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.
