Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome Consortium

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Children’s Hospital of Philadelphia is collaborating with the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome at Mayo Clinic. Hypoplastic Left Heart Syndrome (HLHS) is a severe form of coronary heart disease requiring a series of surgical palliations to create a functional single ventricle circulation. Although great strides have been made in the field of HLHS in the past few decades, evidence indicates that up to 40 percent of patients who have completed the final surgery (Stage III Fontan) require heart transplant or major surgical revisions. The HLHS consortium studies are designed to evaluate whether umbilical cord blood-derived stem cells from a patient’s own body may be used as a therapy to strengthen the patient’s right ventricle and improve outcomes. The central hypothesis of these studies is that receiving umbilical cord blood-derived mononucleated cells (UCB-MNCs) during a scheduled cardiac operation will improve cardiac function, reaching growth milestones, and quality of life in the short-term, while also reducing inpatient stay following scheduled cardiac surgeries.

Research Project Highlights

Researchers in the Todd and Karen Wanke Family Program for Hypoplastic Left Heart Syndrome are engaged in the following efforts:

  • Umbilical Cord Blood Collection and Processing for Cardiac Regeneration in HLHS: The purpose of this research study is to collect umbilical cord blood at birth from infants diagnosed with HLHS. Autologous stem cells (from baby’s own body) can be derived from the umbilical cord blood, which can be stored at Mayo Clinic so that they may be available to baby in a future clinical trial. Parents are contacted at approximately 60 days after delivery to hear about potential cardiac stem cell trials.
  • Phase I Safety Study of UCB-MNCs in HLHS: The purpose of this study is to assess whether it is safe and feasible to deliver autologous UCB-MNCs during scheduled Stage II surgery. The central hypothesis of this study is that receiving UCB-MNCs during Stage II Glenn surgical palliation is safe and may be used as a therapy to therapy to strengthen HLHS patients’ right ventricles and improve outcomes for these patients.  
  • Observational Study of HLHS Post Stage II: The purpose of this observational study is to track health outcomes and cardiac function of subjects with HLHS after undergoing elective Stage II Glenn surgical palliation. There is no experimental treatment/intervention involved in this study. Results of this study will be used to provide a better understanding of clinical course post-Stage II surgery, and act as a case control comparator for the Phase I Safety Study of UCB-MNCs in HLHS.
  • Phase II Efficacy Study of UCB-MNCs in HLHS: This study assesses whether autologous UCB-MNCs, delivered during scheduled Stage II surgery, are an effective therapy to strengthen right ventricles and improve outcomes for HLHS patients. A treatment group receives experimental cell therapy, and a control group receives no experimental treatment. The central hypothesis of this study is that receiving UCB-MNCs during Stage II Glenn surgical palliation will improve cardiac function, reaching growth milestones, and quality of life in the short-term, while also reducing inpatient stay following Stage II surgery, when compared with the control group not receiving the cells.

Additional Contact info:

Mayo Clinic
hlhs@mayo.edu
507-266-6459