NCT02612155

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
35

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Mar 2017

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 19, 2015

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 23, 2015

Completed
1.4 years until next milestone

Study Start

First participant enrolled

March 30, 2017

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 5, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 5, 2019

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

August 25, 2020

Completed
Last Updated

May 16, 2024

Status Verified

May 1, 2024

Enrollment Period

2.4 years

First QC Date

November 19, 2015

Results QC Date

August 5, 2020

Last Update Submit

May 14, 2024

Conditions

Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns

Keywords

Hypoxic-ischemic encephalopathyautologous cord blood cellsnewborn infants

Outcome Measures

Primary Outcomes (2)

  • Survival at One Year

    Number of participants alive at one year.

    1 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.

    1 year

Secondary Outcomes (6)

  • Mortality Rate

    1 year

  • 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

  • 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

  • +1 more secondary outcomes

Study Arms (2)

Intervention cell recipients

EXPERIMENTAL

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

Biological: Infusion of autologous cord blood

Placebo recipients

PLACEBO COMPARATOR

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

Biological: Placebo

Interventions

Infusion of autologous cord bloodBIOLOGICAL

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 cell recipients
PlaceboBIOLOGICAL

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.

Placebo recipients

Eligibility Criteria

Age0 Hours - 6 Hours
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Related Publications (3)

  • 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.

    PMID: 24388332BACKGROUND

  • 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.

    PMID: 16221780BACKGROUND

  • 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.

    PMID: 15901860BACKGROUND

MeSH Terms

Conditions

Lymphoma, FollicularHypoxia-Ischemia, Brain

Condition Hierarchy (Ancestors)

Lymphoma, Non-HodgkinLymphomaNeoplasms by Histologic TypeNeoplasmsLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesBrain IschemiaCerebrovascular DisordersBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHypoxia, BrainVascular DiseasesCardiovascular DiseasesHypoxiaSigns and Symptoms, RespiratorySigns and SymptomsPathological Conditions, Signs and Symptoms

Results Point of Contact

Title
Dr. Kim Fisher, Ph.D., FNP-BC
Organization
Duke University
kimberley.fisher@duke.edu
919-681-4913

Study Officials

  • Michael Cotten, MD

    Duke University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Associate Professor

Study Record Dates

First Submitted

November 19, 2015

First Posted

November 23, 2015

Study Start

March 30, 2017

Primary Completion

August 5, 2019

Study Completion

August 5, 2019

Last Updated

May 16, 2024

Results First Posted

August 25, 2020

Record last verified: 2024-05

Locations

US(1)