsor Protocol Number:

    12-008521

About this study

This is a Phase I study to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of HLHS children undergoing a scheduled Glenn surgical procedure.

The investigators are doing this research study to find out if autologous stem cells from the individual's own umbilical cord blood can be used to strengthen the muscle of the right side of their heart. This will help determine the safety and feasibility of using cell-based regenerative therapy as an additional treatment for the management of HLHS.

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 with autologous cord blood product enrolled, consented and collected under IRB #11-007176, that met all release criteria according to that protocol
  • Individuals with HLHS undergoing planned Stage II palliative surgeries
  • Ages up to 18 months are eligible if written informed consent can be obtained from both parents (unless one parent is not reasonably available) and/or legal guardians

Exclusion Criteria:

  • Individuals with UCB collected under consent and processed according to IRB #11-007176, not meeting specified release criteria or have not previously collected UCB
  • History of DMSO reaction for either the infant or mother
  • Individuals with families unwilling to participate
  • Individuals with severe chronic diseases, extra-cardiac syndromes, or cancer
  • Infants with the following conditions within 15 days prior to the date of the Stage II Glenn surgery:
    • Cardiogenic shock
    • Pulmonary hypertension requiring chronic medical therapy (e.g. supplemental oxygen, vasodilator)
    • Arrhythmia that required medication for control
    • Any documented infection requiring treatment with IV antibiotics, and/or current infection being treated with antibiotics
  • Infants with the following complications of their congenital heart disease:
    • Any cardiac condition requiring urgent, or unplanned procedure 15 days prior to Stage II Glenn surgery
    • Tricuspid repair and/or aortic arch repair at the time of Stage II Glenn surgery
    • Length of hospitalization of more than 60 days for Stage I Norwood procedure
    • Chylothorax requiring dietary modifications
    • Seizure or neurological injury
    • Moderate to severe tricuspid regurgitation prior to Stage II Glenn surgery
    • History of extracorporeal membrane oxygenator (ECMO) support

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Timothy Nelson, M.D., Ph.D.

Open for enrollment

Julia Thebiay

(507)538-8425

Thebiay.Julia@mayo.edu