Department of Pediatrics researchers, both MD and PhD scientists, run more than 60 research groups across diverse specialties within the 16 divisions. Of the more than $55 million in research awards the department received in 2023, over half the funding supported clinical research. “Clinical research” is research that involves people — it studies the safety and effectiveness of new drugs, devices, diagnostic tests, techniques, and processes in patient medical diagnosis and care. “Clinical trials,” specifically, test the safety and effectiveness of medical interventions, which can be medications, tools, procedures, and investigational medicinal products (IMPs).
Several of the department’s divisions that are engaged in numerous clinical trials manage their studies internally. For example, the Division of Hematology, Oncology, and Bone Marrow Transplant supports a robust clinical trials effort and also participates in multi-institutional group trials. The Division of Allergy, Immunology, and Rheumatology is the home of several very active research groups. Smaller groups and individual PIs in other divisions have access to research project help within the department.
Researchers can find assistance for every step in the clinical research process from the Pediatric Clinical Research Coordination (PCRC) Program. The PCRC is a fee-for-service team that provides comprehensive clinical research support to department PIs conducting research at the UW, UW Health, Meriter, and the Waisman Center. Bridget Johnson, BSN, RN, CCRC, is the clinical research manager of PCRC. The team includes research nurses and non-nurse clinical research coordinators with experience and training in all areas of clinical research. PCRC also works closely with the Department of Pediatrics administration and regulatory teams.
“We can help PIs navigate the nuances and complexities associated with conducting clinical research here,” Johnson said. “I am happy to consult with any PI to discuss our team’s capacity to support specific research, the steps to operationalize the research, and study-specific feasibility related to subject recruitment, consenting, and study procedures. Interested researchers can complete the Pediatric Research Support Request form to request PCRC support or reach out directly with any questions.” (Please note that the PCRC webpage is currently being extensively updated.)
Learn about some of the clinical trials in progress in the Department of Pediatrics
Dinushan Kaluarachchi, MBBS, is associate professor and director of neonatal clinical research in the Division of Neonatology and Newborn Nursery. He runs the Kaluarachchi Research Group.
Study title: “Pilot Study of Late Surfactant Therapy with Budesonide for Prevention of Bronchopulmonary Dysplasia in Extremely Preterm Infants,” or “Little Lungs Study,” for short.
The problem and goal: Bronchopulmonary dysplasia (BPD), or chronic lung disease of prematurity, has increased in the last decade, increasing neonates’ risk of mortality and of respiratory insufficiency and abnormal neurodevelopment. BPD treatment takes up resources and increases health care costs.
Treatments that may prevent BPD could improve the outlook of extremely preterm infants. Late surfactant treatment with budesonide is an innovative treatment strategy that has not been studied well. Dual therapy with surfactant to treat lung dysfunction and intratracheal steroids to treat lung inflammation may prevent the development of BPD. The pilot study aims to evaluate the feasibility and safety of late surfactant treatment with budesonide. The study may generate important data that could inform the design of future large randomized clinical trials.
Funding: The study has recently received funding from the Meriter Foundation.
Matthew Harer, MD, is associate professor in the Division of Neonatology and Newborn Nursery. He runs the Improving Kidney Outcomes in Neonates into Childhood: IKONIC Research Group.
Study title: “Investigating Kidney Tissue Oxygenation Monitoring in Preterm Neonates for the Early Detection of Acute Kidney Injury” (KNIRS study)
The problem and goal: This is an observational study to validate kidney oxygenation monitoring with near-infrared spectroscopy (NIRS) in preterm neonates born before 32 weeks’ gestation. The study has multiple goals: evaluate differences in right to left kidney oxygenation, identify the kidney location with point of care ultrasound to ensure appropriate measurements, identify changes in kidney oxygenation as preterm neonates mature, and correlate changes in kidney oxygenation with the development of acute kidney injury.
The early identification and diagnosis of acute kidney injury (AKI) through kidney tissue oxygenation monitoring at a time when injury is reversible will facilitate the development of clinical renal tissue oxygenation monitoring guidelines and treatment algorithms that can improve the short- and long-term kidney function of preterm neonates.
Funding: Jointly funded by a Wisconsin Partnership Program New Investigator Award and an ICTR KL2 Scholar Award.
Daniel O’Connell, MD, is associate professor and interim chief of the Division of Gastroenterology, Hepatology, and Nutrition.
Study title: “A Phase 3 Multicenter Study to Evaluate Efficacy, Safety, and Pharmacokinetics of Upadacitinib with Open-Label Induction, Randomized, Double-Blind Maintenance and Open-Label Long-Term Extension in Pediatric Subjects with Moderately to Severely Active Ulcerative Colitis and Inadequate Response, Intolerance, or Medical Contraindications to Corticosteroids, Immunosuppressants, and/or Biologic Therapy.”
The problem and goal: Upadacitinib is an oral once-a-day JAK inhibitor used to treat various inflammatory conditions. It has had FDA approval for adults with ulcerative colitis (UC) since 2022. Access to novel therapeutics in pediatric ulcerative colitis often lags adult medicine by an average of eight to 10 years after initial FDA approval. There is an urgent need to understand the dosing, tolerance, and safety for our pediatric patients with UC as metabolism and disease-specific factors often lead to modifications from the typical adult dosing regimens.
Funding: This study is an industry partnership with AbbVie.
