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Hypoxic-ischemic EncephalopathyPhase 1/2NEATONCT01913340

Neonatal Erythropoietin And Therapeutic Hypothermia Outcomes in Newborn Brain Injury (NEATO)

A Phase 1/2 study testing Erythropoietin and Normal saline in people with Hypoxic-ischemic Encephalopathy and Neonatal Encephalopathy, run by University of California, San Francisco at 7 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Hypoxic-ischemic encephalopathy (HIE), a condition of reduced blood and oxygen flow to a baby's brain near the time of birth, may cause death or neurologic disability. Cooling therapy (hypothermia) provides some protection, but about half of affected infants still have a poor outcome. Read more about this study

Who is it for?
Ages 30 minutes to 24 hours
What is being tested?
Erythropoietin and Normal saline
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
50 participants
Where
7 sitesUnited States
Study dates
Sep 2013to Sep 2016
Phase
Phase 1/2
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 30 minutes to 24 hours
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Newborns ≥ 36 weeks gestation, < 23 hours of age at time of consent, must meet all 3 Inclusion Criteria to be eligible for the study:
  • Perinatal depression = at least one of the following: a) Apgar ≤5 at 10 min or b) required resuscitation (endotracheal or mask ventilation, or chest compressions) at 10 min or c) pH < 7.0 or base deficit ≥15 in cord, arterial, or venous blood obtained at <60 min of age;
  • Moderate to severe encephalopathy = at least 3 of 6 modified Sarnat criteria present between 1-6 h of birth: a) reduced level of consciousness; b) decreased spontaneous activity; c) hypotonia; d) decreased suck; e) decreased Moro reflex; or f) respiratory distress including periodic breathing or apnea; and
  • Hypothermia = passive or active cooling begun by 6 hours of age.

Who cannot join

  • Intrauterine growth restriction (BW <1800 g);
  • Major congenital malformation; suspected genetic syndrome, metabolic disorder or TORCH infection;
  • Head circumference < 2 SD for gestation;
  • Infant for whom withdrawal of supportive care is being considered; or
  • Anticipated inability to collect primary endpoint at 12 months of age.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites

  • Arkansas Children's Hospital Research Institute

    Little Rock, Arkansas, 72202

  • Stanford University

    Palo Alto, California

  • UCSF

    San Francisco, California, 94143

  • Kaiser Permanente, Santa Clara

    Santa Clara, California

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Washington University

    St Louis, Missouri, 63110

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: Neonatal Erythropoietin And Therapeutic Hypothermia Outcomes Study

Hypoxic-ischemic encephalopathy (HIE), a condition of reduced blood and oxygen flow to a baby's brain near the time of birth, may cause death or neurologic disability. Cooling therapy (hypothermia) provides some protection, but about half of affected infants still have a poor outcome. This clinical trial will determine if the drug erythropoietin, given with hypothermia, is safe to use as a treatment that may further reduce the risk of neurologic deficits after HIE.

Read the detailed description

This phase I/II clinical trial is designed to demonstrate:

  1. The feasibility of recruiting, enrolling and following 50 patients with moderate to severe HIE at 5 sites, while meeting specified recruitment and follow-up target goals.
  2. The safety of high-dose Epo therapy in neonates with HIE with respect to systemic organ function and general growth parameters.
  3. The value of brain MRI/MRS performed at 4-7 days of age as a biomarker of motor function at 12 months of age.
Being tested
Erythropoietin, Normal saline
Keywords
erythropoietin, therapeutic hypothermia
Medical subject headings
Hypoxia-Ischemia, Brain, Asphyxia Neonatorum

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ErythropoietinDrug

    Also called Procrit

    1000 U/kg/dose IV x 5 doses

  • Normal salineDrug

    placebo: NS IV x 5 doses

Study groups in NCT01913340
Study groupRoleWhat they receive
ErythropoietinActive comparator1000 U/kg/dose x 5 doses
Receives: Drug: Erythropoietin
Normal salinePlacebo comparatorNot described
Receives: Drug: Normal saline

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Markers of Organ Function

    The investigators will monitor organ function and adverse events until hospital discharge from the neonatal intensive care unit

    Measured: Participants will be followed for the duration of hospital stay, an expected average of 2 weeks

Secondary outcomes (1)

