Clinical Trials

Elsie
NICU Graduate

​Eunice Kennedy Shriver NICHD Neonatal Research Network

• Generic Database: Survey of Morbidity and Mortality Among High Risk Preterm Infants
• Generic Database Follow Up: Follow-Up of High Risk Infants
• Milrinone in Congenital Diaphragmatic Hernia (CDH)
• PDA Trial
• Cycled Phototherapy to Control Bilirubin
• Budesonide in Babies (BiB)
• Darbe-KIDS

​Additional Trials

• New Technology to Determine Visual and Neurological Development of Preterm Infants
• An Observational Study of Infants with Congenital Diaphragmatic Hernia (CDH)
• ​Registry for Vermont Oxford Network
• Extremely Preterm Infant Biorepository
• Efficacy of IBP-9414 in Preterm Infants in Prevention of Necrotizing Enterocolitis
• Neonatal Seizure Registry--Developmental Functional Evaluation (NSR-DEV)
• Growth and Tolerance of Preterm Infants Fed an Experimental Human Milk Fortifier
• Phase 2 Study Evaluating RLS-0071 Study Drug in Newborns with HIE
• Multisite Inventory of Neonatal-Perinatal Interventions for Infants Less than 24 Completed Weeks
• Model for Extubation Readiness Timing among Preterm Infants
• Vermont Oxford Network (VON) Quality Improvement Collaborative: All Care is Brain Care
• Instrument Validation for Blood Gas Analyzer
• Neonatal Seizure Registry, Genetics of post-Neonatal Epilepsy
• Impact of anti-viral treatment on congenital CMV clinical outcomes, antibody response, and viral evolution
• Decision Making Support for Parents and Caregivers
• Pharmacokinetics of Drugs Administered to Children per Standard of Care
• Phase 1-2 Open Label Study of Study Drug in Infants with NEC
• Instrument Validation using Capillary Whole Blood Specimens
• Phase 2/3 Study of AT-100 (rhSP-D) in Preterm Neonates at High Risk for BPD

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 Propst, 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 Propst, 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 Propst, 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
 

PDA Trial

Title: Management of the Patent Ductus Arteriosus in Premature Infants Trial (PDA Trial)
Principal Investigator: Jennifer Peterson, MD
Research Coordinator: Joanne Propst, 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 Propst, 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.

Learn more on ClinicalTrials.gov
 

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 Propst, 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.

Learn more on ClinicalTrials.gov

Darbe-KIDS

Title: The Darbepoetin Kindergarten Development Study
Principal Investigator: William Malcolm, MD
Research Coordinator: 
Funding Source: National Institute of Child Health & Human Development (NICHD)

About this study

Infants who were previously enrolled in the Darbe Trial can now participate in a follow-up study at 4-5 and 6-7 years old. Babies in the Darbe Trial were randomized to receive darbepoetin or placebo once a week until a month before their actual due date to find out if darbepoetin helped babies make more red blood cells and if darbepoetin could improve babies’ brain development. Darbe-Kids is a follow up study to know if there is a difference in development at 4-7 years old after receiving darbepoetin. Both study visits of Darbe-Kids include parent questionnaires and developmental testing; the 4-5 year of age visit also includes a physical exam. Parents can use this information to determine where their child might need extra help in school.

Learn more on ClinicalTrials.gov

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 Propst, 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: Jessicka Hamilton, BA
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: Jessicka Hamilton, BA
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.
 

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.

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

Title: Neonatal Seizure Registry – Developmental Functional Evaluation (NSR-DEV)
Principal Investigator: Monica Lemmon, MD
Research Coordinator: Jessicka Hamilton, BA
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.
 

Growth and Tolerance of Preterm Infants Fed an Experimental Human Milk Fortifier

Title: Growth and Tolerance of Preterm Infants Fed an Experimental Human Milk Fortifier
Principal Investigator: Noelle Younge, MD
Research Coordinator: Jessicka Hamilton, BA
Funding Source: Abbott Nutrition

About this trial
Scientific evidence indicates that human milk is the best form of nutrition for all infants, including preterm infants. Premature and extremely low birth weight infants who are fed maternal and/or donor milk sometimes have human milk fortifier (HMF) added to their feedings to help facilitate weight gain, while in the neonatal intensive care unit (NICU). The purpose of this research study is to evaluate how premature babies grow and tolerate an experimental HMF compared to the standard of care (SOC) HMF. This experimental HMF contains human milk oligosaccharides (HMOs are special prebiotics) and nucleotides (essential components of cellular energy) are naturally found in human breastmilk and may provide a benefit to immune and digestive health of premature infants.

