Pilot-Feasibility

Hugo W. Moser Res Inst Kennedy Krieger, Baltimore MD

Investigators

Abstract

PILOT STUDY CORE – PROJECT SUMMARY/ABSTRACT The Batten Disease Clinical Research Consortium (BDCRC) Pilot Study Core will be a cornerstone of the Consortium's overarching mission to advance knowledge and provide pragmatic research approaches to advance early diagnosis, management, and treatment; to improve clinical trial readiness; and to address other key unmet areas of need for the Batten disease community. Through a highly collaborative, patient-centric approach, the Pilot Study Core will solicit, rigorously evaluate, and fund high-quality, innovative translational and clinical pilot studies. Studies will address one or more of the Top Research Priorities identified through a recent survey of Batten disease community stakeholders, developed using an established Research Priority Setting Partnership methodology. Leveraging existing collaborations, resources, and infrastructure of the BDCRC, along with the Batten Disease Support, Research, and Advocacy Foundation, the Batten Disease Global Research Initiative, and other strategic partners, the Pilot Study Core will solicit proposals from a wide range of researchers, including early career investigators and established investigators from outside the Batten disease field. The Pilot Core Committee will draw upon its diverse professional expertise to employ a systematic, rigorous, NIH-based process for evaluating and selecting at least one project per year based on eligibility, relative merit, feasibility, and alignment with stakeholders' Top Research Priorities. The Core will facilitate the successful execution and completion of these pilot studies though provision of practical support and mentorship throughout the entire lifecycle of the project: from commencement of the Award (Phase I), through the active research period (Phase II), project completion (Phase III), and the post-research phase (Phase IV). By providing end-to-end support for promising, innovative proof-of-concept studies, the Pilot Study Governance Core will be a central component of the BDCRC and its mission to address unmet research needs and to meaningfully improve clinical outcomes for those living with Batten disease.