Erythropoetin Neuroprotection for Neonatal Cardiac Surgery

NCT ID: NCT00513240

Last Updated: 2020-02-07

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

62 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-09-30

Study Completion Date

2015-09-30

Brief Summary

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Brain problems occur in neonatal open heart surgery with a frequency of 20-70%, seen on neurological examination, brain imaging such as magnetic resonance imaging (MRI), or long term development problems such as learning disorders and hyperactivity syndromes. This study aims to determine if erythropoetin, a natural hormone made in the body, protects the brain from damage when given in high doses before and during neonatal open heart surgery. We will use brain MRI, brain wave tests (EEG), neurological examination, and long term developmental outcome testing to see if erythropoetin is better than salt water injection (placebo) in protecting the brain.

Detailed Description

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Hypothesis: Erythropoetin (EPO) will protect the neonatal brain in the perioperative period for congenital heart surgery.

Using a prospective, randomized, placebo-controlled, double-blinded design, the specific aims of this study are:

1. To determine the effect of perioperative EPO on short and long term neurological outcomes in neonates undergoing cardiac surgery with an optimized cardiopulmonary bypass strategy.
2. To determine EPO tolerability and safety with short term administration.
3. To determine EPO pharmacokinetics in this population.
4. To determine the relationship of neurological monitoring, specifically NIRS, to neurological outcomes with an optimized cardiopulmonary bypass technique in neonates that avoids deep hypothermic circulatory arrest, and to determine if EPO affects this relationship.

Protocol: Neonates undergoing arterial switch, Norwood, or aortic arch advancement/other complete 2 ventricle repair, \>35 weeks gestation and ≥2.0 kg are eligible.

Preop day 1:NIRS for 12-24 hours, neuro exam, and Study drug dose #1: EPO 500 units/kg or saline placebo 12-72 hours before surgery. EPO Pharmacokinetic data for 25-50 consenting patients.

Day of surgery: Brain MRI immediately preop. Anesthesia/CPB per our standard practice (fentanyl 100-200 mcg/kg, midazolam, isoflurane, epsilon-aminocaproic acid, 75 mg/kg IV load to patient and CPB prime, and 75 mg/kg/hr infusion in OR) with ACP guided by TCD, pH stat, hct 30-35, avoid DHCA.

POD #1: Study drug dose #2: EPO 500 units/kg or saline placebo 24 hours after dose #2.

For 72 hours postop, NIRS monitoring. All monitor data collected electronically.

POD #3: Study drug dose #3: EPO 500 units/kg or saline placebo 48 hours after dose #3.

7 days postop: Brain MRI. (pentobarbital IV). Neuro exam before discharge. 3-6 months: Brain MRI immediately before or after 2nd surgery, or as outpatient (IV pentobarb or propofol/midazolam-may use N2O/sevo for induction, cannot intubate if outpatient; OR if cardiac MRI at same time, any indicated anesthetic technique). NIRS x 24h after 2nd surgery.

1,and 3 years: Bayley Scales of Infant Development III. 5 years: Battery of neurodevelopmental tests.

Early primary outcome variable: MRI severity of injury score (decrease by 25%). Late outcome variable Bayley Scales of Infant Development score: improvement by 18% at age 1 years.

Sample size: 60 patients: stratified into 3 groups to give power 0.85, alpha 0.05. Expect to accrue 2-4 patients per month.

Conditions

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Congenital Heart Disease Hypoplastic Left Heart Syndrome Transposition of the Great Arteries Aortic Arch Hypoplasia or Interruption

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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EPO group

Patients randomized to receive the 3 doses of erythropoetin.

Group Type EXPERIMENTAL

Erythropoetin

Intervention Type DRUG

Erythropoetin 500 units/kg IV x 3 : dose 1. 12-72 hours preoperatively, dose 2. Postoperative day #1, 48 hours after separating from cardiopulmonary bypass, and dose 3. postoperative day #3, 48 hours after dose #2

Control group.

Patients randomized to receive 3 doses of normal saline control.

