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Clinical Trials

NICU baby

The Neonatal Perinatal Research Unit was established to explore and disseminate the most up-to-date evidence-based practices for newborn care. At Duke University, we have the unique ability to offer our families access to cutting-edge practices and treatments that are focused on our mission of providing evidence-based practices aimed at improving the care for our babies.

Study Enrollment Guidelines for NICU Care Team

To assess whether or not an infant admitted to the NICU is eligible for an active research study, please click here [PDF, 93KB].

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Our mission is to provide the professional infrastructure and clinical expertise directed toward improving the quality of care and long-term outcomes for our babies. Find out how you can help support our babies and our research.

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Current Active Clinical Trials

 

Use of Donor Cord Blood for Treatment of HIE

Eunice Kennedy Shriver NICHD Neonatal Research Network

​​Babies, Inc with the National Institutes of Health

Additional Trials

 


​Current Active Clinical Trials

Use of Donor Cord Blood for Treatment of HIE

Title: A Phase I Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in newborn infants with moderate or severe hypoxic ischemic neonatal encephalopathy​
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Mandy Marion, BS, MT (ASCP)

About this trial
The purpose of this study is to assess the safety of one and two intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), the first administered in the first 48 postnatal hours, and the second at two months postnatal age, in term and near term infants with moderate to severe neonatal hypoxic-ischemic encephalopathy (HIE).  This is a phase I, prospective, open-label trial designed to assess the safety of one or two intravenous doses of hCT-MSC in newborn infants with moderate to severe HIE who are recipients of therapeutic hypothermia.

This product allows for a readily available ‘off-the-shelf’ cellular intervention product for neonatal brain injury. Duke has been funded by the Duke Clinical & Translational Science Institute to demonstrate the safety of infusion of these cells in a small cohort of newborns both with hypoxic-ischemic encephalopathy. 

 


Eunice Kennedy Shriver NICHD Neonatal Research Network

Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

In 1986, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), part of the National Institutes of Health, initiated the Neonatal Research Network (NRN) to conduct multi-center clinical trials and observational studies in neonatal medicine. The network was created because many of the treatment and management strategies in 1986 had become standards without being properly evaluated. In addition, multiple clinical centers were necessary to provide a large enough population from which appropriate (with adequate statistical power to detect clinically important differences) samples could be drawn. At the same time, NICHD also established a companion Maternal Fetal Medicine Unit (MFMU) Network to do similar research in obstetrics and maternal-fetal medicine.

Learn more

Generic Database: Survey of Morbidity and Mortality Among High Risk Preterm Infants
 

Title: Survey of Morbidity and Mortality Among High Risk Preterm Infants (GDB)
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
The purpose of this project is to provide a registry of baseline and outcome information for high-risk preterm infants, based on data collected in a uniform manner from neonatal intensive care units (NICUs) at major hospitals participating in the Eunice Kennedy Shriver NICHD Neonatal Research Network. This project has been going on for over 25 years, and more than 60,000 babies have participated.

This information represents care at a number of major academic centers. Although centers serve varying populations, the data exemplifies the neonatal morbidity problems of the 1980's to now. These data are used to characterize the infants admitted to the units, to examine the relationships between certain entry characteristics and outcome, to measure trends in incidence of various disease entities, and to provide the basis for hypothesis formulation for future multi-center studies.

Learn more at ClinicalTrials.gov

Generic Database Follow Up: Follow-Up of High Risk Infants
 

Title: Follow Up of High Risk Infants
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
The purpose of this study is to gather information on the growth and development of babies born earlier than 27 weeks gestational age (GA) when the infant is between 22 and 26 months corrected age (age based on their due date). The study also examines the characteristics of families of premature babies, the special support services and early intervention programs utilized by these families, and the impact of a premature baby on the family. All data collected comes from major hospitals participating in the Eunice Kennedy Shriver NICHD Neonatal Research Network.

Learn more at ClinicalTrials.gov


Milrinone in Congenital Diaphragmatic Hernia (CDH)

Title: A Phase II Pilot Trial on Milrinone in Congenital Diaphragmatic Hernia (CDH)
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
This initial pilot study will help us to understand how milrinone works to help the heart and lungs give oxygen to the tissues and other organs in infants with CDH. We are hoping this pilot study will support and look at the possibility and safety of conducting a much larger study.

Milrinone is a medication that is currently approved by the Food and Drug Administration (FDA) for short-term use in adults with heart failure. In a large study involving 238 children, milrinone was shown to help the heart work better after the children had heart surgery. From our past survey, we found out that about 17% of babies with CDH who are patients in some large hospitals are given milrinone as a treatment for CDH. Some doctors use milrinone to help improve blood flow to the lungs in babies. Milrinone may help in CDH, by opening up the blood vessels in the lung which may help the heart and lungs give enough oxygen to tissues and other organs.

Learn more at ClinicalTrials.gov

Darbepoetin Trial to Improve Red Cell Mass and Neuroprotection in Preterm Infants
 

Title: Darbepoetin Trial to Improve Red Cell Mass and Neuroprotection in Preterm Infants
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
The purpose of this trial is to study the use of a potential neuroprotective therapy which involves administering a drug called Darbepoetin (Darbe) to preterm infants born 23 weeks gestation up to 28 6/7 weeks gestation. Infants born this early are at an increased risk for potential neurodevelopment problems. Although a variety of neuroprotective treatment strategies have been evaluated, no specific treatment has been identified to reduce or prevent brain injury in these most vulnerable preterm infants.

