Clinical Trials | Duke Department of Pediatrics

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.

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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

Eunice Kennedy Shriver NICHD Neonatal Research Network

Babies, Inc with the National Institutes of Health

Illicit Drug Exposure in Newborns

Additional Trials

Eunice Kennedy Shriver NICHD Neonatal Research Network

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

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, JD
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, JD
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, JD
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.

Budesonide in Babies (BiB)

Title: Rantomized Controlled Trial of Budesonide + Surfactant versus Surfactant Alone in Extremely Preterm Infants
Principal Investigator: Samia Aleem, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
NICUs, including the Duke NICU, commonly use a drug called surfactant to treat the underdeveloped lungs of a premature baby. The surfactant coats the inside of the lungs and helps babies to breathe easier. The purpose of this study is to find out if using a steroid called budesonide mixed together with surfactant will help reduce chronic lung disease called bronchopulmonary dysplasia (BPD) better than surfactant alone. Budesonide is already used in the NICU population, but this study will test whether the two drugs used together have a positive combined effect. Infants born between 401 and 1000 grams and between 22 and 29 weeks of age at birth may be eligible for this study. Infants participating in the study will return to the Special Infant Care Clinic at Duke for developmental testing at 2 years of age.

TOP5 Early School Age Follow Up

Title: Transfusion of Prematures Early School Age Follow Up (TOP 5)
Principal Investigator: William Malcolm, MD
Research Coordinator: Joanne Finkle, RN, JD
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this trial
Infants who were previously enrolled in the Transfusion of Prematures (TOP) Trial are now eligible to participate in a follow up study at 5-6 years of age. Infants in the original trial were assigned to either high or low transfusion levels to treat anemia (low red blood cell count). This study will be looking at how maintaining different hemoglobin levels (a protein in red blood cells that carries oxygen) in the first few weeks of life may impact neurological outcomes at school age. The study includes developmental testing, parent questionnaires and a physical exam. Parents can use this information to determine where their child might need extra help in school.

Baebies, Inc with the National Institutes of Health

Illicit Drug Exposure in Newborns

Title: A Novel Workflow to Screen for Illicit Drug Exposure in Newborns
Principal Investigator: William Malcolm, MD
Research Coordinator: Anne Love, BS
Funding Source: Baebies, Inc

About this trial
Neonatal abstinence syndrome (NAS) refers to a spectrum of withdrawal symptoms in newborns who were exposed to illicit or addictive substances in utero. Babies with NAS have higher rates of fetal anomalies (congenital malformations, growth restriction, ischemic placental complications) and perinatal issues (preterm delivery, poor feeding, sleep difficulties, diarrhea, seizures), which together raise the risk for adverse long term outcomes. Early identification of NAS is essential for referral of affected babies for interventions, including pharmacological treatments and behavioral/social support for the family.

To combat these challenges, a novel workflow has been proposed that will enable rapid toxicology screening of urine or meconium samples in the hospital. The potential benefits from implementation of this study include reduced length of hospitalization for unaffected newborns, accelerated time to confirmatory results, faster resolution of acute withdrawal symptoms, and improved referral to family/maternal support services. Results of the panel can be integrated into existing NAS treatment plans to facilitate rapid decisions regarding supportive or pharmacological therapies for NAS.

Additional Trials

Analyzing Retinal Microanatomy in Retinopathy of Prematurity to Improve Care

Title: Analyzing Retinal Microanatomy in Retinopathy of Prematurity to Improve Care - Follow On Study (BabySTEPS2)
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 take pictures of the back of the eye (retina) in preterm babies at risk for Retinopathy of Prematurity (ROP), using a special type of research camera called the investigational Ultracompact 3 or 4 Optical Coherence Tomography (OCT) system (UC3 or UC4). This camera is an imager friendly version of our non-contact OCT camera and we would like to use it as an alternative tool for ROP documentation, screening and future telemedicine. Retinal images taken with the UC3 or UC4 OCT will also be compared with color photographs taken with the standard of care FDA approved RETCam. During this study, participants will have eye imaging with the UC3 or UC4 OCT and the RETCam. These pictures will be taken while the infant is in the Neonatal Intensive Care Nursery at Duke.

