Clinical trial NCT02612155: A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy
Official title: A Phase II Multi-site Study of Autologous Cord Blood Cells for Hypoxic (HIE)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
Keywords: Hypoxic-ischemic encephalopathy
autologous cord blood cells
newborn infants
Lead sponsor: Duke University
Sponsor class: OTHER
Collaborators: name: The Robertson Foundation
First posted: 2015-11-23
Enrollment: 35
Sex: ALL
Minimum age (years): 0
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

1. NICHD Neonatal Research Network Hypothermia Trial inclusion criteria
2. 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
3. The infant must be able to receive at least one dose of autologous cord blood before 48 hours of age
4. All infants must have signs of encephalopathy within 6 hours of age

Exclusion Criteria:

1. Major congenital or chromosomal abnormalities
2. Severe growth restriction (birth weight \<1800 g)
3. Opinion by attending neonatologist that the study may interfere with treatment or safety of subject
4. Moribund neonates for whom no further treatment is planned
5. Infants born to mothers are known to be HIV, Hepatitis B, Hepatitis C or who have active syphilis or CMV infection in pregnancy
6. Infants suspected of overwhelming sepsis
7. ECMO initiated or likely in the first 48 hours of life

Primary outcomes:
measure: Survival at One Year
timeFrame: 1 year
measure: Number of Participants With Bayley III Scores in All Three Domains > or Equal to 85
timeFrame: 1 year
Secondary outcomes:
measure: Mortality Rate
timeFrame: 1 year
measure: Number of Subjects Who Experience Seizures
timeFrame: During hospitalization, approximately 4-92 days
measure: Number of Subjects Who Require iNO (Inhaled Nitric Oxide) Use
timeFrame: During hospitalization, approximately 4-92 days
measure: Number of Subjects Who Require ECMO
timeFrame: During hospitalization, approximately 4-92 days
measure: Number of Subjects Who Require Gastrostomy Tube (G-tube) Feeding
timeFrame: During hospitalization, approximately 4-92 days
measure: Number of Subjects Who Are Discharged on Anti-epileptic Medication
timeFrame: At hospital discharge, approximately 4-92 days
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
Study officials:
affiliation: Duke University
name: Michael Cotten, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Cotten CM, Murtha AP, Goldberg RN, Grotegut CA, Smith PB, Goldstein RF, Fisher KA, Gustafson KE, Waters-Pick B, Swamy GK, Rattray B, Tan S, Kurtzberg J. Feasibility of autologous cord blood cells for infants with hypoxic-ischemic encephalopathy. J Pediatr. 2014 May;164(5):973-979.e1. doi: 10.1016/j.jpeds.2013.11.036. Epub 2013 Dec 31.
citation: Shankaran S, Laptook AR, Ehrenkranz RA, Tyson JE, McDonald SA, Donovan EF, Fanaroff AA, Poole WK, Wright LL, Higgins RD, Finer NN, Carlo WA, Duara S, Oh W, Cotten CM, Stevenson DK, Stoll BJ, Lemons JA, Guillet R, Jobe AH; National Institute of Child Health and Human Development Neonatal Research Network. Whole-body hypothermia for neonates with hypoxic-ischemic encephalopathy. N Engl J Med. 2005 Oct 13;353(15):1574-84. doi: 10.1056/NEJMcps050929.
citation: Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604.