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Hydroxychloroquine Treatment for Prevention of Systemic Lupus Erythematosus

羟氯喹治疗预防系统性红斑狼疮

基本信息

批准号：
8996136
负责人：
DAVID R KARP
金额：
$ 32.1万
依托单位：
PENNSYLVANIA STATE UNIV HERSHEY MED CTR
依托单位国家：
美国
项目类别：
财政年份：
2015
资助国家：
美国
起止时间：
2015-02-01 至 2017-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8996136
关键词：
Adult Affect Agreement Applications Grants Biological Markers Budgets Case Report Form Child Classification Clinical Clinical Protocols Clinical Trials Conduct Clinical Trials Consent Forms Controlled Clinical Trials Cross-Sectional Studies Data Databases Development Disease Disease Progression Double-Blind Method Early Diagnosis Early treatment Elements End stage renal failure Enrollment Event Goals Grant Health Heart Hydroxychloroquine Immune response Individual Institution Intervention Kidney Laboratories Longevity Lung Lupus Manuals Measures Minority Morbidity - disease rate Office for Human Research Protections Organ Outcome Patients Pharmaceutical Preparations Pharmacotherapy Phase Placebo Control Placebos Population Population Intervention Populations at Risk Positioning Attribute Prevention Procedures Protocols documentation Questionnaires Request for Proposals Research Research Personnel Risk Sampling Secure Site Skin Source Staging Statistical Data Interpretation Survival Rate Symptoms System Systemic Lupus Erythematosus Target Populations Testing Training Work base data management design drug mechanism evidence base high risk improved outcome innovation insight novel outcome forecast placebo controlled study potential biomarker pre-clinical prevent primary outcome prospective randomized placebo controlled trial retention rate secondary outcome tool treatment trial young woman

项目摘要

DESCRIPTION: Systemic lupus erythematosus (SLE) is a disease that causes major organ damage and shortens lifespan. Early diagnosis and treatment are essential to improving outcomes for SLE patients. However, tools for the reliable identification of SLE in early stages and evidenced based approaches to early treatment are not available. We propose that individuals who are at high risk of SLE can be identified in early or preclinical stages with sufficient reliability to serve as a target population for initiating treatment with a safe therapythat has potential to delay or abort full expression of disease. This population has some elements of lupus but does not fully satisfy classification criteria, and may be referred to as incomplete lupu erythematosus (ILE). The present proposal requests support for the planning phase of a multicenter, placebo controlled trial of hydroxychloroquine in ILE patients to determine whether this intervention can ameliorate, delay or prevent progression to SLE. The planned trial will be carried out with intensive sampling for potential biomarkers to elucidate underlying disease and drug mechanisms. During the planning phase, the four cooperating sites will finalize a protocol, prepare all needed study documents and manuals, secure a source for the medication and placebo and obtain regulatory approvals. In addition, mock recruitment scenarios will be carried out to confirm that enrollment is feasible. The results of this planning grant will be a UM1 application to conduct the clinical trial that incorporates all of the developed materials and that will be ready for implementation. The long-term objectives are to change the trajectory of lupus management by targeting early events that can be modulated to prevent damage and improve outcomes.

描述：系统性红斑狼疮（SLE）是一种导致主要器官损伤和缩短寿命的疾病。早期诊断和治疗对于改善系统性红斑狼疮患者的预后至关重要。然而，在早期阶段的SLE和证据为基础的早期治疗方法的可靠识别工具是不可用的。我们建议SLE高危个体可以在早期或临床前阶段被识别出来，并具有足够的可靠性，可以作为开始安全治疗的目标人群，有可能延迟或中止疾病的完全表达。该人群具有狼疮的一些要素，但不完全满足分类标准，并且可以被称为不完全红斑狼疮（ILE）。本提案要求支持在ILE患者中进行羟氯喹多中心、安慰剂对照试验的计划阶段，以确定这种干预是否可以改善、延迟或预防SLE进展。计划中的试验将进行密集采样，以获得潜在的生物标志物，以阐明潜在的疾病和药物机制。在计划阶段，四个合作研究中心将最终确定一项方案，准备所有需要的研究文件和手册，确保药物和安慰剂的来源，并获得监管部门的批准。此外，还将进行模拟招募场景，以确认招募是否可行。这项计划拨款的结果将是一个UM1申请进行临床试验，其中包括所有开发的材料，将准备实施。长期目标是通过针对早期事件来改变狼疮管理的轨迹，这些事件可以被调节以防止损害并改善结果。