Group Type PLACEBO_COMPARATOR

Normal saline

Intervention Type DRUG

Normal saline placebo in 3 doses:dose 1. 12-72 hours preoperatively, dose 2. Postoperative day #1, 48 hours after separating from cardiopulmonary bypass, and dose 3. postoperative day #3, 48 hours after dose #2.

.

Interventions

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Erythropoetin

Erythropoetin 500 units/kg IV x 3 : dose 1. 12-72 hours preoperatively, dose 2. Postoperative day #1, 48 hours after separating from cardiopulmonary bypass, and dose 3. postoperative day #3, 48 hours after dose #2

Intervention Type DRUG

Normal saline

Normal saline placebo in 3 doses:dose 1. 12-72 hours preoperatively, dose 2. Postoperative day #1, 48 hours after separating from cardiopulmonary bypass, and dose 3. postoperative day #3, 48 hours after dose #2.

.

Intervention Type DRUG

Other Intervention Names

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Procrit Epoetin alpha Saline placebo

Eligibility Criteria

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Inclusion Criteria

* Neonates (\<30 days) undergoing cardiac surgery with cardiopulmonary bypass will be enrolled.

* single ventricle: hypoplastic left heart syndrome or variant undergoing Norwood Stage I or Sano palliation (SV group);
* patients with D-transposition of the great vessels with or without ventricular septal defect (VSD) undergoing arterial switch operation with VSD closure if needed (ASO group); and
* patients with interrupted or hypoplastic aortic arch with intracardiac defects (VSD, ASD, or subaortic stenosis) who are undergoing complete 2- ventricle repair including aortic arch advancement(AAA group), any other 2 ventricle lesion scheduled for complex anatomic repair.

Exclusion Criteria

* Gestational age less than 35 weeks at birth
* Weight less than 2 kg
* Known recognizable dysmorphic syndrome
* Surgery not requiring cardiopulmonary bypass
* Preoperative cardiac arrest requiring chest compressions for greater than 3 minutes
* Inability to enroll the patient greater than 12 hours preoperatively
* Aortic crossclamping is not used
* CPB times are anticipated to be less than 60 minutes
* A nadir temperature on bypass greater than 25° C is planned.
* Presence of known contraindications to EPO administration-sustained systolic blood pressure \>100, hemoglobin .18 g/dL, known allergy to EPO or one of its components
* Platelet count \>600,000 per dL, INR \<0.8.
* Maternal history of major vascular thrombosis, or multiple fetal loss (3 or more spontaneous abortions).
Minimum Eligible Age

1 Day

Maximum Eligible Age

30 Days

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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The Dana Foundation

OTHER

Sponsor Role collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role collaborator

Baylor College of Medicine

OTHER

Sponsor Role lead

Responsible Party

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Dean Andropoulos

Professor, Chief of Pediatric Anesthesiology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Dean B. Andropoulos, M.D.

Role: PRINCIPAL_INVESTIGATOR

Baylor College of Medicine - Texas Children's Hospital

Locations

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Texas Children's Hospital

Houston, Texas, United States

Site Status

Countries

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United States

References

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Kellert BA, McPherson RJ, Juul SE. A comparison of high-dose recombinant erythropoietin treatment regimens in brain-injured neonatal rats. Pediatr Res. 2007 Apr;61(4):451-5. doi: 10.1203/pdr.0b013e3180332cec.

Reference Type BACKGROUND
PMID: 17515870 (View on PubMed)

McPherson RJ, Demers EJ, Juul SE. Safety of high-dose recombinant erythropoietin in a neonatal rat model. Neonatology. 2007;91(1):36-43. doi: 10.1159/000096969. Epub 2006 Nov 10.

Reference Type BACKGROUND
PMID: 17344650 (View on PubMed)

Galli KK, Zimmerman RA, Jarvik GP, Wernovsky G, Kuypers MK, Clancy RR, Montenegro LM, Mahle WT, Newman MF, Saunders AM, Nicolson SC, Spray TL, Gaynor JW. Periventricular leukomalacia is common after neonatal cardiac surgery. J Thorac Cardiovasc Surg. 2004 Mar;127(3):692-704. doi: 10.1016/j.jtcvs.2003.09.053.

