Autologous Cord Blood Cell Therapy for Neonatal Encephalopathy

Study Overview
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Brief Summary
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This is a pilot study to test feasibility and safety of intravenous infusion of autologous umbilical cord blood cells in the first 72 hours after birth if a neonate is born with signs of encephalopathy.

Detailed Description
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This is a multicenter pilot study to evaluate the feasibility and safety of intravenous infusions of autologous (the patient's own) umbilical cord blood cells in term gestation newborns with neonatal encephalopathy (hypoxic-ischemic encephalopathy). If a neonate is born with signs of moderate to severe encephalopathy and cooled for the encephalopathy, the neonate can receive their own non-cryopreserved volume- and red blood cell-reduced cord blood cells. The cord blood cells are divided into 3 doses and infused at 12-24, 36-48, and 60-72 hours after the birth. Infants will be followed for safety and neurodevelopmental outcome up to 18 months.

Official Title
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A Pilot Feasibility and Safety Study of Autologous Umbilical Cord Blood Cell Therapy in Infants With Neonatal Encephalopathy

Conditions
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Neonatal Encephalopathy, Hypoxic-ischemic Encephalopathy

Intervention / Treatment
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* Other: Autologous umbilical cord blood cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Infants are eligible if they meet all the following inclusion criteria except 4. ≥36 weeks gestation Either a 10-minute Apgar score ≤5, continued need for resuscitation for at least 10 minutes, or severe acidosis, defined as pH <7.0 or base deficit ≥16 mmol/L in a sample of umbilical cord blood or any blood during the first hour after birth Moderate to severe encephalopathy (Sarnat II to III) A moderately or severely abnormal background amplitude-integrated EEG (aEEG) voltage, or seizures identified by aEEG, if monitored Up to 24 hours of age Autologous umbilical cord blood available to infuse within 3 days after birth A person with parental authority must have consented for the study. Exclusion Criteria: Known major congenital anomalies, such as chromosomal anomalies, heart diseases Major intracranial hemorrhage identified by brain ultrasonography or computed tomography Severe growth restriction, with birth-weight less than 1800 g Severe infectious disease, such as sepsis Hyperkalemia Outborn infants (Infants born at hospitals other than the study sites) Volume of collected cord blood <40 ml Infants judged critically ill and unlikely to benefit from neonatal intensive care by the attending neonatologist

Ages Eligible for Study
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Maximum Age: 24 Hours

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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cell therapy<br>Infants who are born at the study sites, have moderate to severe encephalopathy, and have cord blood available for infusion | Other: Autologous umbilical cord blood cells<br>* Autologous non-cryopreserved volume- and red blood cell-reduced cord blood cells will be intravenously infused<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse event rates | Adverse event rates (combined rate of death, continuous respiratory support, and continuous use of vasopressor) will be compared between the cell recipients and historical controls at 30 days of age. | first 30 postnatal days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy | Neuroimaging at 12 months of age and neurodevelopmental function at 18 months of age will be compared between the cell recipients and historical controls. | 18 months | 

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