It is hoped that this drug will help the body make red blood cells and may also protect the brain and help the brain grow. This study will test learning and development at 2 years of age in infants getting Darbe, compared to infants who did not receive Darbe. All data collected comes from major hospitals participating in the Eunice Kennedy Shriver NICHD Neonatal Research Network.

Learn more at ClinicalTrials.gov

Preemie Hypothermia: Examining the Use of Hypothermia for Premature Infants

Title: A Randomized Trial of Targeted Temperature Management with Whole Body Hypothermia for Moderate and Severe Neonatal Encephalopathy in Premature Infants 33-35 Weeks Gestational Age
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
Neonatal Encephalopathy (NE) is a serious condition, which usually occurs secondary to problems during birth and results when too little oxygen rich blood flows to the brain. Infants with NE have an increased risk of brain injury, long-term developmental problems, and death.

In many neonatal intensive care units across the country, body cooling or hypothermia is standard therapy for full-term babies with NE. These full-term babies are placed on a special blanket to decrease their temperature before they reach 6 hours of age and continue to be cooled for 72 hours. In these babies, body cooling has reduced the number of deaths and the amount of serious disabilities when the babies are evaluated at the age of 18-22 months. While this treatment shows some effectiveness in full-term babies, cooling has not yet been studied in premature babies who develop NE.

Learn more at ClinicalTrials.gov

Evaluation of Healthy Term Infants
 

Title: Evaluation of Healthy Term Infants to Define Impairment Thresholds and Promote Unbiased Assessments of High-Risk Infants: A Quality Assurance Measure for Network Follow-Up Assessments
Principal Investigator: William Malcolm, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source:  National Institute of Child Health & Human Development (NICHD)

About this trial
The purpose of this study is to compare the developmental test results of healthy term infants and premature infants who are now 2 years old. Children who are born too early (premature) can have problems with their learning and development as they grow. We follow our premature infants until they are about 2 years old to see if the treatments they had as newborns have been helpful. We use tests, including one called the “Bayley,” to see how these children are doing with their development.

At the study visit, your child will have a physical exam, including height and weight measurements. Also, a developmental test called a Bayley exam will be completed. You, your child, and the Bayley examiner (a research person who is specially trained to give the Bayley exam) will be in a room that has special types of toys. The examiner will use these toys to test your child’s development. You will also be asked various questions about your baby's health and development since birth.

We need children who were born healthy at full term to take some of the same tests so we can compare the premature infants to them.

Moderately Preterm Infants with Caffeine at Home for Apnea (MoCHA) Trial
 

Title: Randomized Controlled Trial of Home Therapy with Caffeine Citrate in Moderately Preterm Infants with Apnea of Prematurity
Principal Investigator: William Malcolm, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source:  National Institute of Child Health & Human Development (NICHD)

About this trial
Apnea of prematurity is a common problem in babies who are born before their due date.  Apnea is when babies stop breathing for a short period of time.  Apnea may also cause slowing of their heart rate (bradycardia).  Babies usually stop having apnea around the time of their due date. However, for some babies, apnea may last past this time and may delay discharge.

The purpose of this research study is to find out if using caffeine until discharge and after discharge at home will help us send babies home sooner. Caffeine is a medicine commonly used to treat apnea of prematurity. Babies who were born between 29 and 33 weeks gestational age and have a history of apnea will be part of this study.

Learn more at ClinicalTrials.gov

PDA Trial
 

Title: Management of the Patent Ductus Arteriosus in Premature Infants Trial (PDA Trial)
Principal Investigator: Jennifer Peterson, MD
Research Coordinator: Joanne Finkle, RN
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
The purpose of this study is to estimate the risks and benefits of active treatment verses expectant management of a symptomatic patent ductus arteriosus (sPDA) in premature infants. Because of the uncertainty about which approach is optimal, there is wide practice variation.  All groups conclude that new controlled, randomized trials to reexamine the benefits and risks of different approaches to PDA treatment are warranted.  This study will include babies 22-28 weeks’ gestational age at birth who have a symptomatic PDA between 48 hours and 21 days of age.  Babies in the active care group will receive one of two common medicines (indomethacin or ibuprofen).  Babies in the expectant management group will receive medicine only if the symptomatic PDA is large and the baby is on a breathing machine.

Learn more on ClinicalTrials.gov

Cycled Phototherapy to Control Bilirubin
 

Title: Cycled Phototherapy: A Safer Effective Method to Control the Serum Bilirubin of Extremely Premature Infants?
Principal Investigator: William Malcolm, MD
Research Coordinator: Joanne Finkle, RN
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
Jaundice (yellow skin with elevated bilirubin amounts) is common in premature babies. It is prevented or treated with phototherapy (special lights). These lights are usually left on all the time (continuous phototherapy) until the bilirubin decreases. The purpose of this study is to test whether using cycled phototherapy (with the lights on for only part of each hour) verses continuous phototherapy will improve the survival of extremely premature babies and keep the bilirubin amounts in a safe range. Babies born less than 27 weeks gestational age and less than or equal to 750 grams may qualify for this study. Babies eligible and consented will be randomized to either continuous or cycled phototherapy. Babies will receive phototherapy in their assigned group until the baby no longer requires light therapy.  Babies will return to the Special Infant Care Clinic at Duke for developmental testing at 2 years of age.

