Abstract

In the planning and design of the Radicava/Edaravone Findings in Biomarkers From Amyotrophic Lateral Sclerosis (REFINE-ALS) study, we sought to elicit feedback from patients with ALS and their caregivers to ensure that patient-centric issues would be addressed. Ten ALS Clinical Research Learning Institute (ALS-CRLI) Research Ambassadors participated in 2 meetings. They provided perspectives on patients’ interest in the study, the schedule of study visits, and data sharing. The findings were used to help revise the study design, as appropriate. Key concerns identified were (1) the frequency of sample collections, (2) participant travel burden, (3) enrollment criteria, and (4) data reporting and sharing with participants. Several of the identified issues were promptly addressed. The number of visits was reduced, travel optimized, entry criteria clarified, and plans for sharing participants’ data with them were codified. The feedback from the Ambassadors was substantive and resulted in constructive patient-centric changes to the study protocol.

摘要

在R adicava / E daravone的规划和设计中来自A myotrophic Lateral S的生物标志物的发现在硬化症 (REFINE-ALS) 研究中，我们试图从 ALS 患者及其护理人员那里获得反馈，以确保解决以患者为中心的问题。十名 ALS 临床研究学习机构 (ALS-CRLI) 研究大使参加了 2 次会议。他们就患者对研究的兴趣、研究访问的时间表和数据共享提供了观点。研究结果被用于帮助修改研究设计，视情况而定。确定的主要问题是 (1) 样本收集的频率，(2) 参与者的旅行负担，(3) 登记标准，以及 (4) 数据报告和与参与者共享。一些已查明的问题得到及时解决。减少了访问次数，优化了旅行，明确了进入标准，并制定了与他们共享参与者数据的计划。