Neonatal Hypoxic Ischemic EncephalopathyPhase 1NCT00593242
Cord Blood for Neonatal Hypoxic-ischemic Encephalopathy
A Phase 1 study testing infusion of autologous cord blood and Neurodevelopmental outcomes in people with Neonatal Hypoxic Ischemic Encephalopathy, run by Michael Cotten at 1 site in United States.
Trial summary
This is a pilot study to test feasibility of collection, preparation and infusion of a baby's own (autologous)umbilical cord blood in the first 14 days after birth if the baby is born with signs of brain injury. Read more about this study
- Who is it for?
- Ages Up to 14 days
- Conditions the study is for
- Neonatal Hypoxic Ischemic Encephalopathy
- What is being tested?
- infusion of autologous cord blood and Neurodevelopmental outcomes
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2008to Jan 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 Up to 14 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Mothers must have consented for cord blood collection at delivery
- cord blood must be available for extraction of stem cells.
- >34 weeks gestation
- cord or neonatal pH<7.0 or base deficit>16 milliequivalents per liter (mEq/L) or history of acute perinatal event
- either a 10 minute Apgar < 5 or continued need for ventilation.
- All infants must have signs of encephalopathy within 6 hours of age.
Who cannot join
- Inability to enroll by 14 days of age.
- Presence of known chromosomal anomaly.
- Presence of major congenital anomalies.
- Severe intrauterine growth restriction (weight <1800g)
- Infants in extremis for whom no additional intensive therapy will be offered by attending neonatologist.
- Parents refuse consent.
- Attending neonatologist refuses consent.
- Failure to collect the infant's cord blood and/or laboratory unable to process cord blood.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy Study 1. Phase I Study of Feasibility and Safety.
This is a pilot study to test feasibility of collection, preparation and infusion of a baby's own (autologous)umbilical cord blood in the first 14 days after birth if the baby is born with signs of brain injury.
Read the detailed description
The purpose of this pilot study is to evaluate the safety and feasibility of infusions of autologous (the patient's own)umbilical cord blood cells in term gestation newborn infants with hypoxic-ischemic encephalopathy. For this study, infants who have signs of moderate to severe encephalopathy at birth whose mothers have previously consented to providing cord blood cells for the Carolinas Cord Blood Bank or other public or private bank that uses accepted standards for collection and handling of cells, or provided verbal consent for cord blood collection for the possibility of their baby's participation in this trial, can receive their own cord blood cells if an adequate number of cells that meet Carolinas Cord Blood Bank Quality standards are available in the first 14 postnatal days. Study activities also include serial blood draws concurrent with clinically indicated blood draws with a total volume of no more than 5 milliliters (1 teaspoon) from all study related tests. Babies will be followed for neurodevelopmental outcome at 4 - 6 and 9 - 12 months at Duke's Special Infant Care Clinic. MRI's will be obtained per clinical routine and results will be analyzed and described in study reports.
- Conditions
- Neonatal Hypoxic Ischemic Encephalopathy
- Being tested
- infusion of autologous cord blood, Neurodevelopmental outcomes
- Keywords
- hypoxic-ischemic encephalopathy, autologous cord blood cells, newborn infants
- Medical subject headings
- 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 for history of moderate to severe hypoxic ischemic encephalopathy in the neonatal period will receive up to 4 infusions of their own volume reduced cord blood cells. The number of doses will be determined by the amount of available cord blood cells. The dose for each infusion is 5x10e7 cells/kg
Neurodevelopmental outcomesOther
historical controls, no experimental intervention, standard therapies of hypoxic ischemic encephalopathy in the newborn period with autologous cord blood
| Study group | Role | What they receive |
|---|---|---|
| infusions | Experimental | infants who arrive at the study site within the first 14 postnatal days and had a history of moderate to severe hypoxic ischemic encephalopathy, and have cells available for infusion that pass Carolinas Cord Blood Bank Quality checks Outcomes will be measured at 22-26 months fby neurodevelopment assessment Receives: Biological: infusion of autologous cord blood |
| historical control | Other | Infants who had moderate to severe hypoxic ischemic encephalopathy in the neonatal period but did not receive autologous cord blood cells. Receives: Other: Neurodevelopmental outcomes |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse event rates occurring in the pilot study population will be compared between the cord blood cell recipients and historical controls.