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: Samia Aleem, MD
Research Coordinator: Caitlin Stone, MA
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.

Registry for Vermont Oxford Network

Title: Vermont Oxford Network Database
Principal Investigator: Michael Cotten, MD
Research Coordinator: Marita Passero, 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: Melissa Babilonia-Rosa, PhD
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).

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: Marita Passero, BS
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: Melissa Babilonia-Rosa, PhD
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.

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: Melissa Babilonia-Rosa, PhD
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 (a probiotic), 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.

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: Melissa Babilonia-Rosa, PhD
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
Research Coordinator: Joanne Finkle, RN, JD
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: Caitlin Stone, MA
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.

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.

Universal Masking by Healthcare Workers and Impact on Infection in NICU

Title: Does Universal Masking by Healthcare Workers and Families Decrease Late-Onset Infections in the NICU? The “MASKING” Study
Principal Investigator: Kristin Weimer, MD
Research Coordinator: Marita Passero, BS
Funding Source: Gerber Foundation

About this trial
Due to COVID-19, there has been an increased use of personal protective equipment (PPE) in the NICU including masking for all health care workers (HCWs) and families and in some cases increased hand hygiene, gloving, and wearing goggles. Masking could help decrease these and other infections as potentially it helps prevent the spread of mucosal bacterial and viral pathogens. Masking may work via decreasing both airborne transmission as well as direct contact transmission from the nose/face to hand to patient transmission. HCW and visitor nasal pathogens can be transmitted to a patient via an airborne transmission well as from hand contact of the face directly to the patient or to a surface around the patient. Masking has already being shown to decrease HCW acquisition of COVID-19, so preventing nasal colonization of viral pathogens is another mechanism of preventing infection.

The purpose of this study is to identify whether masking by healthcare workers and family members in the NICU has decreased the incidence of late onset sepsis and viral infections.

VON-Rady Genomic Network Biorepository

Title: VON-Rady Genomic Network Biorepository
Principal Investigator: Jennifer Cohen, MD
Research Coordinator: Isabella Pallottto, MPH
Funding Source: Duke University

About this trial
In October 2018, Rady Children's Institute for Genomic Medicine (RCIGM) collaborated with the Vermont Oxford Network to establish the VON-Rady Children’s Genomic Network. The VON-Rady Children’s Genomic Network includes NICU teams from 173 hospitals around the world learning together from expert faculty about the application of genomics to the care of newborn infants and their families.

There are estimated to be ~25,000 childhood single locus diseases and to be several thousand which remain to have the causal locus discovered. The purpose of the VON-Rady Genomics Network Biorepository is to provide access to rWGS to families who may benefit and to generate consented samples and data for future hypothesis-driven clinical research studies related to the cause and treatment of neonatal genetic disorders. Blood samples from babies meeting inclusion criteria and blood or saliva samples from parents will be collected for rWGS.

Active Surveillance of Respiratory Syncytial Virus (RSV) in Healthy US Infants

Title: Active Surveillance of Respiratory Syncytial Virus (RSV) in Healthy US Infants: A Pilot Study to Determine Feasibility of Establishing a Network
Principal Investigator: William Malcolm, MD
Research Coordinator: Marita Passero, BS
Funding Source: Sanofi

About this trial
RSV testing has been limited in the U.S. by the lack of support from the American Academy of Pediatrics and is not always reimbursed by payers. Therefore, an active, ongoing, clinical, and viral surveillance in the ED, inpatient, and outpatient settings is essential. Before a comprehensive surveillance system can be built and implemented, pilot studies at various medical facilities will be conducted to define the surveillance activities and identify any issues with patient recruitment/response rate or study procedures and assess solutions.

