Nicotine Treatment for Pulmonary Sarcoidosis: A Clinical Trial Pilot Study

尼古丁治疗肺结节病：临床试验试点研究

基本信息

批准号：
8753389
负责人：
ELLIOTT D CROUSER
金额：
$ 24.65万
依托单位：
OHIO STATE UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2014
资助国家：
美国
起止时间：
2014-09-01 至 2017-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8753389
关键词：
Abate Adrenal Cortex Hormones Adult Adverse effects Affect Animal Model Antigens Attenuated Cells Chronic Clinic Clinical Clinical Trials Clinical Trials Design Computers Data Disease Dose Double-Blind Method Drug Addiction Dyspnea Exposure to Fatigue Female Foundations Future Goals Gold Granuloma Granulomatous Health Human Image Analysis Immune Immune response Industry Institution Interstitial Lung Diseases Investigation Knowledge Link Lung Lung diseases Mediating Metric Minor Mission Multi-Institutional Clinical Trial Nicotine Nicotinic Receptors Obstructive Lung Diseases Ohio Pathogenesis Patients Phase Pilot Projects Population Pulmonary Sarcoidosis Pulmonary function tests Qualifying Quality of life Randomized Randomized Controlled Clinical Trials Randomized Controlled Trials Refractory Research Research Infrastructure Risk Safety Sarcoidosis Serious Adverse Event Site Smoking Study Subject Surveys Symptoms Testing Texture Time Tobacco United States National Institutes of Health Universities Vital capacity X-Ray Computed Tomography base burden of illness case control cigarette smoking clinical efficacy clinically relevant computerized conventional therapy cost effective design disability efficacy trial experience improved large bowel Crohn&apos s disease middle age nicotine patch novel patient population response tool

项目摘要

DESCRIPTION (provided by applicant): Sarcoidosis is a common interstitial lung disease of unknown cause for which current therapies are often ineffective and/or inaccessible. The ultimate goal of this project is to deliver a safe alternative to current therapies that will be wel-tolerated and immediately available to sarcoidosis patients. In this regard, chronic exposure to nicotine-containing (tobacco) products is consistently shown to reduce the risk of developing sarcoidosis, and recent studies, including our pilot clinical trial, indicate that nicotine has potnt immune modulating effects that could alter the course of pulmonary sarcoidosis. We hypothesize that nicotine treatment will be efficacious for the treatment of pulmonary sarcoidosis. The Clinical Trial Pilot Study proposed herein will provide data required for the planning of subsequent, definitive multi-institutional trials to assess clinical efficacy. Namely, Aim 1 will assess changes in forced vital capacity (FVC) in response to nicotine treatment. Aim 2 will determine if nicotine treatment reduces the burden of lung disease, using objective radiographic criteria, compared to conventional therapies alone. This aim will employ a novel computerized lung CT image analysis tool that is demonstrated to sensitively detect various manifestations of pulmonary sarcoidosis. Aim 3 will consider alternative clinical endpoints for a future Phase II and III studies, including improvements in fatigue and disease-specific symptoms. Aim 4 will assess the safety and tolerability of sustained-release transdermal nicotine in the pulmonary sarcoidosis population. In this regard, nicotine has an extensive track record of safety in patients with obstructive lung disease (e.g., smoking-related), and was well tolerated in our previous pilot study. However, study subject compliance and safety will be important determinants used for the design of future clinical efficacy studies. The participating centers, Ohio State University and Cleveland Clinic Foundation, have large sarcoidosis patient populations and extensive experience with sarcoidosis clinical trials, and are well-qualified to complete this study.

描述（由申请人提供）：结节病是一种原因不明的常见间质性肺病，目前的治疗方法通常无效和/或无法实现。该项目的最终目标是为结节病患者提供一种耐受性良好且可立即使用的安全替代疗法。在这方面，长期接触含尼古丁（烟草）产品始终被证明可以降低患结节病的风险，最近的研究，包括我们的试点临床试验，表明尼古丁具有有效的免疫调节作用，可能会改变肺病的病程。结节病。我们假设尼古丁治疗对肺结节病有效。本文提出的临床试验试点研究将为规划后续、明确的多机构试验以评估临床疗效提供所需的数据。也就是说，目标 1 将评估尼古丁治疗引起的用力肺活量 (FVC) 的变化。目标 2 将使用客观的放射学标准，与单独的传统疗法相比，确定尼古丁治疗是否可以减轻肺部疾病的负担。该目标将采用一种新型计算机化肺部 CT 图像分析工具，该工具被证明可以灵敏地检测肺结节病的各种表现。目标 3 将考虑未来 II 期和 III 期研究的替代临床终点，包括疲劳和疾病特异性症状的改善。目标 4 将评估缓释透皮尼古丁在肺结节病人群中的安全性和耐受性。在这方面，尼古丁在阻塞性肺病（例如与吸烟相关）患者中具有广泛的安全性记录，并且在我们之前的试点研究中具有良好的耐受性。然而，研究对象的依从性和安全性将成为未来临床疗效研究设计的重要决定因素。参与中心俄亥俄州立大学和克利夫兰诊所基金会拥有大量结节病患者群体和丰富的结节病临床试验经验，并且完全有资格完成这项研究。