Eating, Sleeping, Consoling for Neonatal Opioid Withdrawal (ESC-NOW) Trial

Title: Eating, Sleeping, Consoling for Neonatal Opioid Withdrawal (ESC-NOW):  a Function-Based Assessment and Management Approach Principal Investigator: William Malcolm, MD
Research Coordinator: Joanne Finkle, RN
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
Neonatal Opioid withdrawal syndrome (NOWS) is something that affects a lot of babies and their families. We are trying to learn as much as we can about the best way to care for babies with NOWS. Babies who, while growing inside their mothers, have been exposed to opioids, can have NOWS. The purpose of this study is to learn more about which method(s) might work better than others for treating NOWS.  Specifically, the research team will be looking to see how different treatment methods for NOWS affect the growth and development of babies after they go home from the hospital. Infants born equal to or greater than 36 weeks gestational age and managed for NOWS may be eligible. Families participating will complete questionnaires at several time points during the first two years of life and will return for to the Special Infant Care Clinic at Duke for developmental testing at 2 years of age.


Baebies, Inc with the National Institutes of Health

Point-of-Care Newborn Screening: Hypoglycemia

Title: Multiplex Platform for Point-of-Care Newborn Screening of Hypoglycemia
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Grace Jefferson, MS
Funding Support: Baebies, Inc.

About this trial
Hypoglycemia is the most common metabolic disorder in newborns and places the neonate at increased risk of seizures and permanent brain injury. The etiology of severe neonatal hypoglycemia -- which affects around 25,000 newborns each year in the U.S. -- is complex, with underlying causes including hyperinsulinism, cortisol and/or growth hormone (GH) deficiencies, and defects in glycogen metabolism. Comprehensive evaluation of these physiological pathways is essential to guide appropriate, effective medical interventions. However, the current panel of laboratory assays (laboratory procedures to measure the amount or quantity of a specific component) requires relatively large volumes (3 mL or more) of whole blood, making newborn assessment challenging, particularly in preterm newborns. More importantly, routine turnaround times for these assays may be 48 hours or more, leading to critical delays in life-saving treatment.

The overall goal of this Fast Track SBIR (Small Business Innovation Research) project is to develop a comprehensive near patient digital microfluidic system, FINDER, for the rapid assessment of severe, persistent hypoglycemia disorders in newborns using microliter volumes of whole blood. FINDER will accept single use cartridges and will multiplex 6 assays for the detection of insulin, cortisol, growth hormone, glucose, β-hydroxybutyrate and free fatty acids using less than 50 μL of whole blood for all assays. Run time will be approximately 45 minutes. The team at Baebies will collaborate with leading clinical investigators in Neonatology (Dr. Michael Cotten) and Pediatric Endocrinology (Dr. Michael Freemark) at Duke University on this project. The system proposed herein addresses a critical unmet clinical need for rapid testing at the time of hypoglycemia; the final product will be substantially differentiated from current laboratory based assays in terms of lower blood volume, lower cost and faster time-to-result.

Point-of-Care Newborn Screening: Hyperbilirubinemia 
 

Title: Multiplex Platform for Point-of-Care Newborn Screening of Hyperbilirubinemia
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Grace Jefferson, MS
Funding Support: Baebies, Inc.

About this trial
Neonatal jaundice is a frequent condition which, when unmonitored or untreated, can lead to severe neonatal hyperbilirubinemia (HBR) and associated negative outcomes. Following guidelines set by the American Academy of Pediatrics, most babies in the U.S. are screened for hyperbilirubinemia by testing bilirubin levels as part of routine newborn screening, which identifies those neonates most at-risk for progressive HBR.

This study aims to develop a comprehensive and biochemically based hyperbilirubinemia testing panel using digital microfluidics. Availability of such a panel would lead to earlier intervention and reduce the incidence of severe neonatal hyperbilirubinemia and resultant risk of bilirubin-induced neurological damage (BIND).

Point-of-Care Newborn Screening: Thyroid Function

Title: Expanding Thyroid Testing in High Risk Newborns
Principal Investigator: Michael Freemark, MD
Research Coordinator: Grace Jefferson, MS
Funding Support: Baebies, Inc.

About this trial
Thyroid dysfunction is one of the most common problems of the neonatal period and can result in permanent cognitive disability and other serious complications if not rapidly identified and treated. While newborn thyroid screening in state public health laboratories has revolutionized the identification of children with severe congenital primary hypothyroidism, this screening does not effectively identify several other common neonatal disorders of thyroid function that may result from preterm birth or maternal hyperthyroidism. Major barriers to comprehensive thyroid testing in newborns currently include the relatively large volume of blood needed for multiple tests, long turn-around times and a need for repeated testing to establish an appropriate treatment plan. These limitations are further complicated by the lack of clear guidelines for identification and treatment of hypothyroidism in preterm newborns.

This study aims to develop a comprehensive and biochemically based thyroid function testing using digital microfluidics. Availability of such a panel would lead to earlier intervention and reduce the incidence of thyroid dysfunction.

Point-of-Care Newborn Global Screening
 

Title: A Low Blood Volume Platform for Global Newborn Screening of Common, Treatable Conditions
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Anne Baez
Funding Support: Baebies, Inc. with National Institutes of Health

About this trial
Severe hyperbilirubinemia, glucose-6-phosphate dehydrogenase (G6pd) deficiency, congenital hyperthyroidism, and classic galactosemia are potentially life-threatening conditions of the neonatal period that can cause permanent mental disability or death if not promptly treated. Routine newborn screening (NBS) has drastically reduced morbidity and mortality resulting from these conditions in developed nations, however fewer than 20% of newborns worldwide currently receives testing for these common conditions. The centralized NBS paradigm that works well in developed countries requires high capital and operating costs and is not easily transferrable to highly populated countries.

