Our commitment to advance the scientific investigation of medical therapies in children begins at the bedside. We know firsthand the limits and risks of providing treatments based on evidence generated through studies of adults.

Through innovative design methods and data monitoring, the DCRI's Pediatrics team is leading the world in expanding the impact of pediatric research.

Protecting Our Most Vulnerable Population

Doctor and child

Our Pediatrics faculty ensure that the safety and well-being of children is at the center of every step of pediatric clinical research. Along with our statisticians, site and data management experts, and project leaders, our faculty work closely with sponsors to ensure accurate and efficient study delivery.

Unparalleled Pediatric Expertise

Our pediatric research experience ranges from pharmacokinetic studies in neonates to large multicenter trials in adolescent populations. We focus on:

  • Allergy
  • Cardiology
  • Critical Care
  • Genetics
  • Infectious disease
  • Neonatology
  • Obesity
  • Primary care
  • Pulmonology
  • Rheumatology


Pediatric Trials Network logo

The Pediatric Trials Network (PTN) is a research network born from a groundbreaking collaboration between the DCRI and several of the country’s preeminent pediatric-medicine experts, hospitals, and medical centers. Many of the DCRI's pediatric clinical trials are conducted through the PTN, which was founded in 2010 with a $95 million contract from the National Institutes of Health (NIH) and renewed in 2018. Through the PTN, our Pediatrics faculty and their colleagues across the nation study the formulation, dosing, efficacy, and safety of drugs—as well as the development of medical devices—used in pediatric patients.

Every PTN study is closely examined to determine how to obtain the most valuable and generalizable results while minimizing the risk to participants. The PTN has enrolled more than 12,000 patients in studies and trials that focus on 20 different therapeutic areas. Data collected from PTN trials has already helped regulators to revise 18 drug labels for safer and more effective use in infants and children. PTN has submitted pediatric data to the FDA for 26 products.

Read the articles below to learn how:

Baby in NICU


DCRI's Pediatrics team contributed to the FDA's draft guidance on research in newborns, which provided clarity on study design, data analysis, and drug dosing. Kanecia Zimmerman, MD, MPH, and Michael Cohen-Wolkowiez, MD, PhD, recently wrote about their work and the importance of the new FDA guidance in STAT News.




DCRI investigators, in collaboration with Duke's Department of Population Health Sciences, are developing a set of endpoints and measures that will be used in clinical trials and spur development of new pain medications for young children.


Laura Schanberg


With over 10,000 patients enrolled across 71 sites in three countries, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry studies disorders ranging from pediatric lupus to juvenile arthritis.


DCRI Releases White Paper Providing Guidance on Pediatric Research

Research publication cover

DCRI pediatric researchers authored a white paper outlining recommendations and insights into planning and designing patient-centered pediatric clinical research. The paper is the culmination of extensive research and direct work with pharmaceutical companies to identify and address challenges specific to pediatric research.


Leveraging Real-World Data to Deliver Insights into Pediatric Migraine

The real-world data collected from pediatric patients diagnosed with migraine via a registry co-led by Christoph Hornik, MD, PhD, MPH, could open the door for future drug development in the space. DCRI’s Research Together program contributed to the registry to ensure it collects data that is important to patients and their families.


Pediatrics Leadership at the DCRI

Brian Smith, MD, MPH, MHS — Therapeutic Lead

Brian Smith

Dr. P. Brian Smith is a neonatologist and the Samuel L. Katz Distinguished Professor of Pediatrics. He is the Therapeutic Area Lead for Pediatrics Research at the Duke Clinical Research Institute. His research is focused on pediatric drug safety, neonatal pharmacology, and the epidemiology of neonatal infections. Smith is the Principal Investigator for the NIH’s Environmental influences on Child Health Outcomes (ECHO) Coordinating Center and has been the protocol chair for more than 14 studies of drugs in infants and children.

Dr. Smith completed his residency in pediatrics and a fellowship in neonatal medicine at Duke University Medical Center in 2004 and 2007, respectively. He completed an MHS in clinical research from Duke University in 2006 and an MPH in biostatistics from the University of North Carolina at Chapel Hill in 2009.