  1. Alberta Infant Motor Scale (AIMS)

    The AIMS consists of 58 items, including 4 positions: prone (21 items), supine (9 items), sitting (12 items), \& standing (16 items). Each item is scored as 'observed' or 'not observed'. Total score range is 0-58, scored as percentile ranks after a raw score is obtained and plotted against age at testing, based on validated norms.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Aug 2013 (estimated)
  2. Study startSep 2013
  3. Primary completionJan 2016
  4. Study completionSep 2016

Resources

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 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01913340
Sponsor's protocol ID
P0055603-01
Acronym
NEATO
Status last verified
Jun 2020
First submitted
29 Jul 2013
Last updated
13 Jul 2020
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Wu YW, Bauer LA, Ballard RA, Ferriero DM, Glidden DV, Mayock DE, Chang T, Durand DJ, Song D, Bonifacio SL, Gonzalez FF, Glass HC, Juul SE. Erythropoietin for neuroprotection in neonatal encephalopathy: safety and pharmacokinetics. Pediatrics. 2012 Oct;130(4):683-91. doi: 10.1542/peds.2012-0498. Epub 2012 Sep 24.

    PubMed 23008465

  • Wu YW, Mathur AM, Chang T, McKinstry RC, Mulkey SB, Mayock DE, Van Meurs KP, Rogers EE, Gonzalez FF, Comstock BA, Juul SE, Msall ME, Bonifacio SL, Glass HC, Massaro AN, Dong L, Tan KW, Heagerty PJ, Ballard RA. High-Dose Erythropoietin and Hypothermia for Hypoxic-Ischemic Encephalopathy: A Phase II Trial. Pediatrics. 2016 Jun;137(6):e20160191. doi: 10.1542/peds.2016-0191. Epub 2016 May 2.

    PubMed 27244862

  • Mulkey SB, Ramakrishnaiah RH, McKinstry RC, Chang T, Mathur AM, Mayock DE, Van Meurs KP, Schaefer GB, Luo C, Bai S, Juul SE, Wu YW. Erythropoietin and Brain Magnetic Resonance Imaging Findings in Hypoxic-Ischemic Encephalopathy: Volume of Acute Brain Injury and 1-Year Neurodevelopmental Outcome. J Pediatr. 2017 Jul;186:196-199. doi: 10.1016/j.jpeds.2017.03.053. Epub 2017 Apr 26.

    PubMed 28456387

  • Darrah J, Bartlett D, Maguire TO, Avison WR, Lacaze-Masmonteil T. Have infant gross motor abilities changed in 20 years? A re-evaluation of the Alberta Infant Motor Scale normative values. Dev Med Child Neurol. 2014 Sep;56(9):877-81. doi: 10.1111/dmcn.12452. Epub 2014 Mar 29.

    PubMed 24684556

  • Massaro AN, Wu YW, Bammler TK, Comstock B, Mathur A, McKinstry RC, Chang T, Mayock DE, Mulkey SB, Van Meurs K, Juul S. Plasma Biomarkers of Brain Injury in Neonatal Hypoxic-Ischemic Encephalopathy. J Pediatr. 2018 Mar;194:67-75.e1. doi: 10.1016/j.jpeds.2017.10.060.

    PubMed 29478510

  • Wu YW, Goodman AM, Chang T, Mulkey SB, Gonzalez FF, Mayock DE, Juul SE, Mathur AM, Van Meurs K, McKinstry RC, Redline RW. Placental pathology and neonatal brain MRI in a randomized trial of erythropoietin for hypoxic-ischemic encephalopathy. Pediatr Res. 2020 Apr;87(5):879-884. doi: 10.1038/s41390-019-0493-6. Epub 2019 Jul 1.

    PubMed 31261373

  • Massaro AN, Wu YW, Bammler TK, MacDonald JW, Mathur A, Chang T, Mayock D, Mulkey SB, van Meurs K, Afsharinejad Z, Juul SE. Dried blood spot compared to plasma measurements of blood-based biomarkers of brain injury in neonatal encephalopathy. Pediatr Res. 2019 Apr;85(5):655-661. doi: 10.1038/s41390-019-0298-7. Epub 2019 Jan 19.

    PubMed 30661082

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Erythropoietin and Normal saline in people with Hypoxic-ischemic Encephalopathy and Neonatal Encephalopathy, run by University of California, San Francisco at 7 sites 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 5 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 13 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites 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 combined 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.

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 13 Jul 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?

Markers of Organ Function (time frame: Participants will be followed for the duration of hospital stay, an expected average of 2 weeks). 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 NCT01913340, which also lists 7 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 13 Jul 2020.

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