Pilot-Feasibility Core

ABSTRACT The NASCARR Pilot-Feasibility Governance Core will serve to accelerate innovation in patient-centered sex chromosome aneuploidy (SCA) research. The core is fundamentally designed to facilitate success of translational research that has direct applicability to patient communities in domains with significant stakeholder investment. To do so, the core will leverage substantial collective resources that will be assembled through the overarching NASCARR RDCRC. This encompasses significant human resources in the form of a comprehensive and collaborative network of SCA researchers, families, expert mentors; and also material and structural resources, including access to financial sponsorship, recruitment channels and administrative support. The ultimate goal of pilot-feasibility awards will be to generate innovative investigations into various domains that are most likely to advance the SCA field toward clinical trial readiness, including (but not limited to) biomarker discovery as associated with common phenotypes associated with morbidity/mortality in SCA conditions; creation and/or validation of data collection tools and salient outcome measures specific to SCA, validation of novel interventions and diagnostic tools in treatment of individuals with SCAs. It is expected that the longitudinal outcomes of funded awards will be successful translation of pilot-feasibility into larger scale clinical trials with the potential to directly impact clinical care of individuals with SCA. To accomplish these goals, the NASCARR Pilot-Feasibility Governance Core is broadly centered on three aims: Firstly, establishment of a robust pilot study grant mechanism – the core will develop infrastructure using best practices in scientific review, to solicit, evaluate and fund innovative research in SCA. Processes will include broad dissemination of a request for proposals to the SCA research and associated communities inviting letters of intent and subsequently, full applications which will be reviewed for impact, innovation, rigor and relevance, by content experts. Meritorious applications will be considered for funding by the NASCARR Executive Council. Secondly, funded awardees will have access to a comprehensive network of established SCA peers, and mentors, as well as resource support, to optimize success of selected grants. This includes intellectual exchange with like-minded researchers, forums to review and incorporate up-to-date understanding of SCA research frameworks, and facilitated access to a broad network of resources within the NASCARR network and the RDCRN more broadly. Regular meetings and milestones will be established to ensure greatest likelihood of success in transitioning from pilot awards to future clinical trials. Lastly, the core itself will undergo regular and systematic review/evaluation to ensure the mission of the core is being met, while also allowing intentional iterations on program structure as required year over year. This includes outcome evaluation of funded awards and success of selected principal investigators, as well as review of progress reports and presentations of completed awards to the NASCARR Executive Council, the DMCC and NIH, and dissemination of findings to stakeholder communities.