Phase 2 Study Evaluating RLS-0071(Study Drug) in Newborns with Hypoxic-Ischemic Encephalopathy (HIE)

Title: Phase 2 Study Evaluating RLS-0071(Study Drug) in Newborns with Hypoxic-Ischemic Encephalopathy (HIE)
Principal Investigator: Samia Aleem, MD
Research Coordinator: Jessicka Hamilton, BA
Funding Source: ReAlta Life Sciences, Inc.

About this trial
Some babies are born with a condition called hypoxic-ischemic encephalopathy (HIE). HIE is a condition characterized by a decreased level of alertness, low muscle tone, and often seizures beginning soon after birth. HIE at birth can be a sign of significant injury to the brain that may lead to permanent brain damage. Standard of care for treatment of HIE is whole body cooling to help reduce brain injury. In addition to whole body cooling, babies in this study may receive an investigational medication called RLS-0071. RLS-0071 acts to decrease the damage done to brain tissue as the body attempts to recover from HIE. RLS-0071 decreases the inflammation (irritation) that the brain experiences after being deprived of blood flow and oxygen. An investigational medication means that it has not been approved by the United States (US) Food and Drug Administration (FDA) to be sold to the public. The FDA is the governmental agency in the United States responsible for approving new medications/devices.

Multisite Inventory of Neonatal-Perinatal Interventions for Infants Less than 24 Completed Weeks

Title: Multisite Inventory of Neonatal-Perinatal Interventions for Infants Less than 24 Completed Weeks
Principal Investigator: Noelle Younge, MD
Research Coordinator: Michelle Sunico, BA
Funding Source: Division Funding

About this trail
The goal of the Tiny Baby Collaborative Multicenter Inventory of Neonatal-Perinatal Interventions (MINI) minimum dataset is to serve as a registry detailing the outcomes and practices for all deliveries at infants admitted to intensive care at 22-23 completed weeks’ gestation at participating hospitals. The minimum dataset will serve to detail the epidemiology of important perinatal interventions and outcomes for this inadequately studied group and suggest hypotheses about how to further improve care of the maternal-infant dyad. Data collected in the registry will be used for real-time assessment of local perinatal care practices and may serve as the basis for local changes/quality improvement. Moreover, they will serve to identify “potentially better practices” that may be desirable for wider implementation.

Model for Extubation Readiness Timing among Preterm Infants

Title: Model for Extubation Readiness Timing among Preterm Infants
Principal Investigator: Rachael Greenberg, MD
Research Coordinator: Michelle Sunico, BA
Funding Source: Chiesi, Inc.

About this trial
Invasive mechanical ventilation (IMV) is critical for many extremely preterm infants to maintain adequate oxygenation and ventilation. Prolonged IMV is associated with both short-term morbidities and neurologic impairment. Ability of clinicians to accurately predict successful extubation is limited and 25%-40% of infants fail extubation. Extubation failure has been associated with significant morbidities, including increased duration of IMV and bronchopulmonary dysplasia (BPD).

In a recent single-center study, investigators from this group developed a prediction tool to calculate the probability of successful extubation for an individual infant (www.extubation.net). External validation at a separate center showed moderate performance with substantial differences in the impact of certain predictors specific to center practices. For this reason, we propose to further refine and validate this extubation readiness prediction tool for individual infants using readily available clinical variables in a well-powered multicenter study. Before this tool can be implemented globally it is imperative that it is validated in multiple centers to test for its diagnostic accuracy.

Vermont Oxford Network (VON) Quality Improvement Collaborative: All Care is Brain Care

Title: Vermont Oxford Network (VON) Quality Improvement Collaborative: All Care is Brain Care
Principal Investigator: Kamlesh Athavale, MD
Research Coordinator: Michelle Sunico, BA
Funding Source: US Division Funding

About this study
VON is offering a QI Collaborative opportunity for their participating sites. This QI collaborative will focus on protection of the brain for infants admitted to the NICU. The overall focus of this project will be directed toward safeguarding the full potential for each infant hospitalized in the NICU.