Measured: during infusions: first 18 postnatal days
Secondary outcomes (2)
Secondary endpoints of this pilot study will include preliminary efficacy as measured by neurodevelopmental function at 4 - 6 months and 9 - 12 months of age
Measured: 1 year
neuroimaging results will be collected and compared with available results from prior trials of therapies in this population, and from a previously collected set of images from normal term newborns through the first year of life.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2008 (estimated)
- Study startJan 2008
- Primary completionJan 2017
- Study completionJan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Neonatal Hypoxic Ischemic EncephalopathyStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Michael Cotten
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Michael Cotten, Duke University
- Investigators
- Charles M Cotten, MD MHS, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00593242
- Sponsor's protocol ID
- Pro00000412
- Other IDs
- Duke NPRI01
- Status last verified
- May 2024
- First submitted
- 2 Jan 2008
- Last updated
- 16 May 2024
- Results posted
- No
- Data monitoring committee
- No
Publications
Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94. doi: 10.1016/j.bbmt.2005.09.016.
PubMed 16443516Kurtzberg J, Lyerly AD, Sugarman J. Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood. J Clin Invest. 2005 Oct;115(10):2592-7. doi: 10.1172/JCI26690.
PubMed 16200191Escolar 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 15901860Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, Allison-Thacker J, Wood S, Wenger DA, Rubinstein P, Hopwood JJ, Krivit W, Kurtzberg J. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004 May 6;350(19):1960-9. doi: 10.1056/NEJMoa032613.
PubMed 15128896McGraw P, Liang L, Escolar M, Mukundan S, Kurtzberg J, Provenzale JM. Krabbe disease treated with hematopoietic stem cell transplantation: serial assessment of anisotropy measurements--initial experience. Radiology. 2005 Jul;236(1):221-30. doi: 10.1148/radiol.2353040716.
PubMed 15987975Cotten 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 24388332
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
- Autologous Cord Blood Cells for Hypoxic Ischemic Encephalopathy Study 1. Phase I Study of Feasibility and Safety.
- ClinicalTrials.gov ID
- NCT00593242
- Acronym / sponsor ID
- Pro00000412
- Conditions
- Neonatal Hypoxic Ischemic Encephalopathy
- Keywords
- hypoxic-ischemic encephalopathy, autologous cord blood cells, newborn infants
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Michael CottenUniversity, hospital or other
- Study start
- Jan 2008
- Primary completion
- Jan 2017 (actual)
- Study completion
- Jan 2017 (actual)
- First posted
- 14 Jan 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a pilot study to test feasibility of collection, preparation and infusion of a baby's own (autologous)umbilical cord blood in the first 14 days after birth if the baby is born with signs of brain injury.
Detailed description
The purpose of this pilot study is to evaluate the safety and feasibility of infusions of autologous (the patient's own)umbilical cord blood cells in term gestation newborn infants with hypoxic-ischemic encephalopathy. For this study, infants who have signs of moderate to severe encephalopathy at birth whose mothers have previously consented to providing cord blood cells for the Carolinas Cord Blood Bank or other public or private bank that uses accepted standards for collection and handling of cells, or provided verbal consent for cord blood collection for the possibility of their baby's participation in this trial, can receive their own cord blood cells if an adequate number of cells that meet Carolinas Cord Blood Bank Quality standards are available in the first 14 postnatal days. Study activities also include serial blood draws concurrent with clinically indicated blood draws with a total volume of no more than 5 milliliters (1 teaspoon) from all study related tests. Babies will be followed for neurodevelopmental outcome at 4 - 6 and 9 - 12 months at Duke's Special Infant Care Clinic. MRI's will be obtained per clinical routine and results will be analyzed and described in study reports.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| infusions | Experimental | Biological: infusion of autologous cord bloodinfants who arrive at the study site within the first 14 postnatal days and had a history of moderate to severe hypoxic ischemic encephalopathy, and have cells available for infusion that pass Carolinas Cord Blood Bank Quality checks Outcomes will be measured at 22-26 months fby neurodevelopment assessment |
| historical control | Other | Other: Neurodevelopmental outcomesInfants who had moderate to severe hypoxic ischemic encephalopathy in the neonatal period but did not receive autologous cord blood cells. |
Who may qualify
- Ages
- Up to 14 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Mothers must have consented for cord blood collection at delivery
- cord blood must be available for extraction of stem cells.
