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

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: 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

  1. Individuals with autologous cord blood product that met all cell release criteria (listed on the certificate of analysis from Mayo Clinic Human Cell Therapy Lab) as follows:
    1. No aerobic or anaerobic bacterial growth after 14 days
    2. Greater than 70% cell viability pre-freeze
    3. Total Nucleated Cells (TNC) concentration of 30-42 x 106 cells/mL (pre-freeze)
    4. Minimum of one (1) vial of cells
    5. Mononuclear cell percentage of greater than 50%
    6. Endotoxin result of less than 16 Endotoxin Units (EU)/mL.
  2. Mother's serology test results are negative for HIV, Hepatitis B, and Hepatitis C.
  3. Individuals with HLHS having undergone Stage I surgical palliation and undergoing planned Stage II palliative Glenn surgery.
  4. 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

  1. Child who's UCB does not meet the specified cell release criteria in Inclusion Criterion #1.
  2. History of dimethyl sulfoxide (DMSO) reaction for either the child or mother.
  3. Parent(s)/child unwilling to participate.
  4. Child with severe chronic diseases, extensive extra-cardiac syndromic features, or history of cancer.
  5. Child not 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.
  6. Child with, or reasonably expected to have, complications during the Stage II Glenn surgery or during post-operative recovery.
  7. Child who's cells have been compromised after meeting cell release criteria (as defined in Inclusion Criterion #1).
  8. Child with the following conditions within 15 days prior to the date of the Stage II Glenn surgery:
    1. Cardiogenic shock
    2. Changes in medical therapy (e.g. supplemental oxygen, vasodilator) for pulmonary hypertension
    3. Changes in arrhythmia medications
    4. A current infection being treated with oral antibiotics or treatment with IV antibiotics within the past 15 days.
  9. Child with the following complications of their congenital heart disease:
    1. Any condition requiring urgent, or unplanned procedure within 15 days prior to Stage II Glenn surgery
    2. Tricuspid repair and/or aortic arch repair at the time of Stage II Glenn surgery
    3. Length of hospitalization of more than 60 days for Stage I surgical palliation, unless cardiac function is normal within 10 days prior to Glenn Surgery
    4. Dietary modifications due to chronic and severe chylothorax
    5. Current or uncontrolled seizure(s) or neurological injury that has resulted in a persistent deficit
    6. Severe tricuspid regurgitation prior to Stage II Glenn surgery
    7. History of mechanical circulatory support, unless cardiac function is normal within 10 days prior to Glenn surgery
    8. Other clinical concerns as documented by a site investigator that could reasonably increase the risk of complications during or after Stage II Glenn surgery.

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 Miller CCRP

(507)266-5510

Miller.Karen1@mayo.edu