Moderate or Severe Hypoxic-ischemic Encephalopathy in NewbornsPhase 2(HIE)NCT02612155
A Multi-site Study of Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy
A Phase 2 study testing Infusion of autologous cord blood in people with Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns, run by Michael Cotten at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 6 hours
- Conditions the study is for
- Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
- What is being tested?
- Infusion of autologous cord blood
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Mar 2017to 5 Aug 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 6 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Medical Center
Durham, North Carolina, 27710
About this study
Official title: A Phase II Multi-site Study of Autologous Cord Blood Cells for Hypoxic (HIE)
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.
Read the 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.
- Being tested
- Infusion of autologous cord blood, Placebo
- Keywords
- Hypoxic-ischemic encephalopathy, autologous cord blood cells, newborn infants
- Medical subject headings
- Lymphoma, Follicular, Hypoxia-Ischemia, Brain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival at One Year
Number of participants alive at one year.
Measured: 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.
Measured: 1 year
Secondary outcomes (6)
Mortality Rate
(Number of participants who died/total number of participants) x 100
Measured: 1 year
Number of Subjects Who Experience Seizures
Measured: During hospitalization, approximately 4-92 days
Number of Subjects Who Require iNO (Inhaled Nitric Oxide) Use
Measured: During hospitalization, approximately 4-92 days
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.
Measured: During hospitalization, approximately 4-92 days
Number of Subjects Who Require Gastrostomy Tube (G-tube) Feeding
Measured: During hospitalization, approximately 4-92 days
Number of Subjects Who Are Discharged on Anti-epileptic Medication
Measured: At hospital discharge, approximately 4-92 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2015 (estimated)
- Study start30 Mar 2017
- Primary completion5 Aug 2019
- Study completion5 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Moderate or Severe Hypoxic-ischemic Encephalopathy in NewbornsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Michael Cotten
- Sponsor type
- University, hospital or other
- Collaborators
- The Robertson Foundation
- Responsible party
- Sponsor-investigator, Michael Cotten, Duke University
- Investigators
- Michael Cotten, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02612155
- Sponsor's protocol ID
- Pro00066647
- Acronym
- (HIE)
- Status last verified
- May 2024
- First submitted
- 19 Nov 2015
- Last updated
- 16 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 24388332Shankaran 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.
PubMed 16221780Escolar 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.
PubMed 15901860
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Multi-site Study of Autologous Cord Blood Cells for Hypoxic (HIE)
- ClinicalTrials.gov ID
- NCT02612155
- Acronym / sponsor ID
- (HIE) / Pro00066647
- Keywords
- Hypoxic-ischemic encephalopathy, autologous cord blood cells, newborn infants
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Michael CottenUniversity, hospital or other
- Collaborators
- The Robertson Foundation
- Study start
- 30 Mar 2017 (actual)
- Primary completion
- 5 Aug 2019 (actual)
- Study completion
- 5 Aug 2019 (actual)
- First posted
- 23 Nov 2015 (estimated)
- Results
- Posted 25 Aug 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention cell recipients | Experimental | Biological: Infusion of autologous cord bloodExperimental: 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 |
| Placebo recipients | Placebo comparator | Biological: PlaceboControl: 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 |
Who may qualify
- Ages
- 0 to 6 hours
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Infusion of autologous cord bloodInfants 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. | Biological | Intervention cell recipients |
| PlaceboInfants 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. | Biological | Placebo recipients |
Primary outcome
Survival at One Year
Time frame: 1 year
Number of participants alive at one year.
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.
6 secondary outcomes
Mortality Rate
Time frame: 1 year
Number of Subjects Who Experience Seizures
Time frame: During hospitalization, approximately 4-92 days
Number of Subjects Who Require iNO (Inhaled Nitric Oxide) Use
Time frame: During hospitalization, approximately 4-92 days
Number of Subjects Who Require ECMO
Time frame: During hospitalization, approximately 4-92 days
Number of Subjects Who Require Gastrostomy Tube (G-tube) Feeding
Time frame: During hospitalization, approximately 4-92 days
Number of Subjects Who Are Discharged on Anti-epileptic Medication
Time frame: At hospital discharge, approximately 4-92 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 24388332Cotten 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.
- Background: PubMed 16221780Shankaran 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.
- Background: PubMed 15901860Escolar 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.
Related trials for Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns
RecruitingPhase 2/3
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Undergoing Therapeutic Hypothermia
Hypoxic-Ischemic Encephalopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Cotten, MD principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Infusion of autologous cord blood in people with Moderate or Severe Hypoxic-ischemic Encephalopathy in Newborns, run by Michael Cotten at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Aug 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival at One Year (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02612155, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2024.