Reference Type BACKGROUND
PMID: 15001897 (View on PubMed)

Andropoulos DB, Stayer SA, Diaz LK, Ramamoorthy C. Neurological monitoring for congenital heart surgery. Anesth Analg. 2004 Nov;99(5):1365-1375. doi: 10.1213/01.ANE.0000134808.52676.4D.

Reference Type BACKGROUND
PMID: 15502032 (View on PubMed)

Andropoulos DB, Stayer SA, McKenzie ED, Fraser CD Jr. Regional low-flow perfusion provides comparable blood flow and oxygenation to both cerebral hemispheres during neonatal aortic arch reconstruction. J Thorac Cardiovasc Surg. 2003 Dec;126(6):1712-7. doi: 10.1016/s0022-5223(03)01027-4.

Reference Type BACKGROUND
PMID: 14688677 (View on PubMed)

Maiese K, Li F, Chong ZZ. New avenues of exploration for erythropoietin. JAMA. 2005 Jan 5;293(1):90-5. doi: 10.1001/jama.293.1.90.

Reference Type BACKGROUND
PMID: 15632341 (View on PubMed)

McQuillen PS, Barkovich AJ, Hamrick SE, Perez M, Ward P, Glidden DV, Azakie A, Karl T, Miller SP. Temporal and anatomic risk profile of brain injury with neonatal repair of congenital heart defects. Stroke. 2007 Feb;38(2 Suppl):736-41. doi: 10.1161/01.STR.0000247941.41234.90.

Reference Type BACKGROUND
PMID: 17261728 (View on PubMed)

Chang YS, Mu D, Wendland M, Sheldon RA, Vexler ZS, McQuillen PS, Ferriero DM. Erythropoietin improves functional and histological outcome in neonatal stroke. Pediatr Res. 2005 Jul;58(1):106-11. doi: 10.1203/01.PDR.0000163616.89767.69. Epub 2005 May 5.

Reference Type BACKGROUND
PMID: 15879287 (View on PubMed)

Ballweg JA, Wernovsky G, Gaynor JW. Neurodevelopmental outcomes following congenital heart surgery. Pediatr Cardiol. 2007 Mar-Apr;28(2):126-33. doi: 10.1007/s00246-006-1450-9. Epub 2007 Jan 29.

Reference Type BACKGROUND
PMID: 17265108 (View on PubMed)

Karl TR, Hall S, Ford G, Kelly EA, Brizard CP, Mee RB, Weintraub RG, Cochrane AD, Glidden D. Arterial switch with full-flow cardiopulmonary bypass and limited circulatory arrest: neurodevelopmental outcome. J Thorac Cardiovasc Surg. 2004 Jan;127(1):213-22. doi: 10.1016/j.jtcvs.2003.06.001.

Reference Type BACKGROUND
PMID: 14752433 (View on PubMed)

Wypij D, Newburger JW, Rappaport LA, duPlessis AJ, Jonas RA, Wernovsky G, Lin M, Bellinger DC. The effect of duration of deep hypothermic circulatory arrest in infant heart surgery on late neurodevelopment: the Boston Circulatory Arrest Trial. J Thorac Cardiovasc Surg. 2003 Nov;126(5):1397-403. doi: 10.1016/s0022-5223(03)00940-1.

Reference Type BACKGROUND
PMID: 14666011 (View on PubMed)

Zhu C, Kang W, Xu F, Cheng X, Zhang Z, Jia L, Ji L, Guo X, Xiong H, Simbruner G, Blomgren K, Wang X. Erythropoietin improved neurologic outcomes in newborns with hypoxic-ischemic encephalopathy. Pediatrics. 2009 Aug;124(2):e218-26. doi: 10.1542/peds.2008-3553. Epub 2009 Jul 27.

