Pediatric Heart Network - GATHER - between Washington University in St Louis and Children's Hospital Colorado

PROJECT SUMMARY Over the past 70 years, research supported by the Pediatric Heart Network (PHN) and the National Heart, Lung, and Blood Institute (NHLBI), among other organizations, has led to significant advances in congenital heart disease (CHD) care and outcomes. The GATeway to tHe wEst consoRtium (GATHER) is a collaboration between Washington University School of Medicine/St. Louis Children’s Hospital and the University of Colorado/Children's Hospital Colorado, aimed at continuing this progress as a PHN Clinical Research Center in the next funding cycle. Recognizing the importance of new and emerging research tools as well as key priorities for future research in CHD, the consortium brings together diverse expertise and resources in basic, clinical, and translational research, including multi-omics research, health services and outcomes research, machine learning and big data approaches, digital health technology, and social determinants of health (SDoH). The GATHER consortium is rooted in a strong track record of past and ongoing collaborations between investigators and is committed to training junior investigators using a shared mentorship model. The GATHER consortium is committed to enhancing diverse perspectives in clinical research through the recruitment and retention of diverse study populations and the training of junior investigators from diverse backgrounds. Strategies such as community engagement, leveraging technology for data collection, and making study participation more accessible are outlined. GATHER represents a unique addition to PHN’s roster of core sites by drawing from a racially, ethnically, and socioeconomically diverse patient population in the Midwest and Mountain regions, areas that have historically been under-represented in PHN studies. The GATHER consortium plans to develop a multi-center prospective registry, PROTECT-HF: Pediatric Registry Of heart failure Therapies to Embed Clinical Trials, its design and content informed by clinical experts, patient and family advocates, and community partners. The goals of this registry will be to study real world practice variation in guideline-directed medical therapy for heart failure, to examine the role of SDoH on healthcare utilization and outcomes, to characterize responders and non-responders to GDMT, and to develop risk-prediction models for disease-specific outcomes. This work will be facilitated by novel analytic approaches and automated data extraction to optimize efficiencies and insights. The PROTECT-HF registry will then be used as a framework in which to embed clinical trials of novel heart failure therapies, starting with an exploratory phase Ib/II clinical trial of omecamtiv mecabril, a cardiac myosin activator. The GATHER consortium and the PROTECT-HF registry’s goals align with the PHN mission of studying and improving health outcomes in pediatric congenital and acquired heart disease, engaging and supporting families, and training and educating new investigators.