We are excited to announce that Rusty McCulloh, MD, and his team competed successfully for funding to continue the Nebraska Pediatric Clinical Trials Unit (NPCTU) for another five years! This $2.1 million grant, awarded by the National Institutes of Health Office of the Director, ensures that the NPCTU can continue to engage in important clinical trials and rural outreach as part of the IDeA States Pediatric Clinical Trials Network (ISPCTN). This network, with a footprint in 18 states, is the interventional component of the NIH Environmental Influences on Child Health Outcomes (ECHO) Program. Its mission is to improve research infrastructure in underfunded states, as well as to include rural and underserved pediatric populations in multisite clinical trials.
During the first grant period (2016-2020), the NPCTU was highly involved in ISPCTN activities, contributing to four clinical trials on a range of topics in child health, including obesity, asthma, neonatal opioid withdrawal syndrome, and characterization of understudied drugs in children. So far, projects supported by the NPCTU during Cycle 1 have resulted in 9 peer-reviewed publications.
The NPCTU partnered in all four of the network’s clinical studies during Cycle 1:
- Dr. McCulloh led the Nebraska team for the Pharmacokinetics of Understudied Drugs Administered to Children per Standard of Care (POP01) study. Investigators collected pharmacokinetic samples from children receiving off-label medications and evaluated biomarkers in order to establish better pediatric dosing guidelines and obtain FDA approval when possible.
- The Advancing Clinical Trials in Neonatal Opioid Withdrawal Syndrome (ACT NOWS) Current Experience study, under the supervision of Ann Anderson Berry, MD, PhD, collected information on the current models of care for infants with NOWS across the ISPCTN and the NIH-funded Neonatal Research Network. The team is in the process of publishing the results of this study and have since launched two subsequent clinical trials involving a novel opioid weaning protocol and the “Eat, Sleep, Console” approach to managing NOWS. Both trials will continue in Cycle 2.
- Similarly, the Vitamin D Oral Replacement in Asthma (VDORA1) study was developed during Cycle 1 but will continue into the second grant period. Led locally by Hana Niebur, MD, assistant professor of Pediatric Allergy and Immunology, this study seeks to establish vitamin D dosing information for overweight children with asthma. In the second funding cycle, the ISPCTN plans to extend the scope of the study to determine whether the anti-inflammatory properties of vitamin D may lead to asthma symptom improvement in this population.
- Finally, Dr. McCulloh and the Nebraska team were one of four ISPCTN sites to participate in the iAmHealthy pilot study during Cycle 1. This study was designed to evaluate the feasibility and efficacy of a remote, interactive, tablet-based behavioral intervention for obese children seen in rural primary care clinics, and the NPCTU successfully implemented the study in the Children’s Physicians clinic in Kearney. Based on the success of this pilot, the ISPCTN may pursue a larger randomized controlled trial later in Cycle 2.
Throughout Cycle 1, the NPCTU also provided infrastructure support to the Pediatric Research Office (PRO) and Child Health Research Institute (CHRI). The NPCTU grew to include a junior investigator, research nurse, clinical study coordinator and research data analyst, with additional funding to support a human factors analyst and grant writer for CHRI. The team also assisted with the development of the CHRI Research Specimen Laboratory and Nebraska Pediatric Research Registry.
“The NPCTU team has made great progress in building research capacity, training researchers and coordinators to conduct clinical trials, and extending research opportunities to children and communities across Nebraska,” commented Dr. McCulloh. “I am immensely grateful to everyone who has helped make our success possible.”
Looking Ahead to Cycle 2
The NPCTU team will continue to engage in ISPCTN clinical trials activities and contribute to the study and publication pipelines throughout the second award period. There will also be an increased emphasis on professional development during Cycle 2, with the addition of Kari Simonsen, MD, professor of Pediatric Infectious Diseases, as Senior Faculty Development Leader. She will provide mentorship to NPCTU junior investigators Kari Neemann, MD, assistant professor of Pediatric Infectious Diseases, and Ashley Deschamp, MD, assistant professor of Pediatric Pulmonology, focusing on clinical trials skills, leadership and team management, scientific writing skills and career development award submission. The ISPCTN also plans to engage junior faculty in the submission and evaluation of pilot proposals throughout the upcoming grant cycle.
Finally, the upcoming award period will see a renewed focus on rural outreach and engagement in Nebraska and throughout the Great Plains region. Kate Heelan, PhD, with the University of Nebraska at Kearney, will act as a co-investigator. She will assist the NPCTU with rural recruitment and retention activities, local healthcare provider training, and engagement of a rural community advisory board to discuss clinical trials protocols and identify areas for pediatric research that are important to the local community.
Dr. McCulloh eagerly anticipates continued achievements. “Despite the pandemic, our NPCTU team has enrolled dozens of patients across greater Nebraska in cutting-edge clinical trials. If the first four years are any clue, the next five years of the NPCTU will see fantastic growth in clinical trials opportunities and new knowledge that will improve the lives of Nebraska children.”
by Rachel Wellman and Matthew Sandbulte | 15 December 2020