- >34 weeks gestation
- cord or neonatal pH<7.0 or base deficit>16 milliequivalents per liter (mEq/L) or history of acute perinatal event
- either a 10 minute Apgar < 5 or continued need for ventilation.
Key exclusion criteria (8 listed)
- Inability to enroll by 14 days of age.
- Presence of known chromosomal anomaly.
- Presence of major congenital anomalies.
- Severe intrauterine growth restriction (weight <1800g)
- Infants in extremis for whom no additional intensive therapy will be offered by attending neonatologist.
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 for history of moderate to severe hypoxic ischemic encephalopathy in the neonatal period will receive up to 4 infusions of their own volume reduced cord blood cells. The number of doses will be determined by the amount of available cord blood cells. The dose for each infusion is 5x10e7 cells/kg | Biological | infusions |
| Neurodevelopmental outcomeshistorical controls, no experimental intervention, standard therapies of hypoxic ischemic encephalopathy in the newborn period with autologous cord blood | Other | historical control |
Primary outcome
Adverse event rates occurring in the pilot study population will be compared between the cord blood cell recipients and historical controls.
Time frame: during infusions: first 18 postnatal days
2 secondary outcomes
Secondary endpoints of this pilot study will include preliminary efficacy as measured by neurodevelopmental function at 4 - 6 months and 9 - 12 months of age
Time frame: 1 year
neuroimaging results will be collected and compared with available results from prior trials of therapies in this population, and from a previously collected set of images from normal term newborns through the first year of life.
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2024.
- Background: PubMed 16443516Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94. doi: 10.1016/j.bbmt.2005.09.016.
- Background: PubMed 16200191Kurtzberg J, Lyerly AD, Sugarman J. Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood. J Clin Invest. 2005 Oct;115(10):2592-7. doi: 10.1172/JCI26690.
- 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.
- Background: PubMed 15128896Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, Allison-Thacker J, Wood S, Wenger DA, Rubinstein P, Hopwood JJ, Krivit W, Kurtzberg J. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004 May 6;350(19):1960-9. doi: 10.1056/NEJMoa032613.
- Background: PubMed 15987975McGraw P, Liang L, Escolar M, Mukundan S, Kurtzberg J, Provenzale JM. Krabbe disease treated with hematopoietic stem cell transplantation: serial assessment of anisotropy measurements--initial experience. Radiology. 2005 Jul;236(1):221-30. doi: 10.1148/radiol.2353040716.
- Results publication: 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.
- Carolinas Cord Blood Bank web pageLink provided by the sponsor.
Related trials for Neonatal Hypoxic Ischemic Encephalopathy
RecruitingPhase 2
The PROTECT-HIE Pilot Trial
Hypoxic-ischemic Encephalopathy (HIE)
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
Not yet recruiting
Levetiracetam as an Adjuvant Therapy to Hypothermia in Neonatal Asphyxia
Hypoxic Ischemic Encephalopathy of Newborn, Neuro-protection
RecruitingPhase 2
A Dose Escalation Study of Levetiracetam in the Treatment of Neonatal Seizures
Neonatal Seizure, Neonatal Encephalopathy
RecruitingObservational study
Continuous Monitoring of Body Temperature in Neonates During Therapeutic Hypothermia
Therapeutic Hypothermia, Neonatal 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.
Charles M Cotten, MD MHS 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 1 study testing infusion of autologous cord blood and Neurodevelopmental outcomes in people with Neonatal Hypoxic Ischemic Encephalopathy, 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 6 inclusion and 8 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adverse event rates occurring in the pilot study population will be compared between the cord blood cell recipients and historical controls. (time frame: during infusions: first 18 postnatal days). 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 NCT00593242, which also lists 6 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.