RLDC: Molecular Pathway-Driven Diagnostics & Therapeutics for Rare Lung Diseases

RLDC：分子途径驱动的诊断

基本信息

批准号：
8765116
负责人：
Bruce C Trapnell
金额：
$ 93.75万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
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：
2014-09-18 至 2019-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8765116
关键词：
Academic Medical Centers Advocacy Advocate Affect Alveolar Award Basic Science Biological Markers Blood Child Childhood Clinic Clinical Clinical Research Clinical Sciences Clinical and Translational Science Awards Collaborations Communities Development Diagnostic Diagnostic tests Disease Educational Materials Electronic Mail Enrollment Evaluation Family Figs - dietary Foundations Funding General Population Goals Grouping Hamman-Rich syndrome Health Hermanski-Pudlak Syndrome Institutes Institution Institutional Review Boards Internet Knowledge Lead Leadership Link Literature Longitudinal Studies Lung Lung diseases Lymphangioleiomyomatosis Medical Medical center Molecular Monitor Natural History Ohio Outcome Assessment Outcome Measure Pathogenesis Pathway interactions Patient advocacy Patients Pediatric Hospitals Pilot Projects Population Heterogeneity Program Development Provider Pulmonary Alveolar Proteinosis Pulmonary Fibrosis Rare Diseases Research Research Personnel Research Training Resources Site Support Groups Telephone Testing Therapeutic Therapy Clinical Trials Training Training Programs Training and Education Translational Research United States United States National Institutes of Health Universities Work Writing base career career development clinical care clinical research site design experience follow-up human FRAP1 protein improved innovation novel novel diagnostics novel therapeutics patient advocacy group patient oriented programs research study success therapeutic development therapy development treatment trial web site

项目摘要

DESCRIPTION (provided by applicant): We seek support for a Rare Lung Diseases clinical research Consortium (RLDC) facilitating clinical research, training, and education for a group of rare lung diseases based on molecular pathway-driven development of novel diagnostics and therapeutics. The proposed Specific Aims include 1) establish a Consortium focused to Lymphangioleiomyomatosis (LAM), Pulmonary Alveolar Proteinosis (PAP), and Hermansky-Pudlak Syndrome (HPS), 2) conduct longitudinal and therapeutic clinical studies related to LAM, PAP, and HPS at abroad network of rare lung disease clinical centers established initially via LAM clinics, 3) conduct of a Pilot and Demonstration program for the development and evaluation of novel diagnostics, therapeutics, and outcome measures for these and additional 'on-deck' rare lung diseases, 4) provide clinical research training at RLDC clinical centers and attract new investigators to the field, and 5) develop rare lung disease educational materials for patients, medical providers, and the public. Synergies between diseases include the use of quantitative radiological outcome assessment, an emphasis on development and evaluation of blood-based biomarkers and diagnostic tests, and the evaluation of the natural history of each disease. We build on prior successes including development of novel diagnostics (in PAP and LAM), the first successful therapeutic trial for LAM conducted through the RLDC ('MILES'), longstanding partnerships with patient advocacy groups, and a track record of training and career development in rare lung diseases (including a former trainee who is now a project leader in this application). The network consists of three translational sites at Cincinnati Children's (the coordinating center), the University of Cincinnati in Cincinnati, Ohio (a co-directorship), and Vanderbilt, and clinical research sites selected from the "existing LAM Clinic Network. Participating patient support groups include the LAM Foundation, PAP Foundation, HPS Network, and the Alpha-1 Foundation. These centers and Foundations are already closely integrated with clinical sites chosen from active networks of collaborating clinical centers that include over 28 sites in 22 states distributed throughout the United States. Participating translational centers will leverage resources from existing NIH Clinical and Translational Science Awards (CTSA) and a racially diverse population that includes pediatric opportunities. Each center provides ongoing longitudinal clinical studies, an excellent clinical training program, and active clinical programs designed to test novel therapies, develop diagnostic tests and evaluate outcome measures that will lead to new therapies and improve the lives of affected

描述（由申请人提供）：我们寻求支持罕见的肺部疾病临床研究联盟（RLDC），促进基于分子途径驱动的新型诊断和治疗疗法发展的一组稀有肺部疾病的临床研究，培训和教育。 The proposed Specific Aims include 1) establish a Consortium focused to Lymphangioleiomyomatosis (LAM), Pulmonary Alveolar Proteinosis (PAP), and Hermansky-Pudlak Syndrome (HPS), 2) conduct longitudinal and therapeutic clinical studies related to LAM, PAP, and HPS at abroad network of rare lung disease clinical centers established initially via LAM clinics, 3) conduct of a Pilot and针对这些新诊断，治疗学和结果的新诊断和评估的演示计划，并为这些疾病和其他“甲板”稀有肺部疾病，4）在RLDC临床中心提供临床研究培训，并吸引新的研究人员进入该领域，5）5）为患者，医疗提供者和公众开发稀有的肺部疾病教育材料。疾病之间的协同作用包括使用定量放射学结局评估，强调对血液的生物标志物和诊断测试的开发和评估，以及对每种疾病的自然史的评估。我们以先前的成功为基础，包括开发新型诊断（在PAP和LAM中），这是LAM通过RLDC（“ Miles”）进行的首次成功治疗试验，与患者倡导组的长期合作伙伴关系，以及稀有肺部疾病的培训和职业发展的记录（包括现在是该应用程序中的项目负责人）。该网络由辛辛那提儿童（协调中心），俄亥俄州辛辛那提大学（协调中心）的三个翻译站点组成，俄亥俄州辛辛那提（共同指导）和范德比尔特（Vanderbilt）以及临床研究站点，从“现有的LAM临床网络”中选择了“现有的LAM临床网络。从积极的临床中心中选择的临床网站，包括在美国的22个州中的28个地点，将利用现有的NIH临床和转化科学奖（CTSA）的资源。以及旨在测试新疗法，开发诊断测试并评估结果指标的活跃临床计划，这将导致新的疗法并改善受影响的生活