This study aims to introduce newborn screening to regions lacking the infrastructure necessary to implement central laboratory based testing by developing a flexible (low to medium) throughput newborn screening platform suitable for use in moderate complexity (hospital) settings.

Point-of-Care Newborn Screening: Acute Kidney Injury Analysis

Title: Near Patient Acute Kidney Injury Biomarker Analysis in Critically Ill Neonates Phase I (RFA-HD-18-028)
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Grace Jefferson, MS
Funding Support: Baebies, Inc. with National Institutes of Health

About this trial
Acute kidney injury (AKI) contributes to mortality and morbidity in critically ill neonates. The identification and treatment of AKI are currently based on serum creatinine (sCr) levels; however, mounting evidence suggests that sCr alone is insufficient to predict or prevent renal injury. sCr is in fact a measure of renal function, not injury; sCr levels usually rise many hours after a renal injury, require a substantial magnitude of renal dysfunction for detection and are diminished by fluid overload. Measurement of sCr levels alone is insufficient to achieve this goal. Earlier detection of acute kidney injury, coupled with improved characterization of the underlying etiology, may halt the progression to severe kidney injury and improve long term damage.

The purpose of this study is to optimize and validate a panel of assays for the patient with minimally invasive analysis of multiple AKI biomarkers using the Baebies FINDER platform. Availability of such a panel would lead to earlier intervention and targeted therapy to those with early AKI.

A Comprehensive Newborn Screening Solution of Duchenne and Congenital Muscular Dystrophies
 

Title: A Comprehensive Newborn Screening Solution for Duchenne and Congenital Muscular Dystrophies
Principal Investigator: Edward Smith, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: Baebies, Inc. with National Institutes of Health

About this trial
The goal of this project is to develop a complete testing solution for efficient newborn screening of Duchenne muscular dystrophy (DMD) and Congenital muscular dystrophies (CMD) from dried blood spot (DBS) specimens. Patients eligible for this study will include patients who have been diagnosed with DMD and CMD and who are being treated at Duke University Medical Center.  Enrolled patients will have blood spot samples collected via fingerstick.

 


Additional Trials

Analyzing Retinal Microanatomy in Retinopathy of Prematurity to Improve Care
 

Title: Analyzing Retinal Microanatomy in Retinopathy of Prematurity to Improve Care
Principal Investigator: Cynthia Toth, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institutes of Health (NIH)

About this trial
The purpose of this study is to evaluate the normal and abnormal microanatomy of the developing retina and optic nerve in very preterm infants and to determine its relationship to brain development, neurodevelopment, visual function and advancement of retinopathy of prematurity.

Infants who are born preterm often have an incompletely developed retina (the light-sensing back layer inside the eye) and may develop Retinopathy of Prematurity (ROP) for which regular eye examinations are required as standard care. The purpose of this study is to determine whether images taken of the retina using a special type of research camera called Swept Source Optical Coherence Tomography (SSOCT), can help predict which preterm infants will go on to require treatment for severe ROP. The imaging may also help predict the final vision of these eyes later on in childhood and the overall development of these children. The SSOCT device to be used in this study is considered “investigational” because this new imaging device is still being tested in research studies and is not approved by the U.S. Food and Drug Administration (FDA).

Learn more at ClinicalTrials.gov

Continued Anticonvulsants after Resolution of Neonatal Seizures
 

Title: Continued Anticonvulsants after Resolution of Neonatal Seizures: A Patient-Centered Comparative Effectiveness Study
Principal Investigator: Monica Lemmon, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: Patient-Centered Outcomes Research Institute (PCORI)

About this trial
The purpose of this study is to see how the treatment of neonatal seizures affects infants’ developmental outcomes and their chances of developing epilepsy. This study will also be investigating the impact on the family of having an infant with seizures.

This study is examining the critical decisions made by practitioners regarding how neonatal seizures are being treated across several institutions. The long-term goal is to improve neurodevelopmental outcomes following seizures in newborns. The objective is to examine whether the duration of treatment with anti-seizure medications has an impact on neurodevelopmental and epilepsy outcomes, as well as parent and family well-being, after neonatal seizures.

Learn more at ClinicalTrials.gov

Investigating Use of Inhaled Nitric Oxide in Neonates
 

Title: The Pattern Registry: Multicenter, Prospectively Defined Observational Registry With Retrospective Data Collection Evaluating Premature and Term-Near-Term Neonates With Pulmonary Hypertension Receiving Inhaled Nitric Oxide via Invasive or Noninvasive Ventilator Support at US Centers.
Principal Investigator: Kamlesh Athavale, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: Mallinkrodt, Inc.

About this trial
The purpose of this registry study is, to collect information on the use of inhaled nitric oxide therapy (INOMAX) in premature and term-near-term neonates for the treatment of acute respiratory related illness, such as persistent pulmonary hypertension of the newborn (PPHN). The information will be used to evaluate impact of INOMAX on participant’s condition, comparing premature and term-near-term infants.

Persistent pulmonary hypertension of the newborn (PPHN) is a serious and sometimes fatal cardiorespiratory complication for infants. PPHN is a clinical syndrome associated with various neonatal cardiorespiratory diseases. Inhaled nitric oxide (INOMAX®) is commonly used for the treatment of neonates with hypoxic respiratory failure (HRF) associated with pulmonary hypertension (PH).

