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A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy ((HIE))

Primary Purpose

Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Infusion of autologous cord blood
Placebo
Sponsored by
Michael Cotten
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns focused on measuring Hypoxic-ischemic encephalopathy, autologous cord blood cells, newborn infants

Eligibility Criteria

undefined - 6 Hours (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Alabama Birmingham
  • University of Florida Gainesville
  • MassGeneral Hospital for Children
  • Brigham and Women's Hospital
  • Wayne State University Hospital
  • Duke University Medical Center
  • University of Florida at Jacksonville
  • Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Intervention cell recipients

Placebo recipients

Arm Description

Experimental: infusions: infants with moderate to severe hypoxic ischemic encephalopathy, begin cooling, and have autologous nucleated cord blood cells available for infusion will receive up to two infusions. Outcomes will be measured at 22-26 months by neurodevelopment assessment

Control: infants with moderate to severe hypoxic ischemic encephalopathy, begin cooling, and have cord blood available for infusion will receive placebo (a mix of autologous cord blood red blood cells and plasma) infusions. Outcomes will be measured at 22-26 months by neurodevelopment assessment

Outcomes

Primary Outcome Measures

Survival at One Year
Number of participants alive at one year.
Number of Participants With Bayley III Scores in All Three Domains > or Equal to 85
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.

Secondary Outcome Measures

Mortality Rate
(Number of participants who died/total number of participants) x 100
Number of Subjects Who Experience Seizures
Number of Subjects Who Require iNO (Inhaled Nitric Oxide) Use
Number of Subjects Who Require ECMO
ECMO (extracorporeal membrane oxygenation) is a technique of providing prolonged cardiac and respiratory support to persons whose heart and lungs are unable to provide an adequate amount of gas exchange or perfusion to sustain life.
Number of Subjects Who Require Gastrostomy Tube (G-tube) Feeding
Number of Subjects Who Are Discharged on Anti-epileptic Medication

Full Information

First Posted
November 19, 2015
Last Updated
August 19, 2020
Sponsor
Michael Cotten
Collaborators
The Robertson Foundation
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1. Study Identification

Unique Protocol Identification Number
NCT02612155
Brief Title
A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy
Acronym
(HIE)
Official Title
A Phase II Multi-site Study of Autologous Cord Blood Cells for Hypoxic (HIE)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2020
Overall Recruitment Status
Completed
Study Start Date
March 30, 2017 (Actual)
Primary Completion Date
August 5, 2019 (Actual)
Study Completion Date
August 5, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Michael Cotten
Collaborators
The Robertson Foundation

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
Keywords
Hypoxic-ischemic encephalopathy, autologous cord blood cells, newborn infants

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
35 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Intervention cell recipients
Arm Type
Experimental
Arm Description
Experimental: infusions: infants with moderate to severe hypoxic ischemic encephalopathy, begin cooling, and have autologous nucleated cord blood cells available for infusion will receive up to two infusions. Outcomes will be measured at 22-26 months by neurodevelopment assessment
Arm Title
Placebo recipients
Arm Type
Placebo Comparator
Arm Description
Control: infants with moderate to severe hypoxic ischemic encephalopathy, begin cooling, and have cord blood available for infusion will receive placebo (a mix of autologous cord blood red blood cells and plasma) infusions. Outcomes will be measured at 22-26 months by neurodevelopment assessment
Intervention Type
Biological
Intervention Name(s)
Infusion of autologous cord blood
Intervention Description
Infants who meet study enrollment criteria will receive up to 2 infusions of their own volume reduced cord blood cells. The number of doses will be determined by the amount of available cord blood cells.
Intervention Type
Biological
Intervention Name(s)
Placebo
Intervention Description
Infants who meet study enrollment criteria will receive up to 2 placebo infusions composed of an equivalent volume (volume of product that would have been administered if the infant randomized to the intervention arm) of packed red blood cells (PRBCs) from the red cell compartment of the separated cord blood unit.
Primary Outcome Measure Information:
Title
Survival at One Year
Description
Number of participants alive at one year.
Time Frame
1 year
Title
Number of Participants With Bayley III Scores in All Three Domains > or Equal to 85
Description
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.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Mortality Rate
Description
(Number of participants who died/total number of participants) x 100
Time Frame
1 year
Title
Number of Subjects Who Experience Seizures
Time Frame
During hospitalization, approximately 4-92 days
Title
Number of Subjects Who Require iNO (Inhaled Nitric Oxide) Use
Time Frame
During hospitalization, approximately 4-92 days
Title
Number of Subjects Who Require ECMO
Description
ECMO (extracorporeal membrane oxygenation) is a technique of providing prolonged cardiac and respiratory support to persons whose heart and lungs are unable to provide an adequate amount of gas exchange or perfusion to sustain life.
Time Frame
During hospitalization, approximately 4-92 days
Title
Number of Subjects Who Require Gastrostomy Tube (G-tube) Feeding
Time Frame
During hospitalization, approximately 4-92 days
Title
Number of Subjects Who Are Discharged on Anti-epileptic Medication
Time Frame
At hospital discharge, approximately 4-92 days

10. Eligibility

Sex
All
Maximum Age & Unit of Time
6 Hours
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael Cotten, MD
Organizational Affiliation
Duke University
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Alabama Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
University of Florida Gainesville
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
MassGeneral Hospital for Children
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Wayne State University Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
University of Florida at Jacksonville
City
Jacksonville
State/Province
North Carolina
ZIP/Postal Code
32209
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
24388332
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.
Results Reference
background
PubMed Identifier
16221780
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.
Results Reference
background
PubMed Identifier
15901860
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.
Results Reference
background

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A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy

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