While we continue to work to improve survival rates we often do so at the expense of or without clear focus on our ability to safeguard the full potential of each infant. Much of what we do in the NICU as healthcare providers is out of necessity, not neuroprotective, and potentially harmful over time. Our goal is that each care interaction with a newborn is approached with a neuroprotective lens, balancing benefit and harm, supporting normal maturation and preventing injury. Every interaction with an infant and their family in the neonatal intensive care setting is an opportunity to be brain-focused and have a direct impact on the developing brain. Duke collaborators will develop quality improvement projects directed toward a focus on brain protection.

Instrument Validation for NICU Bedside Blood Gas Analyzer

Title: Instrument Validation for NICU Bedside Blood Gas Analyzer
Principal Investigator: C. Michael Cotten, MD
Co-Investigator: Melissa Babilonia-Rosa, PhD
Funding Source: Radiometer

About this study
To validate performance claims by method comparison for the ABL90 FLEX PLUS running an updated software version when measuring total bilirubin and fraction of fetal hemoglobin (FHbF) in heparinized neonatal arterial, venous, and capillary whole blood in a POC setting.

Information on blood gas status in the critically ill newborn is necessary to provide good clinical care. The ABL90 FLEX PLUS analyzer is a portable, automated analyzer that measures pH, blood gases, electrolytes, metabolites, and oximetry in whole blood – utilizing 3 measuring modes to measure samples from a syringe, test tube or a capillary tube.  Specially designed for demanding hospital units like the ICU, NICU and ED, the ABL90 FLEX PLUS analyzer has been CLIA licensed and is widely used in these clinical settings.  Duke Pediatrics clinical laboratories has 3 ABL90 FLEX PLUS analyzers currently in use. The NPRU team will provide validation of the device using scavenged blood samples.

Neonatal Seizure Registry, Genetics of post-Neonatal Epilepsy

Title: Neonatal Seizure Registry, Genetics of post-Neonatal Epilepsy
Principal Investigator: Monica Lemmon, MD
Research Coordinator: Jessicka Hamilton, BA
Funding Source: National Institutes of Health

About this study
Neonatal seizures due to brain injury (acute symptomatic seizures) are associated with high risk of postneonatal epilepsy. Although clinical risk factors can help predict which children are at highest risk for epilepsy, little is known about how genetic factors modify the risk for epilepsy after acute symptomatic neonatal seizures. This study will test the central hypothesis that children who develop post-neonatal epilepsy are more likely to have pathogenic variants in epilepsy genes, and enrichment in single nucleotide polymorphisms within key inflammatory, neurotransmitter transport and homeostasis, and neurotrophic gene pathways as compared with children who do not develop unprovoked seizures before age five years, and that these can be added to traditional clinical risk factors to predict epilepsy after neonatal seizures.

This observational study leverages the infrastructure of the Neonatal Seizure Registry. This unique cohort of children, as well as eligible children enrolled from outpatient clinics and their parents will be invited to participate in non-invasive genetic testing. Using neonatal clinical, EEG, and MRI measures available through the Registry, along with genetic testing results, we will build robust models to predict risk of epilepsy and elucidate mechanisms of epileptogenesis.

Learn more on ClinicalTrials.gov

Impact of Anti-viral Treatment on Congenital CMV Clinical Outcomes, Antibody Response, and Viral Evolution

Title: Impact of Anti-viral Treatment on Congenital CMV Clinical Outcomes, Antibody Response, and Viral Evolution
Principal Investigator: Kristin Weimer, MD
Research Coordinator: Michelle Sunico, BA
Funding Source: Divisional funding

About this study
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 Congenital cytomegalovirus (CMV) in infants from birth until at least 24 months of age.  These data will be used in the development of future screening strategies and vaccine development. Infants with symptoms concerning for congenital CMV infection or findings that qualify for targeted screening are assessed with a saliva PCR per newborn nursery and intensive care nursery protocols. 

Decision Making Support for Parents and Caregivers

Title: Decision Making Support for Parents and Caregivers
Principal Investigator: Monica Lemmon, MD
Research Coordinator: Jessicka Hamilton, BA
Funding Source: National Institutes of Health

About this study
The impact of decision making interventions in pediatric neurology populations is unknown. Existing decision aids have been developed primarily for adult populations and typically target discrete clinical decisions; for example, whether to undergo a surgical procedure or obtain cancer screening. The decisions facing parents of critically ill infants and children—whether to obtain a tracheostomy or remove the ventilator—are complex, and often made and remade over time. For many parents, an early decision to proceed with life-sustaining treatments is followed by recurrent hospitalizations and a lifetime of medical complexity. For these parents, the neonatal and pediatric periods represent an introduction to collaborative decision making and have the potential to frame how they make and participate in decisions for their child over time. A decision aid for this population was developed based on the outcomes of a previous study funded by the National Institutes of Health.

