Study of hCT-MSC in Newborn Infants With Moderate or Severe HIE
Condition: Moderate to Severe Hypoxic-ischemic Encephalopathy
MeSH terms: Brain Diseases; Brain Ischemia; Hypoxia-Ischemia, Brain; Ischemia; Hypoxia
Study type: Interventional
Phase: Phase 1

Summary: 
      To determine the safety of single and repeated intravenous doses of hCT-MSC in newborn
      infants with HIE.
    

Detailed description: 
      The purpose of this study is to assess the safety of one and two intravenous infusions of
      human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), the first
      administered in the first 48 postnatal hours, and the second at two months postnatal age, in
      term and near term infants with moderate to severe neonatal hypoxic-ischemic encephalopathy
      (HIE). This is a phase I, prospective, open-label trial designed to assess the safety of one
      or two intravenous doses of hCT-MSC in newborn infants with moderate to severe HIE who are
      recipients of therapeutic hypothermia. Infants born at 36 0/7 weeks gestation or later who
      have moderate to severe hypoxic-ischemic encephalopathy and are receiving therapeutic
      hypothermia will be eligible to participate. Investigators project an accrual of 6 patients.
      All infants will receive intravenous infusion(s) of hCT-MSCs. The first cohort of three
      infants will receive a single dose in the first 48 postnatal hours. If there are no safety
      concerns, the second cohort of three infants will receive two doses, with the first dose
      given in the first 48 postnatal hours and the second dose given approximately two months
      after the first dose.

      The potential risks associated with infusion of MSCs include a reaction to the product (rash,
      shortness of breath, wheezing, difficulty breathing, hypotension, swelling around the mouth,
      throat or eyes, tachycardia, diaphoresis), transmission of infection, and HLA sensitization.
      Another risk of this study is loss of confidentiality or privacy. Every effort will be made
      to keep the infant's medical record confidential. The results will be summarized using
      descriptive statistics and statistical testing as appropriate. Continuous secondary endpoints
      will be summarized using mean, standard deviation, CV%, median, minimum, and maximum.
    

Intervention name(s): Infusion of hCT-MSC
Intervention type(s): Biological
Intervention description(s): Infants who meet enrollment criteria for moderate to severe hypoxic ischemic encephalopathy will receive 1 infusion of hCT-MSC within the first 48 postnatal hours. hCT-MSCs are a product of allogeneic cells manufactured from digested umbilical cord tissue that is expanded in culture, cryopreserved and banked. hCT-MSCs are manufactured from umbilical cord tissue donated to the Carolinas Cord Blood Bank, an FDA-licensed, FACT-accredited, public cord blood bank at Duke University Medical Center, after written informed consent from the baby's mother. Cord tissue is harvested from the placentas of male babies delivered by elective C-section after a normal, full- term pregnancy.
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Intervention model description: The first cohort of three patients will receive a single dose in the first 48 postnatal hours. If there are no safety concerns, the second cohort of three patients will receive two doses, with the first dose given in the first 48 postnatal hours and the second dose given approximately two months after the first dose.
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: hypoxic-ischemic encephalopathy; newborn infants; therapeutic hypothermia; hCT-MSC, an Umbilical Cord Tissue-Derived Mesenchymal Stromal Cell Product