This study is being done to try and answer the question as to whether or not iNO is a safe and effective treatment for infants with serious respiratory complications. The purpose of this registry study is, to collect information on the use of inhaled nitric oxide therapy (INOMAX), as prescribed per standard of care in the treatment of hypoxic respiratory failure/pulmonary hypertension (HRF/PH).

Learn more at ClinicalTrials.gov

Inguinal Hernia Repair in Premature Infants

Title: A Randomized Trial Comparing Inguinal Hernia (IH) Repair In Premature Infants Prior To Versus After NICU Discharge
Principal Investigator: Henry Rice, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
Children born prematurely often are born with an inguinal hernia. This is a hole in the wall of the abdomen that is at risk for having a piece of intestine get stuck. If the intestines become stuck (incarcerate) they may lose their blood flow (strangulate), and significant and life-threatening problems may occur. Standard care for this condition is surgical repair of this inguinal hernia.

Currently there are two common practices for the timing of repair of the hernia. One practice is performing the repair soon before discharge from the NICU. The other option is repair after discharge from the NICU, generally by 5 months after discharge. Both of these treatments approaches are commonly used and are both considered standard of care at Duke; however, there have not been studies to show which approach is better.

There are known risks to either approach, including potentially increased risks of anesthetic related complications or technical complications with repair before discharge (early) which can be life-threatening, as well as potentially increased risks of hernia related complications (incarceration) for repair after discharge (late).

The purpose of this study is to determine which approach (repair before discharge or repair after discharge) is best. The investigators of this study want to know which timing results in the best overall outcomes and fewest complications. Another goal of this study is to develop a set of guidelines that will help doctors and other healthcare professionals figure out the safest time to perform an inguinal hernia repair in premature babies.

Learn more at ClinicalTrials.gov

An Observational Study of Infants with Congenital Diaphragmatic Hernia (CDH)
 

Title: Advancing Clinical Research in Pediatric Surgery: An Observational Study of Infants with Congenital Diaphragmatic Hernia
Principal Investigator: Jen Peterson, MD
Research Coordinator: Anne Baez, BS
Funding Source: Divisional Funds

About this trial
Congenital diaphragmatic hernia (CDH) is an anatomical defect affecting 1 in every 5000 live births. CDH is caused by the diaphragm (the large muscle between the lungs and stomach that helps you breathe) not closing or forming at around 8 weeks gestation. The cause of CDH is unknown. In the past, the likelihood for recurrence in subsequent pregnancies was thought to be slight. Recent papers though have reported familial cases, suggesting the risk for affected siblings to be 2%.

This is a multicenter/multinational, observational registry of children born with CDH. Since inception in 1995, over 3500 children have been entered, from 90 centers represented by 10 countries around the world. The purpose of this Quality Improvement (QI) registry and the CDH Study Group is to collect and analyze information on CDH with the hope that with careful delineation of the natural history of this disease the information will lead to identifying appropriate interventions.

SPASMS: Neonatal Seizure Registry
 

Title: Neonatal Seizure Registry II – Spasm Prediction After Symptomatic Neonatal Seizures
Principal Investigator: Monica Lemmon, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: Pediatric Epilepsy Research Foundation (PERF)

About this trial
Neonatal seizures are common and affect about 16,000 newborns every year in the United States. Parents of infants with seizures indicate major concerns about the risk of developing epilepsy. Our long-term goal is to improve neurodevelopmental outcomes and prevent epilepsy in children with neonatal seizures. The overall objective of this application is to develop a risk classification system for infantile spasms for children who are enrolled into this study. Our central hypothesis is that we can collect the results of imaging done during an infant’s hospitalization, along with their clinical characteristics to predict the risk of infantile spasms happening as the infant grows older.

QPL: List for Infants in the Neonatal Intensive Care Unit

Title: QPL: A Question Prompt List for Infants in the Neonatal Intensive Care Unit
Principal Investigator: Monica Lemmon, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: National Palliative Care Research Center

About this trial
The question prompt list (QPL) is a promising communication tool that guides patient interactions with their healthcare team. We have developed a QPL for use in the intensive care nursery (ICN) using data from families, including semi-structured interviews and recorded family meetings. We then performed acceptability testing of the QPL content with clinicians and parents. Both groups found the tool universally acceptable and would recommend the QPL to other families. We now hope to pilot the QPL with parents of infants at risk of brain injury prior to a planned family conference.

Registry for Vermont Oxford Network

Title: Vermont Oxford Network Database
Principal Investigator: Michael Cotten, MD
Research Coordinator: Anne Baez, BS
Funding Support: Duke Medicine Healthcare System

About this trial
The Vermont Oxford Network is a non-profit voluntary collaboration of health care professionals dedicated to increasing the effectiveness and efficiency of neonatal intensive care through an integrated program of outcomes research, randomized clinical trials and quality improvement projects. In support of its mission, the Network maintains the database that contains information about the care and outcomes of high-risk newborn infants. The Network database:

  • Provides unique, reliable and confidential data to participating units for us in quality management, process improvement, internal audit, and peer review.
  • Provides core data for preparation of randomized clinical trials that focus on quality improvement, outcomes research, and epidemiological studies.
  • Creates the foundations for educational materials and programs for healthcare professionals, policy makers, families of high-risk infants, and the public.

