A Study of Autologous Umbilical Cord Blood Derived Mononuclear Cells During Surgical Stage II Palliation of Hypoplastic Left Heart Syndrome


  • Study type

  • Study phase

  • 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: 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 Hypoplastic Left Heart Syndrome (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:

  • Child’s UCB-MNC product is determined by the sponsor to be acceptable for release to the investigational site for investigational use.
  • Mother’s serology test results are negative for HIV, Hepatitis B, and Hepatitis C.
  • Individuals with HLHS having undergone Stage I surgical palliation and undergoing planned Stage II palliative Glenn surgery.
  • 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

  • History of DMSO reaction for either the child or mother.
  • Parent(s)/child unwilling to participate.
  • Child with severe chronic diseases, extensive extra-cardiac syndromic features, or history of cancer.
  • Child has not completed or will not be completing all pre-procedure work-up within 10 days of the Stage II Glenn surgery as listed in section 6 of this protocol AND lack of pre-procedure work-up documented as a safety concern by a site investigator.
  • Child whose cells have been compromised after cells released to investigational site.
  • Child with the following complications of their congenital heart disease:
    • Any condition requiring urgent, or unplanned procedure within 15 days prior to Stage II surgical repair;
    • Severe pulmonary hypertension (reported in the medical record as >70% systemic pressure);
    • Other clinical concerns as documented by a site investigator that would predict (more likely to happen than not to happen) a risk of severe     complications or very poor outcome during or after Stage II surgical repair.

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.

Closed for enrollment

Contact information:

Karen Miller CCRP



More information


Publications are currently not available

Study Results Summary

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Supplemental Study Information

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