This pilot study of active RSV surveillance will determine the burden of medically attended, laboratory confirmed, RSV associated lower respiratory tract infection (LRTI-RSV) among children under one year of age who present to either an outpatient, ED, or inpatient setting with LRTI symptoms. This pilot study will involve both a clinical component along with a retrospective surveillance of the Duke University Healthcare System. It will focus on the identification of eligible children (<1 year of age) who present to the ED with symptoms of a possible RSV infection during this current RSV season (2020/2021).

Use of Lacosamide in Neonates with Seizures

Title: A Multicenter, Open-Label, Randomized, Active Comparator Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Lacosamide in Neonates with Repeated Electroencephalographic Neonatal Seizures.
Principal Investigator: Samia Aleem, MD
Research Coordinator: Marita Passero, BS
Funding Source: UCB Biopharma

About this trial
Neonatal seizures are abnormally excessive neurological activity in the brain that occur in infants. Current treatments for neonatal seizures don’t always work and have unwanted side effects; no treatments for seizures in babies are currently officially approved in the US by the Food and Drug Association (FDA). Researchers are looking into the investigational drug “lacosamide.” Lacosamide has been approved for treating seizures in adults and children down to 4 years of age many years and has been used to treat tens of thousands of children in the US and elsewhere. Recent studies in children down to the age of 1 month have showed no safety concerns.

The purpose of this study is to see if lacosamide helps reduce or stop neonatal seizures when other treatments have not. We are also looking to find the safest and best dose of lacosamide for babies. The population will be infants who are at least 34 weeks gestational age and are experiencing seizures still after first line treatment.

Implementation of an Evidence-Based Parentally Administered Intervention for Preterm Infants

Title: Implementation of an Evidence Based Parentally Administered Intervention for Preterm Infants
Principal Investigator: Debra Brandon, PhD, RN
Research Coordinator: Angel Barnes, RN, BSN
Funding Source: National Institutes of Health (NIH)

About this trial
Early developmentally-based behavioral intervention has well-established positive effects and is recommended as the standard of care to support early brain maturation, health, and development. However, few neonatal intensive care units (NICUs) provide this early intervention. H-HOPE (Hospital to Home: Optimizing the Preterm Infant’s Environment) has established efficacy, and has a standardized protocol, making it ready for widespread implementation. The infant-directed component of H-HOPE, provides Auditory (voice), Tactile (moderate touch massage), Visual (eye to eye), and Vestibular (rocking) stimulation starting when infants are ready for social interaction. The parent-directed component of H-HOPE includes participatory guidance and support to help parents engage with their infants in the NICU and the transition to home. During a prior pilot study, H-HOPE improved growth, developmental maturity and mother-infant interaction, and reduced initial hospitalization costs and acute care visits through 6-weeks corrected age.

Currently, we are testing if H-HOPE can be implemented and sustained in four diverse NICUs. We hope that widespread H-HOPE implementation will make a significant change in clinical practice and improve preterm infant health and health care costs.

Safety and Tolerability of AT-100 (rhSP-D) in Preterm Neonates at High Risk for BPD

Title: Safety and Tolerability of AT-100 (rhSP-D) in Preterm Neonates at High Risk for BPD
Principal Investigator: Samia Aleem, MD
Research Coordinator: Melissa Babilonia-Rosa, PhD
Funding Source: Airway Therapeutics, Inc.

About this trial
Babies born prematurely often need breathing support. Airway Therapeutics Inc. has developed a medication (AT-100) that is given through the breathing tube to help reduce inflammation in the lungs. The purpose of this research study is to determine if the study drug (AT-100) is safe and tolerated, when compared to a push of air (“air-sham”) alone, in children who were born premature. The research is also studying whether or not AT-100 reduces the amount of time babies spend on breathing support provided by a machine called a mechanical ventilator and reduces the occurrence of Bronchopulmonary Dysplasia (BPD), a condition sometimes developed by babies that require breathing support.

AT-100 is an investigational drug, and this is the first time it is being tested in humans. It is not yet approved by the U.S. Food and Drug Administration (FDA).

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.