The research program of the Network includes out-comes research and randomized clinical trials. The goal of Network outcomes research is to identify and explain the variations in clinical practice and patient outcomes that are apparent among NICUs. Network trials are designed to answer practical questions of importance to practitioners and families using pragmatic designs that can be integrated into the daily practice of neonatology.

Vaccine Study
 

Title: A Prospective, Randomized, Open-label Clinical Trial to Assess Apnea and Other Adverse Effects Following Administration of 13-valent Conjugate Pneumococcal Vaccine, Diphtheria Toxoid, Tetanus Toxoid, and Acellular Pertussis Vaccine, Hepatitis B vaccine, and Haemophilus influenzae Type B Vaccine in Preterm Infants
Principal Investigator: Rachel Greenberg, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: Centers for Disease Control and Prevention (CDC)

About this trial
Premature infants (<37 weeks gestational age at birth) are at high risk for undervaccination. The Centers for Disease Control and Prevention Advisory Committee on Immunization Practices (ACIP) and the American Academy of Pediatrics have recommended that most premature infants should receive all routinely recommended vaccines at the same chronological age as term infants. Despite this recommendation, premature infants have been shown to be underimmunized at 6 months, 12 months, 24 months, and 36 months chronological age. Infants who are discharged from the neonatal intensive care unit (NICU) after lengths of stay ≥60 days are at particularly high risk, with only approximately 50% up-to-date at the time of discharge. Vaccination delay at this earlier age thus contributes to undervaccination at later ages.

This study will compare infants who are vaccinated according to their chronological age to those that are vaccinated on a delayed scheduled. Infants will be randomized to the standard vaccinated group or to a delayed vaccination group. The study will then monitor any side effects such as changes in respiratory support or the occurrence of apnea (absence of breathing for more than 20 seconds).

Umbilical Cord-Derived Mesenchymal Stromal Cell Product in Newborn Infants with Hypoxic-ischemic Encephalopathy

Title:  A Phase I Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal
Stromal Cell Product, in newborn infants with moderate or severe hypoxic ischemic
neonatal encephalopathy.
Principal Investigator: C. Michael Cotten, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: Clinical and Translational Science Awards /CTSI Grant

About this trial
The purpose of this study is to assess the safety of one or two intravenous infusions of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in infants at least 36 weeks gestational age and born with moderate to severe neonatal hypoxic-ischemic encephalopathy (HIE) requiring whole body cooling therapy. Mesenchymal stromal cells (MSCs) are isolated from umbilical cord tissue donated by healthy mothers delivering full term babies. These cells are cryopreserved and stored in the Robertson CT2 GMP laboratory (Duke University, Durham).  Enrolled infants will receive the first dose of cells in the first 48 postnatal hours. Infants enrolled in cohort 2 will also receive a second dose of cells at two months postnatal age.  The safety of providing hCT-MSC infusion(s) to babies with HIE will be measured by survival at one year of age as well as  neuro and developmental testing performed at 1 year of age.

Central and Peripheral Body Temperature in Very Low Birth Weight (VLBW) Preterm Infants
 

Title: A study examining central & peripheral body temperature in very low birth weight (VLBW) preterm infants during the neonatal period and the relationship to neonatal infection and necrotizing enterocolitis (NEC)
Principal Investigator: Kimberley Fisher, PhD
Research Coordinator: Grace Jefferson, MS
Funding Source: National Institutes of Health (NIH) / National Institute of Nursing Research (NINR)

About this trial
Very low birth weight (VLBW) infants, who are less than 1500 grams and usually less than 32 weeks gestational age (GA) at birth, have inefficient thermoregulation due to immature systems. To decrease morbidity and mortality in VLBW preterm infants, studies have examined the consequences of immature thermoregulation to morbid conditions to generate knowledge that allows clinicians to intervene for better infant outcomes. The purpose of this study is confirm the relationship between longitudinal body temperature (abdominal and foot skin temperature) and infection in very low birth weight infants.

Extremely Preterm Infant Biorepository
 

Title: Extremely Preterm Infant Biorepository
Principal Investigator: Noelle Younge, MD
Research Coordinator: Mandy Marion, BS, MT(ASCP)
Funding Source: Duke University

About this trial
The purpose of this project is to acquire and store biological specimens and clinical data from infants born <28 weeks gestation and admitted to the Duke Intensive Care Nursery (ICN) or Duke’s Newborn Nursery (NBN) to use for studies to better understand mechanisms that contribute to short and long term health outcomes for high risk neonates. Healthy full-term infants born at Duke University Hospital will be enrolled as a control group. With parental consent, samples will be collected from the infants during their birth hospitalization and subsequent follow-up visits in the Duke Health System through school age. These de-identified, stored samples will be used for current and future research studies investigating the mechanisms and predictors of short- and long-term health outcomes, including but not limited to growth failure, morbidities, treatments, and biomarkers on subsequent growth, neurodevelopment, and health outcomes. Sample collection will continue from infancy through childhood.

Pharmacokinetics and Safety of Ceftobiprole in Neonate
 

Title: A Phase 1 Open-Label Study to Evaluate the Single-Dose Pharmacokinetics and Safety of Ceftobiprole in Neonate and Infant Subjects Aged Up to 3 Months Undergoing Treatment With Systemic Antibiotics
Principal Investigator: Chi Dang Hornik, PharmD
Research Coordinator: Grace Jefferson, MS
Funding Support: Basilea Pharmaceutica International Ltd.

