Planing an Early RA Prevention Trial

描述（由申请人提供）： 该规划补助金的目标是完成类风湿性关节炎（RA）一级和二级预防临床试验的开发，并为试验的实施建立基础设施。 该试验名为RAPIDT（通过立即治疗预防风湿性关节炎），旨在确定DMARD治疗对早期滑膜炎患者的影响。该研究将有5个组，包括接受DMARD治疗（甲氨蝶呤单药或甲氨蝶呤+英夫利西单抗）3个月或12个月的患者，对照组接受常规治疗3个月。主要结局为治疗结束时的缓解和停止治疗后1年的缓解。因此，这项试验解决了“机会之窗”假设。预计将向NIH提交一笔赠款，用于本试验的协调中心，其他资金来自行业。为此，建议提供规划补助金，以完成试验设计和制定操作程序以及确定地点。提出了三个具体目标：1）完成协议的开发。这一具体目标将包括最终确定试验设计，完善主要和次要结局指标，并解决旨在诱导缓解的试验中的统计分析问题; 2）在RA预防策略的背景下解决知情同意问题。由于RAPIDT设计为预防研究，因此知情同意过程将不同于过去的其他RA试验。这一目标将考虑早期治疗问题，以建立适当的知情同意; 3）完成操作和程序手册，实施数据管理方法，并确定执行地点。这一目标将为试验建立基础设施，并提供所有必要的要素，以允许入组开始。

DESCRIPTION (provided by applicant): The goal of this planning grant is to complete development of a clinical trial for primary and secondary prevention of rheumatoid arthritis (RA) and to establish the infrastructure for the performance of the trial. The trial is titled RAPIDT (Rheumatoid Arthritis Prevention by Immediate Definitive Therapy) and is designed to determine the impact of DMARD therapy in patients with early synovitis. The study will have 5 arms consisting of patients administered DMARD therapy (methotrexate alone or methotrexate plus infliximab) for either 3 months or 12 months, with a control arm receiving conventional therapy for 3 months. The primary outcomes will be remission at the end of treatment and remission 1 year after the cessation of therapy. As such, this trial addresses the "window of opportunity" hypothesis. It is anticipated that a grant will be submitted to the NIH for a coordinating center for this trial, with other funding obtained from industry. A planning grant is proposed at this tune to finalize trial design and develop procedures of operation as well as identify sites. Three specific aims are proposed: 1) To complete development of the protocol. This specific aim will include finalization of the trial design, refining primary and secondary outcome measures and addressing issues of statistical analysis in a trial aimed at the induction of remission; 2) To address issues of informed consent in the context of prevention strategies for RA. Since RAPIDT is designed to be a prevention study, the informed consent process will differ from other trials on RA in the past. This aim will consider issues of early treatment to construct an appropriate informed consent; and 3) To complete a manual of operations and procedures, implement approaches for data management and identify sites for performance. This aim will establish the infrastructure for the trial and provide all the essential elements to allow enrollment to begin.