Planning for Prevention of Parkinson's Disease: a trial design forum

帕金森病预防规划：试验设计论坛

• 批准号: 10827547
• 负责人: MICHAEL A SCHWARZSCHILD
• 金额: $ 6万
• 依托单位: MASSACHUSETTS GENERAL HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-15 至 2028-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10827547
• 关键词: Planning; Prevention; Parkinson; Disease; trial

Project summary/ abstract: The proposed conference, “Planning for Parkinson’s Prevention: A trial design forum” will be held May 5th to 7th, 2024, and as a biannual series recurring in 2026 and 2028. The overall aim of this educational program is to facilitate the rational design and timely implementation of the first clinical trials for the prevention of Parkinson’s disease (PD). It will be pursued through six specific aims (SAs): SA #1 is to educate attendees on the critical design elements for PD prevention trials via symposium sessions. SA #2 is to gain regulatory perspective on how the selection of outcomes and other design features may be informed by evolving, biomarker-based disease staging systems, and how these design features may support a pre-diagnostic therapeutic indication. SA #3 is to foster partnership and collaboration within the emerging community of stakeholders in PD prevention, including the unique, newly developing advocacy constituency of people at substantially increased risk of PD based on their genetic or prodromal features. SA #4 is to facilitate pre-competitive knowledge exchange and collaboration among researchers developing the first prevention trials by inviting presentations and panel discussions of their early protocols. SA #5 is to enhance diversity among trainees, investigators, and subsequently trial participants, via targeted outreach to underrepresented groups in medicine. SA #6 is to ensure enduring and wide access to the knowledge and insights gained at the meetings through their peer-reviewed, open-access publication. The conference series will engage, educate, and motivate a diverse collaborative community of prevention stakeholders to accelerate development of rigorously designed early trials for PD prevention, thereby enhancing prospects for their success.

项目总结/摘要： 拟议的会议，“规划帕金森病的预防：一个试验设计论坛”将于5月举行 第五至第七，2024年，并作为一个两年期系列在2026年和2028年重复。本次教育的总体目标 该计划是为了促进合理设计和及时实施的第一个临床试验， 帕金森病（PD）的预防。它将通过六个具体目标来实现：SA #1是 通过专题讨论会向与会者介绍PD预防试验的关键设计要素。SA #2 是获得关于如何选择结果和其他设计特征的监管观点， 通过不断发展的，基于生物标志物的疾病分期系统，以及这些设计特征如何 支持诊断前治疗适应症。SA #3是促进内部的伙伴关系和协作 PD预防的新兴利益相关者社区，包括独特的，新发展的 根据其遗传或遗传学特征，PD风险大幅增加的人群的倡导群体 前驱特征SA #4是促进竞争前知识交流和合作， 研究人员通过邀请他们的演讲和小组讨论来开发第一个预防试验， 早期协议SA #5旨在增强学员、研究者和后续试验之间的多样性 参与者，通过有针对性的外联，以代表性不足的群体在医学。#20066;，以确保持久 以及通过同行评审广泛获得会议上获得的知识和见解， 开放获取出版物。会议系列将参与，教育和激励多元化的 预防利益攸关方协作社区，以加快制定严格设计的 预防PD的早期试验，从而提高其成功的前景。