A Phase II Multi-site Study of Autologous Cord Blood Cells for Hypoxic (HIE)
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
- Sponsor
- Michael Cotten
- Enrollment
- 35
- Locations
- 1
- Primary Endpoint
- Number of Participants With Bayley III Scores in All Three Domains > or Equal to 85
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
This study will test the safety and efficacy of an infusion of a baby's own (autologous) umbilical cord blood as compared with placebo in babies born with history and signs of hypoxic-ischemic brain injury.
Detailed Description
The purpose of this phase II study is to assess the safety and efficacy of up to two intravenous infusions of autologous volume and red blood cell reduced nucleated umbilical cord blood cells as compared with placebo in neonates with neonatal encephalopathy undergoing hypothermia treatment. Efficacy will be estimated by one year survival and score on Bayley III scores in all three domains equal to or greater than 85. This will be a randomized, double-blind, placebo controlled multi-site trial of up to 160 infants who qualify for cooling.
Investigators
Michael Cotten
Associate Professor
Duke University
Eligibility Criteria
Inclusion Criteria
- •NICHD Neonatal Research Network Hypothermia Trial inclusion criteria
- •Mothers must have consented or given verbal assent for cord blood collection at delivery, and cord blood must be available for volume and red blood cell reduction before 45 hours of age
- •The infant must be able to receive at least one dose of autologous cord blood before 48 hours of age
- •All infants must have signs of encephalopathy within 6 hours of age
Exclusion Criteria
- •Major congenital or chromosomal abnormalities
- •Severe growth restriction (birth weight \<1800 g)
- •Opinion by attending neonatologist that the study may interfere with treatment or safety of subject
- •Moribund neonates for whom no further treatment is planned
- •Infants born to mothers are known to be HIV, Hepatitis B, Hepatitis C or who have active syphilis or CMV infection in pregnancy
- •Infants suspected of overwhelming sepsis
- •ECMO initiated or likely in the first 48 hours of life
Outcomes
Primary Outcomes
Number of Participants With Bayley III Scores in All Three Domains > or Equal to 85
Time Frame: 1 year
The Bayley is a standardized, norm-referenced measure that assesses development in Cognitive, Language and Motor domains. Composite standard scores can be derived that have a mean of 100 and a standard deviation of 15.
Survival at One Year
Time Frame: 1 year
Number of participants alive at one year.
Secondary Outcomes
- Number of Subjects Who Are Discharged on Anti-epileptic Medication(At hospital discharge, approximately 4-92 days)
- Mortality Rate(1 year)
- Number of Subjects Who Require ECMO(During hospitalization, approximately 4-92 days)
- Number of Subjects Who Require Gastrostomy Tube (G-tube) Feeding(During hospitalization, approximately 4-92 days)
- Number of Subjects Who Experience Seizures(During hospitalization, approximately 4-92 days)
- Number of Subjects Who Require iNO (Inhaled Nitric Oxide) Use(During hospitalization, approximately 4-92 days)