Nebraska Pediatric Clinical Trials Unit

Although clinical trials are the highest standard of evidence for the effectiveness and safety of medical interventions, children often receive medical therapies that have never been studied in clinical trials involving pediatric subjects. Knowledge of the etiology, prevention, and/or treatment of these illnesses often must be extrapolated from adult-derived data. Children often respond to medications differently from adults due to differences in physiology and drug metabolism, placing them at increased risk of experiencing adverse, even life-threatening events from off-label medical treatment use. Clinical trials furnish patients opportunities to receive well-supervised treatments and procedures before they are widely available, gain improved understanding of their health conditions, and contribute to knowledge that benefits others. Importantly for IDeA states like Nebraska, children from communities with lower access to advanced medicine (e.g., rural regions) are less likely to have access to clinical trials. During the first two cycles of the ECHO IDeA States Pediatric Clinical Trials Network (ISPCTN), the Nebraska Pediatric Clinical Trials Unit (NPCTU) has led the first network-developed randomized controlled trial, enrolled participants in almost all ISPCTN studies, and expanded human and organizational capital in Nebraska to conduct clinical trials statewide. The NPCTU is poised to build upon this success to ensure that all Nebraska children have access to clinical trials. We propose the following Specific Aims to drive continued growth in clinical trials access and participation for Nebraska children: (1) Develop, conduct, and disseminate findings from multicenter clinical trials research, assuring the participation of children living in IDeA states. The NPCTU will solicit trial proposals from community stakeholders and faculty and propose at least three multicenter clinical trial concepts to the ISPCTN, participate in implementing all five anticipated multicenter network trials, including our proposed trial to test the effectiveness of a multi-level mHealth-based intervention to increase adherence to recommended infant immunizations and lab screenings. (2) Build pediatric clinical trial research capacity at the clinical site. The NPCTU will continue to expand clinical trials performance capabilities by enacting a multifaceted training and mentoring plan for developing early-stage faculty into proficient leaders of multicenter clinical trials and train clinical and research staff to conduct trials. (3) Engage interested parties such as community members, nonprofit organizations, and professional societies to enhance ECHO ISPCTN clinical trial impact, transferability, rigor, and feasibility. The NPCTU will sustain and build on our robust engagements with professional societies that issue pediatric clinical practice guidelines and with a wide range of individual- and organization-level community stakeholders. Existing and new collaborations will provide rich insight into communities’ research priorities, help us deploy trials in ways that more fully engage Nebraska children, and disseminate results back to communities.