PILOT-FEASIBILITY

项目概要（试点/可行性核心） BBDC试点/可行性核心（PFC）的目标是在我们1-5年的经验基础上， 继续从BBDC内部和外部征集、审查、资助和报告试点研究 研究人员社区，用于利用BBDC基础设施和数据的研究项目。最终 这些试点项目的目标是推进BBDC的使命，特别是与临床试验有关的使命 在成骨不全症以及更广泛的罕见骨疾病方面做好准备。在第1-5年，两名飞行员 已在先前的申请中提出，两项试验研究均已完成。这些数据表明， 通知的设计，纵向研究的OI（项目1），特别是在该地区的焦虑和疼痛 干扰OI），并导致了两篇关于PROMIS和患者参与的出版物， research.此外，我们还制定了征求、审查和资助新试点研究的程序。 这导致BBDC内外提交了17份提案。我们最终资助了 关于胶原蛋白X生物标志物测量的项目，支持当前项目的科学前提 3关于OI生长的纵向生物标志物研究的建议。我们建议扩大和改善这项试验计划 通过广泛征求OI和骨骼健康研究人员的广泛社区的应用程序，我们 将继续以NIH的形式进行审查，同时强调支持BBDC的项目 使命，利用BBDC基础设施和数据，并推进临床试验准备。

PROJECT SUMMARY (Pilot/Feasibility Core) The objective of the BBDC Pilot/Feasibility Core (PFC) is to build on our experience in years 1-5 to continue a solicitation, review, funding, and reporting of pilot studies from both inside and outside the BBDC investigator community for research projects that leverage the BBDC infrastructure and data. The ultimate goal for these pilot projects is to advance the mission of the BBDC, especially as it relates to clinical trial readiness in Osteogenesis Imperfecta as well as more broadly on rare bone diseases. In years 1-5, two pilots were proposed in the prior application and both pilot studies have been completed. These produced data that informed the design of the longitudinal study of OI (Project 1) specifically in the area of anxiety and pain interference in OI), and that led to two publications on the topic of PROMIS and patient engagement in research. In addition, we also developed a process for soliciting, reviewing, and funding of new pilot studies. This resulted in submission of 17 proposals from both inside and outside of the BBDC. We ultimately funded on project on collagen X biomarker measurement which supported the scientific premise of the current Project 3 proposal on longitudinal biomarker study of growth in OI. We propose to extend and improve on this pilot mechanism by broadly soliciting applications from a wide community of OI and bone health researchers. We will continue a review process that is in the NIH format while emphasizing projects that support the BBDC mission, leverage the BBDC infrastructure and data, and advance clinical trial readiness.