This feasibility study will use a single-arm design. All infants admitted to an intensive care unit will be screened for enrollment. We will enroll up to 40 infants, their parent(s), and their physician(s). We will deliver the decision guide in advance of the goals-of-care conference. A study team member will deliver the tool (decision aid), discuss its use with parents, and be available for questions as they arise. We will survey parents at baseline. Parents and clinicians will complete surveys and interviews following the goals-of-care conference. 

Learn more on ClinicalTrials

Pharmacokinetics of Drugs Administered to Children per Standard of Care

Title: Pharmacokinetics of Drugs Administered to Children per Standard of Care
Principal Investigator: Rachel Greenberg, MD
Research Coordinator: Jessicka Hamilton, BA
Funding Source: Divisional Funding

About this study
The purpose of this study is to characterize the PK of understudied drugs administered to children per standard of care as prescribed by their treating caregiver. This will be accomplished by the collection of biological samples during the time of drug administration per standard of care as prescribed by the caregiver. The prescribing of drugs to children will not be part of this protocol. This protocol will serve to evaluate the PK of understudied drugs in children.

Only 25% of approved drugs marketed in the United States have sufficient pediatric data to support approval of product labeling by the US Food and Drug Administration (FDA) for dosing, safety, or efficacy in children. Similarly, in the European Union only 33% of drugs are authorized for use in children by the European Medicines Agency (EMA). Existing gaps in pediatric drug dosing, safety and efficacy places children at risk for adverse events and potential therapeutic failures. 

Several limitations inherent to trials involving children prevent researchers from pursuing the study of medications in this population. These include low rates of informed consent among caregivers; limited blood volume necessary to conduct drug profiling studies; lack of pediatric clinical pharmacology expertise to design, conduct, and analyze data derived from clinical trials; lack of validated and sensitive drug and biomarker assays; difficulties associated with rigid biological sample timing collection used in traditional drug trials; lack of robust infrastructure to support pediatric clinical trials; and the lack of application of opportunistic methodologies to study drugs in children.

Phase 1-2 Open Label Study of Study Drug in Infants with NEC

Title: A Randomized, Controlled, Phase 1-2 Open Label Study of ST266 IV Administration to Assess the Safety, Tolerability, and Preliminary Efficacy of ST266 in Infants with Necrotizing Enterocolitis
Principal Investigator: Trevor Burt, MD
Research Coordinator: Jessicka Hamilton
Funding Source: Noveome Biotherapeutics, Inc.

About this study

This is a Phase 1-2, multi-site clinical trial. The population are infants from birth to 2 months (gestational age of 26 weeks to 36 weeks) with confirmed diagnosis of Necrotizing Enterocolitis (NEC). NEC is an inflammatory condition that damages portions of the intestines.  The purpose of this study is to determine the safety and tolerability of intravenous administration of ST266 (study drug) for the treatment of preterm infants with NEC. In this study, infants will be randomly assigned (by chance, like the flip of a coin) to standard of care alone or standard of care plus the study drug ST266.

Instrument Validation using Capillary Whole Blood Specimens

Title: Clinical Evaluation of the i-STAT Crea, CG4+ and CG8+ Cartridges using Neonatal Capillary Whole Blood Specimens with the i-STAT 1 Analyzer
Principal Investigator: C. Michael Cotton, MD
Research Coordinator: Michelle Sunico, BS
Funding Source: Abbott

About this study

This is a method comparison study of the i-STAT 1 system using a comparator device. The i-STAT is a handheld device that is used at the bedside in the NICU to perform quantitative determination of specific analytes in blood (i.e. blood gas). Specimens for use in this study will be prospectively collected with informed consent. Each test will be performed in duplicate on two i-STAT cartridges. The study will require no more than 250 microliters of capillary blood collected from a heel-stick.

This study poses no greater than minimal risk as all heel sticks will accompany a standard of care blood draw.

Phase 3 Study of AT-100 (rhSP-D) in Preterm Neonates at High Risk for BPD

Title: Phase 2/3 Study 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 study

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 the efficacy of the study drug (AT-100), in preterm infants when compared to a push of air (“air-sham”) alone. 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. 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). 

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. 

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.