Rare Lung Disease Clinical Research Network

描述（申请人提供）：我们寻求建立一个研究网络，通过1）促进已经专注于罕见肺部疾病临床研究的中心之间的合作，2）吸引和培训高素质的研究人员，3）从地理上分散的患者收集临床数据到一个大型的集中数据库，以及4）将积累的临床数据提供给受罕见肺病影响或可能受罕见肺病影响的人、他们的临床医生、临床和基础研究者以及公众。该网络最初关注的疾病包括：α-1抗胰蛋白酶缺乏症（AATD）、淋巴管平滑肌瘤病（LAM）、肺泡蛋白沉积症（PAP）和遗传性特发性肺纤维化（hIPF）。该网络由俄亥俄州（协调中心）、科罗拉多、佛罗里达、马里兰州、马萨诸塞州、俄勒冈州、南卡罗来纳州和德克萨斯州以及日本和澳大利亚的临床中心组成。要求中心拥有并保持积极临床研究的典型记录和足够的罕见肺病患者基础。参与的患者支持团体包括Alpha-1基金会、LAM基金会和肺纤维化基金会。其中许多中心已经密切合作。这些中心和基金会也已经紧密结合。例如，所有三个参与基金会的科学主任都是网络内临床研究中心的活跃临床研究人员。此外，临床研究中心选自三个目前活跃的合作临床中心网络，包括分布在美国24个州的50多个研究中心。所有参与的国内临床研究中心均与NIH支持的活跃的一般临床研究中心（GCRC）相关。每个中心都提供拟议网络所需的组件，包括正在进行的纵向临床研究，优秀的临床培训计划，旨在测试新疗法的积极临床研究试验计划，开发诊断测试或评估罕见肺部疾病的结果措施。 每个基金会都为病人、普通公众和医学界提供教育。重要的是，准备这一申请的一个结果是成立了“罕见肺病基金会联盟”，该联盟允许拥有更大基础设施的患者支持团体“培育”欠发达团体的发展。它还为受目前没有基础的罕见肺部疾病影响的个人提供支持。在选定的中心进行的临床，基础和转化研究已经对肺功能和健康和疾病防御的分子机制产生了重要的见解。

DESCRIPTION (provided by applicant): We seek to a establish a research network that will facilitate clinical research in rare lung diseases by 1) promoting collaboration among centers already focused to clinical research on rare lung diseases, 2) attracting & training highly qualified investigators, 3) collecting clinical data from geographically distributed patients into a large, centralized database, and 4) making the accumulated clinical data available to those affected or possibly affected by a rare lung disease, their clinicians, clinical and basic investigators and the general public. Disorders chosen for the initial focus of this network include: alpha-1 antitrypsin deficiency (AATD), lymphangioleiomyomatosis (LAM), pulmonary alveolar proteinosis (PAP) and hereditary idiopathic pulmonary fibrosis (hIPF). The network consists of clinical centers in Ohio (the coordinating center), Colorado, Florida, Maryland, Massachusetts, Oregon, South Carolina and Texas, as well as in Japan and Australia. Centers are required to have and maintain an exemplary record of active clinical research and an adequate rare lung disease patient base. Participating patient support groups include the Alpha-1 Foundation, LAM Foundation and the Pulmonary Fibrosis Foundation. Many of these centers already work together closely. These centers and Foundations are also already closely integrated. For example, the Scientific Directors of all three participating foundations are active clinical investigators at clinical sites within the network. Furthermore, clinical sites were chosen from three currently active networks of collaborating clinical centers that include over 50 sites in 24 states distributed throughout the United States. All participating domestic clinical sites are associated with an active, NIH-supported general clinical research center (GCRC). Each center provides components required of the proposed network including ongoing longitudinal clinical studies, an excellent clinical training program, an active clinical research trials program designed to test novel therapies, develop diagnostic tests or evaluate outcome measures for rare lung diseases. Each of the Foundations provide education for patients, the lay public and the medical community. Importantly, one consequence of preparing this application has been the formation of the "Rare Lung Disease Foundation Consortium," which permits patient support groups with greater infrastructure to "nurture" the growth of less well-developed groups. It also provides support for individuals affected by a rare lung disease for which there is currently no foundation. Ongoing clinical, basic and translational studies at the centers chosen have already yielded critical insights into molecular mechanisms underlying lung function and defense in health and disease.