About this trial
Bacterial infection is a leading cause of morbidity and mortality in children. Initial therapy in infants with severe infections is generally based on how the child presents because pathogen identification often requires impractical invasive sampling. Accordingly, it is important to use antimicrobials that provide broad-spectrum antibacterial activity. Ceftobiprole is a beta-lactam antibiotic with bactericidal activity against a broad spectrum of bacteria. In adults, ceftobiprole is approved in Europe for treatment of hospital-acquired pneumonia and for community-acquired pneumonia. In the pediatric population, the safety and efficacy of ceftobiprole have not yet been established.

The purpose of this study is to evaluate the safety and tolerability of ceftobiprole and to characterize the pharmacokinetics of a single dose infusion in neonates and infants born with a gestational age ≥ 28 weeks and aged ≤ 3 months. The hope for this study is that researchers can learn more about ceftobiprole and its pharmacokinetic functions at the neonate level to aid in the treatment of bacterial infections.

Study to Efficacy of IBP-9414 in Preterm Infants in Prevention of Necrotizing Enterocolitis

Title: A randomized, double blind, parallel-group, placebo controlled study to evaluate the efficacy and safety of IBP-9414 in premature infants ≤1500g birth weight in the prevention of necrotizing enterocolitis – The Connection study
Principal Investigator: Patricia Ashley, MD
Research Coordinator: Grace Jefferson, MS
Funding Source: Infant Bacterial Therapeutics

About this trial
Necrotizing enterocolitis (NEC) is an inflammatory condition that damages portions of the intestines. In infants, this condition can be severe and is better if treated early on per standard of care. There are no warning signals prior to the onset of NEC and no way to predict whether an infant will get NEC. There is no established preventive treatment of NEC and prevention strategies are urgently needed. A previous study done at Duke, by this same sponsor and research team, compared two different doses of IBP-9414 (one low dose and one higher dose) to a placebo. This study aims to use the higher dose compared to placebo for infants enrolled.

The purpose of this study is to evaluate the safety and efficacy of an investigational drug, IBP-9414, for preventing NEC in infants weighing less than 1500 grams at birth. The knowledge gained from the study may benefit care for premature babies in the future.

Monoclonal Antibody for Respiratory Syncytial Virus (RSV) in High-Risk Children
 

Title: A Phase 2/3 Randomized, Double-blind, Palivizumab-controlled Study to Evaluate the Safety of MEDI8897, a Monoclonal Antibody With an Extended Half-life Against Respiratory Syncytial Virus, in High-risk Children
Principal Investigator: William Malcolm, MD
Research Coordinator: Anne Baez, BS
Funding Source: Medimmue

About this trial
Respiratory Syncytial Virus (RSV) is a virus that is present in communities from late fall to early spring. Infection with RSV is common in all children. In the first year of life, almost half of all infants are infected with RSV. There are currently no medications to prevent RSV in healthy infants (born greater than 35 weeks gestational age), and there are no medications to treat an active RSV infection.  Synagis® is the only FDA approved medicine for the prevention of serious illness caused by RSV but it has limited use.

This study is evaluating the drug MEDI8897 to determine if it is effective in preventing lower respiratory tract infection due to RSV. Synagis® requires monthly injections for 5 months while MEDI8897 is active for a longer period of time and it is expected that one shot would provide protection to infants and young children for the entire RSV season.

Exclusive Human Milk Diet for Infants with Congenital Gastrointestinal Disorders
 

Title: Effects of an Exclusive Human Milk Diet on Enteral Feeding Outcomes of Neonates with Congenital Gastrointestinal Disorders
Principal Investigator: Jennifer Peterson, MD
Research Coordinator: Anne Baez, BS
Funding Source: Prolacta Bioscience, Inc. 

About this trial
Human milk (HM) is the ideal source of nutrition for all infants. HM feeding has been associated with a reduced incidence of gastroenteritis, otitis media, respiratory illnesses, allergic and autoimmune diseases and is recommended as the exclusive diet for infants less than six months of age. In premature infants, a HM diet has been associated with a decreased incidence of necrotizing enterocolitis, late-onset sepsis, increased intestinal motility and gastric emptying, improved feeding tolerance and general anti-inflammatory effects.

The purpose of this study is to determine whether or not an all exclusive human milk diet (from mother’s breast milk or from human donor milk), as compared to diets including formula, often based on cow’s milk, will be of benefit to infants who were born with a congenital gastrointestinal disorder (CGD) while they are in the NICU. We hope to see if an exclusive human milk diet will shorten the amount of time that it takes infants to reach full feedings, shorten the time infants require intravenous nutrition, shorten infants’ stay in the hospital and improve blood work values as compared to similar infants who received formula as part of their diet.

Early Check Provides Voluntary Screening of Newborns for a Selected Panel of Conditions
 

Title: Early Check: Expanded Screening in Newborns
Principal Investigator: C. Michael Cotten, MD
Funding Source: National Institutes of Health and John Merck Foundation 

About this trial
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health conditions in addition to providing important data on the implementation of this model program. Early diagnosis may result in health and development benefits for the newborns.

The goal of Early Check is to test babies for rare health problems and look for better treatments. To do this, Early Check will provide extra tests for babies beyond those that are part of regular newborn screening. Early Check uses the same blood sample from the baby’s heel already taken in the hospital for regular newborn screening. Newborn screening is done by the North Carolina State Laboratory of Public Health (NCSLPH) for all babies born in the state. The NCSLPH is working with the University of North Carolina at Chapel Hill, Wake Forest School of Medicine, Duke University, and RTI International to make the Early Check study possible.