Reference Type BACKGROUND
PMID: 19651565 (View on PubMed)

Andropoulos DB, Mizrahi EM, Hrachovy RA, Stayer SA, Stark AR, Heinle JS, McKenzie ED, Dickerson HA, Meador MR, Fraser CD Jr. Electroencephalographic seizures after neonatal cardiac surgery with high-flow cardiopulmonary bypass. Anesth Analg. 2010 Jun 1;110(6):1680-5. doi: 10.1213/ANE.0b013e3181dd5a58. Epub 2010 Apr 30.

Reference Type BACKGROUND
PMID: 20435942 (View on PubMed)

Andropoulos DB, Hunter JV, Nelson DP, Stayer SA, Stark AR, McKenzie ED, Heinle JS, Graves DE, Fraser CD Jr. Brain immaturity is associated with brain injury before and after neonatal cardiac surgery with high-flow bypass and cerebral oxygenation monitoring. J Thorac Cardiovasc Surg. 2010 Mar;139(3):543-56. doi: 10.1016/j.jtcvs.2009.08.022. Epub 2009 Nov 11.

Reference Type BACKGROUND
PMID: 19909994 (View on PubMed)

McPherson RJ, Juul SE. Erythropoietin for infants with hypoxic-ischemic encephalopathy. Curr Opin Pediatr. 2010 Apr;22(2):139-45. doi: 10.1097/MOP.0b013e328336eb57.

Reference Type BACKGROUND
PMID: 20090525 (View on PubMed)

Juul SE, McPherson RJ, Bauer LA, Ledbetter KJ, Gleason CA, Mayock DE. A phase I/II trial of high-dose erythropoietin in extremely low birth weight infants: pharmacokinetics and safety. Pediatrics. 2008 Aug;122(2):383-91. doi: 10.1542/peds.2007-2711.

Reference Type BACKGROUND
PMID: 18676557 (View on PubMed)

Fauchere JC, Dame C, Vonthein R, Koller B, Arri S, Wolf M, Bucher HU. An approach to using recombinant erythropoietin for neuroprotection in very preterm infants. Pediatrics. 2008 Aug;122(2):375-82. doi: 10.1542/peds.2007-2591.

Reference Type BACKGROUND
PMID: 18676556 (View on PubMed)

Brown MS, Eichorst D, Lala-Black B, Gonzalez R. Higher cumulative doses of erythropoietin and developmental outcomes in preterm infants. Pediatrics. 2009 Oct;124(4):e681-7. doi: 10.1542/peds.2008-2701. Epub 2009 Sep 28.

Reference Type BACKGROUND
PMID: 19786428 (View on PubMed)

Andropoulos DB, Brady K, Easley RB, Dickerson HA, Voigt RG, Shekerdemian LS, Meador MR, Eisenman CA, Hunter JV, Turcich M, Rivera C, McKenzie ED, Heinle JS, Fraser CD Jr. Erythropoietin neuroprotection in neonatal cardiac surgery: a phase I/II safety and efficacy trial. J Thorac Cardiovasc Surg. 2013 Jul;146(1):124-31. doi: 10.1016/j.jtcvs.2012.09.046. Epub 2012 Oct 23.

Reference Type RESULT
PMID: 23102686 (View on PubMed)

Related Links

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http://TexasChildrensHospital.org

Texas Children's Hospital Web Site

http://www.bcm.edu

Baylor College of Medicine Web Site

http://www.pedsanesthesia.org/ccas/

Congenital Cardiac Anesthesia Society Web Page

http://www.americanheart.org/children.

American Heart Association Web Page--Congenital Heart Disease

http://www.nichd.nih.gov

National Institutes of Health: National Institute of Child Health and Developement Web Site

Other Identifiers

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IND #100011

Identifier Type: OTHER

Identifier Source: secondary_id

0942

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

1R21HD055501-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

Not Assigned

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

R21HD5550101

Identifier Type: -

Identifier Source: org_study_id

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