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）：我们寻求对罕见肺病临床研究联盟（RLDC）的支持，该联盟基于分子途径驱动的新型诊断和治疗方法的开发，促进一组罕见肺病的临床研究、培训和教育。拟议的具体目标包括 1) 建立一个专注于淋巴管平滑肌瘤病 (LAM)、肺泡蛋白沉积症 (PAP) 和赫曼斯基-普德拉克综合征 (HPS) 的联盟，2) 开展与 LAM、PAP 和 HPS 相关的纵向和治疗性临床研究最初通过 LAM 诊所建立的国外罕见肺病临床中心网络，3) 开展试点和示范计划，以开发和对这些和其他“现场”罕见肺病的新诊断、治疗和结果测量进行评估，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) 开发罕见肺病教育材料对于患者、医疗服务提供者和公众。疾病之间的协同作用包括使用定量放射学结果评估、强调基于血液的生物标志物和诊断测试的开发和评估以及每种疾病的自然史的评估。我们以先前的成功为基础，包括开发新型诊断方法（PAP 和 LAM）、通过 RLDC（“MILES”）进行的首次成功的 LAM 治疗试验、与患者倡导团体的长期合作伙伴关系以及培训和职业发展的跟踪记录罕见肺部疾病（包括一名前实习生，现在是该应用程序的项目负责人）。该网络由位于辛辛那提儿童医院（协调中心）、俄亥俄州辛辛那提辛辛那提大学（联席主任）和范德比尔特大学的三个转化中心以及选自“现有 LAM 诊所网络”的临床研究中心组成。团体包括 LAM 基金会、PAP 基金会、HPS 网络和 Alpha-1 基金会。这些中心和基金会已经与从活跃网络中选择的临床站点紧密结合。合作临床中心，包括分布在美国 22 个州的超过 28 个中心，参与的转化中心将利用现有 NIH 临床和转化科学奖 (CTSA) 的资源以及包括儿科在内的多元化人群。临床研究，优秀的临床培训计划，以及旨在测试新疗法、开发诊断测试和评估结果措施的积极临床计划，这些措施将带来新疗法并改善受影响者的生活