Neonatal Seizure Registry – Developmental Functional Evaluation (NSR-DEV)

Title: Neonatal Seizure Registry – Developmental functional Evaluation (NSR-DEV)
Principal Investigator: Monica Lemmon, MD
Research Coordinator: Grace Jefferson, MS
Funding source: National Institute of Health/National Institute of Neurological Disorders and Stroke 

About this trial

Neonatal seizures due to brain injury (acute symptomatic seizures) are associated with high risk of neurodevelopmental disability in infancy. Although prognosis in early childhood is a critical question for parents and providers, outcomes beyond infancy are largely unknown. Further, parents of infants with neonatal seizures are at risk for mental health disorders, which can undermine their ability to care for a child with medical complexity and may contribute to impaired child development. This study builds upon a previous study done at Duke (Continued Anticonvulsants after Resolution of Neonatal Seizures) that studied how the treatment of neonatal seizures affected the infants’ developmental outcomes, their chances of developing epilepsy and the impact on the family.

The purpose of this multi-center study is to determine developmental outcomes after neonatal seizures. The knowledge gained from this study will provide novel, clinically relevant answers to questions about long-term outcomes in this highly vulnerable patient population, along with a deeper insight of how parent well-being can alter the risk for disability among children with prior seizures

Impact of anti-viral treatment on congenital CMV clinical outcomes

Title: Impact of anti-viral treatment on congenital CMV clinical outcomes, antibody response, and viral evolution
Principal Investigator: Kristin Weimer, MD, PhD
Research Coordinator: Grace Jefferson, MS
Funding source: Duke University

About this trial
Congenital cytomegalovirus (CMV) infection causes long-term neurologic deficits, most commonly sensorineural hearing loss, in up to 5,000 births each year in the United States.  Primary infection of the mother during pregnancy leads to significantly more frequent and severe cases of congenital CMV than non-primary or reactivated maternal infection. Unfortunately, a vaccine to prevent congenital CMV infection has yet to be developed.  However, anti-viral treatment with valganciclovir decreases the incidence of hearing loss and improves neurodevelopmental outcomes in neonates with congenital CMV. A major barrier to treatment is identification and diagnosis of neonates with congenital CMV infection.

Maternal and Neonatal Group B Streptococcus Seroepidemiology

Title: Maternal and Neonatal Group B Streptococcus Seroepidemiology
Principal Investigator: Rachel Greenberg, MD
Research Coordinator: Melissa Babilonia-Rosa, PhD
Funding Source: Centers for Disease Control and Prevention (CDC)

About this trial
Group B Streptococcus (GBS) remains a leading cause of serious infection among term newborns, preterm newborns, and peripartum women. The purpose of this study is to characterize of the relationship between serotype-specific GBS colonization and maternal antibody levels. As part of this multicenter study, Duke will collect discarded maternal sera from leftover RPR testing. Pregnant females (aged 16-50) admitted to the Labor and Delivery Units at Duke University Hospital will be screened for this study. De-identified sera will be shipped to the CDC for analysis.

The purpose of this project is to describe clinical characteristics at birth, determine adherence rates to six months of anti-viral therapy, quantify rates of hearing loss and neurodevelopmental outcomes, determine antibody responses, and determine the viral evolution of CMV in infants from birth until at least 24 months of age.  The knowledge gained from the study will be used in the development of future screening strategies and vaccine development.

Improved CMV Dried Blood Spot PCR for universal CMV screening

Title: Improved CMV Dried Bloods Spot PCR for universal CMV screening through Early Check
Principal Investigator: Kristin Weimer, MD, PhD
Research Coordinator: Melissa Babilonia-Rosa, PhD
Funding Source: Merck

About this trial
Congenital cytomegalovirus (CMV) is the leading cause of neurodevelopmental impairment and non-genetic sensorineural hearing loss (SNHL) in the developed world. Annually, within the United States, 20,000 to 40,000 infants are born infected, 100-200 infants die and 4,000 to 8,000 infants have permanent neurologic complications as a result of congenital CMV infection. Only 10-15% of infants with congenital CMV are symptomatic at birth, with symptoms ranging from mild to severe multi-organ dysfunction. Up to 20% of the asymptomatic infants will have neurodevelopmental impairment by 2 years of age.

The goal of this project is to develop a dried blood spot (DBS) assay for newborn screening of congenital CMV to start pharmacological treatment as soon as possible. The findings of this study will allow us to add this DBS assay for congenital CMV to the Early Check panel. Early Check is a statewide research program investigating the clinical burden of disease, performance of laboratory assays in a newborn screening system, and the benefits of early treatment and surveillance of multiple congenital diseases. Scavenged cord blood from term infants and discarded blood from infants (< 1 yr old) will be used to develop the DBS assay.


How you can participate

If you currently have a baby in the Duke Intensive Care Nursery, and you think you may want to participate in any of our research studies, please ask your baby’s nurse or doctor to contact us. The NPRU team will then contact you to explain the criteria for study eligibility and describe the potential benefits and risks for entering the study. Learn about our current research opportunities.

Learn more

To learn more about the Duke Intensive Care Nursery, visit the Duke Division of Neonatology website. For more information about the Neonatal Perinatal Research Unit or to learn more about our clinical trials, email us at npru@duke.edu, or call 919-681-4913.

 

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