O’Connell noted that he deeply appreciates “the tireless and amazing work of the department research leadership and infrastructure to help guide me through the process of starting an industry-sponsored clinical trial.”
Neil Paloian, MD, is associate professor in the Division of Nephrology.
Study title: “ORBIT: Study to Assess Dose, Efficacy, and Safety of Setrusumab in Participants with Osteogenesis Imperfecta”
The problem and goal: This is a randomized, placebo-controlled phase 3 study evaluating Setrusumab versus placebo in children with osteogenesis imperfecta (OI). It is a genetic bone disease, also called “brittle bone disease.” The main study focus is to compare fracture rates between the two groups; a secondary focus is to compare bone mineral density between the two groups. The hypothesis is that children treated with Setrusumab will have fewer fractures and higher bone mineral density than those treated with placebo.
Paloian noted, “Setrusumab is given as a once-monthly IV injection. These occur in the American Family Children’s Hospital (AFCH) day treatment unit. We are one of 47 sites around the world participating in this study.”
Funding: This study is funded by Ultragenyx pharmaceuticals, the makers of Setrusumab.
Pelin Cengiz, MD, is professor in the Division of Critical Care. She runs the Cengiz Research Group and offers overviews of two ongoing clinical trials here.
#1 Study title: “Systemic Hydrocortisone in Pediatric Severe Sepsis (SHIPSS)”
The problem and goal: Stress dose hydrocortisone has been used to address issues of adrenal insufficiency that can be a complication in pediatric septic shock. However, because hydrocortisone can influence many biological functions, including immune system responses, it is critical to weigh the potential benefits and risks, such as increased infections or other complications related to hydrocortisone’s immunosuppressive effects. The study is a National Institutes of Health (NIH)-funded clinical trial that evaluates those benefits and risks.
The SHIPSS trial is a crucial step toward enhancing treatment protocols for pediatric septic shock, aiming to provide evidence-based guidelines for the use of stress dose hydrocortisone in this vulnerable population. Read more at ClinicalTrials.gov.
Funding: NIH-R01
Cengiz explained, “The study is planning to enroll up to 1,000 children from multiple countries, including the United States, Canada, Brazil, various Asian and European countries, and more. The children are evaluated at the start of the trial, then again at 28 days and 90 days after enrollment.” Cengiz is the site PI for this study.
#2 Study title: “Healthy Little Eyes”
The problem and goal: Hypoxic-ischemic encephalopathy (HIE) is recognized as a leading cause of visual impairments among children in developed countries, affecting approximately 20,000 newborns nationwide annually. Visual impairments can have pervasive and far-reaching impact across multiple developmental domains including motor, social and communication, and cognitive development.
Overall, the goal of this project is to institute novel techniques at birth to characterize eye function in order to diagnose and predict the outcomes of babies with HIE.
This study employs the “RETeval” device, an FDA-approved, non-invasive tool designed to assess the retinal and visual functions of infants from birth up to late infancy. It aims to correlate these visual assessments with neurodevelopmental outcomes and neuroimaging results, building a robust foundation for potential therapeutic and rehabilitative innovations.
Cengiz is joined in this study by Bikash Pattnaik, PhD, associate professor, Division of Neonatology and Newborn Nursery, and Ryan McAdams, MD, professor and division chief, Division of Neonatology and Newborn Nursery, and Division of Global Pediatrics, as well as Maria Stanley, MD, professor and chief, Division of Developmental Pediatrics & Rehabilitative Medicine, and interim chief, Division of Genetics and Metabolism, and Teresa Chapman, MD, professor in the Department of Radiology.
Cengiz and Pattnaik are dedicated to laying the groundwork for a multi-PI National Institutes of Health R01 application in which they can test the therapeutic interventions and rehabilitative measures in this vulnerable population.
Funding: Currently funded with a Unity-Meriter Foundation Pilot Grant. The project was previously funded through the Waisman Center and ICTR Pilot Grants.
“The implications of this research extend beyond the immediate benefits to newborns with HIE,” Cengiz explained. “By improving diagnostic and prognostic capabilities, our understanding of central nervous system impairments will be enhanced more broadly, which can be applied to conditions such as traumatic brain injury and brain tumors.”
Ryan McAdams, MD, professor and chief, Division of Neonatology and Newborn Nursery, and Division of Global Pediatrics. He runs the McAdams Research Group.
Study title: “Association of Neurovascular Dysfunction with Fetal Acidosis and Mast Cell Activation (FAMC)”
The problem and goal: The clinical trial aims to investigate the role of mast cell activation in severe neonatal conditions like hypoxic ischemic encephalopathy. We hypothesize that fetal metabolic acidosis triggers mast cell activation, which may contribute to the severity of neonatal diseases.
Our research focuses on the correlation between umbilical cord blood mast cell tryptase levels and fetal acidosis to better understand these mechanisms. Additionally, we are exploring how interventions such as caffeine treatment can potentially reduce mast cell activation and improve outcomes for high-risk neonates. The ultimate goal is to develop more effective predictive tools and treatments to decrease infant morbidity and mortality.
Funding: Meriter Foundation
“Our study is particularly focused on understanding and addressing the health disparities that affect neonatal outcomes,” McAdams explained. “For example, our research considers the disproportionate rates of premature births and neonatal complications among Black and Indigenous People of Color. By assessing how fetal acidosis and mast cell activation vary across different demographic groups, we aim to develop tailored interventions that can significantly improve the health and survival rates of all neonates, particularly those from underrepresented and underserved populations.”