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Trial record 1 of 1 for:    A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy
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A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy ((HIE))

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ClinicalTrials.gov Identifier: NCT02612155
Recruitment Status : Completed
First Posted : November 23, 2015
Results First Posted : August 25, 2020
Last Update Posted : August 25, 2020
Sponsor:
Collaborator:
The Robertson Foundation
Information provided by (Responsible Party):
Michael Cotten, Duke University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
Interventions Biological: Infusion of autologous cord blood
Biological: Placebo
Enrollment 35
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Period Title: Overall Study
Started 17 18
Completed 14 15
Not Completed 3 3
Reason Not Completed
Death             1             1
Lost to Follow-up             2             2
Arm/Group Title Intervention Cell Recipients Placebo Recipients Total
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Total of all reporting groups
Overall Number of Baseline Participants 17 18 35
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 18 participants 35 participants
<=18 years
17
 100.0%
18
 100.0%
35
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 18 participants 35 participants
Female
8
  47.1%
9
  50.0%
17
  48.6%
Male
9
  52.9%
9
  50.0%
18
  51.4%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 18 participants 35 participants
Hispanic or Latino
1
   5.9%
1
   5.6%
2
   5.7%
Not Hispanic or Latino
16
  94.1%
17
  94.4%
33
  94.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 18 participants 35 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
6
  35.3%
8
  44.4%
14
  40.0%
White
8
  47.1%
7
  38.9%
15
  42.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
3
  17.6%
3
  16.7%
6
  17.1%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 17 participants 18 participants 35 participants
17
 100.0%
18
 100.0%
35
 100.0%
1.Primary Outcome
Title Survival at One Year
Hide Description Number of participants alive at one year.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
16
  94.1%
17
  94.4%
2.Primary Outcome
Title Number of Participants With Bayley III Scores in All Three Domains > or Equal to 85
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with known one year outcome.
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 14 15
Measure Type: Count of Participants
Unit of Measure: Participants
12
  85.7%
6
  40.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Intervention Cell Recipients, Placebo Recipients
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.06
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
3.Secondary Outcome
Title Mortality Rate
Hide Description (Number of participants who died/total number of participants) x 100
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 17 18
Measure Type: Number
Unit of Measure: percentage of participants
5.88 5.56
4.Secondary Outcome
Title Number of Subjects Who Experience Seizures
Hide Description [Not Specified]
Time Frame During hospitalization, approximately 4-92 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
6
  35.3%
6
  33.3%
5.Secondary Outcome
Title Number of Subjects Who Require iNO (Inhaled Nitric Oxide) Use
Hide Description [Not Specified]
Time Frame During hospitalization, approximately 4-92 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
3
  17.6%
2
  11.1%
6.Secondary Outcome
Title Number of Subjects Who Require ECMO
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
7.Secondary Outcome
Title Number of Subjects Who Require Gastrostomy Tube (G-tube) Feeding
Hide Description [Not Specified]
Time Frame During hospitalization, approximately 4-92 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
2
  11.8%
3
  16.7%
8.Secondary Outcome
Title Number of Subjects Who Are Discharged on Anti-epileptic Medication
Hide Description [Not Specified]
Time Frame At hospital discharge, approximately 4-92 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

Overall Number of Participants Analyzed 17 18
Measure Type: Count of Participants
Unit of Measure: Participants
2
  11.8%
1
   5.6%
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Intervention Cell Recipients Placebo Recipients
Hide Arm/Group 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

Infusion of autologous cord blood: 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.

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

Placebo: 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.

All-Cause Mortality
Intervention Cell Recipients Placebo Recipients
Affected / at Risk (%) Affected / at Risk (%)
Total   1/17 (5.88%)   1/18 (5.56%) 
Hide Serious Adverse Events
Intervention Cell Recipients Placebo Recipients
Affected / at Risk (%) Affected / at Risk (%)
Total   1/17 (5.88%)   2/18 (11.11%) 
Gastrointestinal disorders     
Necrotizing Enterocolitis *  0/17 (0.00%)  1/18 (5.56%) 
General disorders     
Death neonatal *  1/17 (5.88%)  1/18 (5.56%) 
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Intervention Cell Recipients Placebo Recipients
Affected / at Risk (%) Affected / at Risk (%)
Total   1/17 (5.88%)   3/18 (16.67%) 
Cardiac disorders     
Hypertension *  0/17 (0.00%)  1/18 (5.56%) 
Infections and infestations     
Cord blood positive culture *  1/17 (5.88%)  0/18 (0.00%) 
Nervous system disorders     
Seizures *  0/17 (0.00%)  2/18 (11.11%) 
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Kim Fisher, Ph.D., FNP-BC
Organization: Duke University
Phone: 919-681-4913
EMail: kimberley.fisher@duke.edu
Layout table for additonal information
Responsible Party: Michael Cotten, Duke University
ClinicalTrials.gov Identifier: NCT02612155    
Other Study ID Numbers: Pro00066647
First Submitted: November 19, 2015
First Posted: November 23, 2015
Results First Submitted: August 5, 2020
Results First Posted: August 25, 2020
Last Update Posted: August 25, 2020