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Provisional Combined Response Index For Patients With Diffuse Systemic Sclerosis

弥漫性系统性硬化症患者的临时综合缓解指数

基本信息

批准号：
7678566
负责人：
Daniel Eric Furst
金额：
$ 34.86万
依托单位：
UNIVERSITY OF CALIFORNIA LOS ANGELES
依托单位国家：
美国
项目类别：
财政年份：
2007
资助国家：
美国
起止时间：
2007-08-01 至 2012-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7678566
关键词：
American Area Under Curve Arthritis Characteristics Clinical Clinical Research Clinical Trials Clinical Trials Design Consensus Cutaneous Data Data Set Development Diffuse Diffuse Cerebral Sclerosis of Schilder Diffuse Scleroderma Disease Exercise Face Funding Goals Gold Heterogeneity Individual International Language Lead Limited Scleroderma Measurement Measures Meta-Analysis Methodology Morbidity - disease rate Natural History Observational Study Organ Outcome Outcome Measure Paper Pathogenesis Patient Outcomes Assessments Patients Principal Investigator Receiver Operating Characteristics Research Research Personnel Rheumatism Rheumatoid Arthritis Rheumatology Scleroderma Sclerosis Sensitivity and Specificity Skin Specific qualifier value Structure Systemic Scleroderma Techniques Testing Therapeutic Agents Time Treatment Efficacy Validation Validity and Reliability Work base college design drug development effective therapy improved indexing interest mortality patient oriented primary outcome programs prospective response sound

项目摘要

DESCRIPTION (provided by applicant): Systemic sclerosis Scleroderma, (SSc) can be associated with a high morbidity and mortality. The complexity and heterogeneity of the disease mandates a composite response index that will capture different organ involvement and patient-reported outcomes. There is a critical need for an SSc-combined response index (SSc-CRI) to facilitate drug development and to allow comparison among clinical trials in SSc, as a similar index did in rheumatoid arthritis. We have assembled a group of international experts who have expertise in clinical trial design and development of response criteria in different arthritides. UCLA Clinical Coordinating Center has successfully coordinated recently concluded NIH-funded SSc clinical trials. We took the first step in the developing an SSc-CRI by conducting a structured Delphi exercise to develop a provisional core set of items for clinical trials. The Delphi exercise identified 11 domains relevant to diffuse SSc clinical trials. Our long-term goals are to improve methodologically sound SSc clinical trials and increase interest in drug development for SSc. This application aims to develop an SSc-CRI to be used as an outcome measure in diffuse SSc clinical trials. Our central hypothesis is that a core set of variables for patients with diffuse SSc can be used to develop a formula for an SSc-CRI with robust test characteristics. Our Specific Aims for the 3-year proposal are: Specific Aim 1. Perform a prospective longitudinal observational clinical study in 200 patients with diffuse SSc to define a reliable, valid and responsive set of SSc measures for an SSc-CRI, based on a recently completed Delphi exercise. Specific Aim 2. Employ a prospective, data-driven, consensus building techniques to develop and quantitatively evaluate candidate definitions for an SSc-CRI for diffuse SSc. The research proposed in this application is significant because it is expected to provide a single SSc-CRI for diffuse SSc, thereby providing an impetus for drug development and improved assessment of efficacy of therapeutic agents in clinical trials. Lay Language: We propose to develop a response criteria for clinical trials in diffuse scleroderma that can facilitate drug development and hopefully, can lead to effective treatments for scleroderma.

描述（由申请人提供）：系统性硬化症硬皮病（SSc）可能与高发病率和死亡率相关。该疾病的复杂性和异质性要求采用综合反应指数来捕获不同的器官受累情况和患者报告的结果。迫切需要 SSc 联合反应指数 (SSc-CRI) 来促进药物开发并允许在 SSc 临床试验之间进行比较，就像类风湿性关节炎中的类似指数一样。我们聚集了一批国际专家，他们在不同关节炎的临床试验设计和反应标准制定方面拥有专业知识。加州大学洛杉矶分校临床协调中心已成功协调最近结束的 NIH 资助的 SSc 临床试验。我们通过进行结构化德尔菲练习来开发临床试验的临时核心项目集，迈出了开发 SSc-CRI 的第一步。 Delphi 练习确定了与弥漫性 SSc 临床试验相关的 11 个领域。我们的长期目标是改进方法学上合理的 SSc 临床试验并提高对 SSc 药物开发的兴趣。本申请旨在开发 SSc-CRI，用作弥漫性 SSc 临床试验的结果测量。我们的中心假设是，弥漫性 SSc 患者的一组核心变量可用于开发具有稳健测试特征的 SSc-CRI 公式。我们的 3 年提案的具体目标是：具体目标 1. 在 200 名弥漫性 SSc 患者中进行一项前瞻性纵向观察性临床研究，根据最近完成的德尔菲练习，为 SSc-CRI 定义一套可靠、有效且反应灵敏的 SSc 测量方法。具体目标 2. 采用前瞻性、数据驱动的共识构建技术来开发和定量评估弥漫性 SSc 的 SSc-CRI 候选定义。本申请中提出的研究意义重大，因为它有望为弥漫性 SSc 提供单一的 SSc-CRI，从而推动药物开发并改进临床试验中治疗剂疗效的评估。外行语言：我们建议制定弥漫性硬皮病临床试验的反应标准，以促进药物开发，并有望带来硬皮病的有效治疗方法。