Cord Blood for Neonatal Hypoxic-ischemic Encephalopathy

Study Overview
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Brief Summary
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This is a pilot study to test feasibility of collection, preparation and infusion of a baby's own (autologous)umbilical cord blood in the first 14 days after birth if the baby is born with signs of brain injury.

Detailed Description
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The purpose of this pilot study is to evaluate the safety and feasibility of infusions of autologous (the patient's own)umbilical cord blood cells in term gestation newborn infants with hypoxic-ischemic encephalopathy. For this study, infants who have signs of moderate to severe encephalopathy at birth whose mothers have previously consented to providing cord blood cells for the Carolinas Cord Blood Bank or other public or private bank that uses accepted standards for collection and handling of cells, or provided verbal consent for cord blood collection for the possibility of their baby's participation in this trial, can receive their own cord blood cells if an adequate number of cells that meet Carolinas Cord Blood Bank Quality standards are available in the first 14 postnatal days. Study activities also include serial blood draws concurrent with clinically indicated blood draws with a total volume of no more than 5 milliliters (1 teaspoon) from all study related tests. Babies will be followed for neurodevelopmental outcome at 4 - 6 and 9 - 12 months at Duke's Special Infant Care Clinic. MRI's will be obtained per clinical routine and results will be analyzed and described in study reports.

Official Title
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Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy Study 1. Phase I Study of Feasibility and Safety.

Conditions
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Neonatal Hypoxic Ischemic Encephalopathy

Intervention / Treatment
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* Biological: infusion of autologous cord blood
* Other: Neurodevelopmental outcomes


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Mothers must have consented for cord blood collection at delivery cord blood must be available for extraction of stem cells. >34 weeks gestation cord or neonatal pH<7.0 or base deficit>16 milliequivalents per liter (mEq/L) or history of acute perinatal event either a 10 minute Apgar < 5 or continued need for ventilation. All infants must have signs of encephalopathy within 6 hours of age. Exclusion Criteria: Inability to enroll by 14 days of age. Presence of known chromosomal anomaly. Presence of major congenital anomalies. Severe intrauterine growth restriction (weight <1800g) Infants in extremis for whom no additional intensive therapy will be offered by attending neonatologist. Parents refuse consent. Attending neonatologist refuses consent. Failure to collect the infant's cord blood and/or laboratory unable to process cord blood.

Ages Eligible for Study
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Maximum Age: 14 Days

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: infusions<br>infants who arrive at the study site within the first 14 postnatal days and had a history of moderate to severe hypoxic ischemic encephalopathy, and have cells available for infusion that pass Carolinas Cord Blood Bank Quality checks Outcomes will be measured at 22-26 months fby neurodevelopment assessment | Biological: infusion of autologous cord blood<br>* infants who meet study enrollment criteria for history of moderate to severe hypoxic ischemic encephalopathy in the neonatal period will receive up to 4 infusions of their own volume reduced cord blood cells. The number of doses will be determined by the amount of available cord blood cells. The dose for each infusion is 5x10e7 cells/kg<br>|
| Other: historical control<br>Infants who had moderate to severe hypoxic ischemic encephalopathy in the neonatal period but did not receive autologous cord blood cells. | Other: Neurodevelopmental outcomes<br>* historical controls, no experimental intervention, standard therapies of hypoxic ischemic encephalopathy in the newborn period with autologous cord blood<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse event rates occurring in the pilot study population will be compared between the cord blood cell recipients and historical controls. |  | during infusions: first 18 postnatal days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Secondary endpoints of this pilot study will include preliminary efficacy as measured by neurodevelopmental function at 4 - 6 months and 9 - 12 months of age |  | 1 year | 
| neuroimaging results will be collected and compared with available results from prior trials of therapies in this population, and from a previously collected set of images from normal term newborns through the first year of life. |  | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hypoxic-ischemic encephalopathy, autologous cord blood cells, newborn infants