IPF Clinical Trial Center at Vanderbilt

描述（由申请人提供）： 特发性肺纤维化（IPF）是一种慢性进行性肺部疾病，目前尚无有效的治疗方法。 大多数患者的结局包括限制性肺病在3-5年内逐渐进展为呼吸衰竭（半数患者）。 患病率估计值表明，美国有100，000例IPF患者，是因终末期肺病等待肺移植的患者中最常见的死亡原因。 许多可用的药物具有预测IPF结局改善的作用，但尚未在患者中进行测试。 至关重要的是，应及时开展设计良好的多中心、随机、安慰剂对照试验，以检查是否可以确定IPF的有效治疗。 我们计划在一项多中心、随机、安慰剂对照临床试验中确定依维莫司治疗IPF的效果，该试验将入组240例IPF患者。 在随后的360例IPF患者试验中，我们将在一项多中心、随机、安慰剂对照临床试验中确定泼尼松或吡非尼酮或两者联合治疗IPF的效果。 最后，我们将作为临床中心全面参与IPF临床研究网络指导委员会指导的所有多中心、随机、安慰剂对照试验。 (End摘要）

DESCRIPTION (provided by applicant): Idiopathic Pulmonary Fibrosis (IPF) is a chronic progressive lung disease with no proven therapy. The outcome in most patients includes gradual progression of restrictive lung disease to respiratory failure in half the patients within a period of 3-5 years. Estimates for prevalence suggest that there are 100,000 patients in the US with IPF, and it is the commonest cause of death among patients awaiting lung transplant for end-stage lung disease. Many available pharmaceutical agents have actions which would predict improved outcomes in IPF but have not been tested in patients. It is crucial that well-designed, multi-center, randomized, placebo-controlled trials be conducted promptly to examine whether an effective therapy for IPF can be identified. We plan to determine the effects of treatment of IPF with everolimus in a multi-center, randomized, placebo-controlled clinical trial which will enroll 240 IPF patients. In a subsequent trial of 360 IPF patients, we will determine the effects of treatment of IPF with prednisone or pirfenidone, or both, in a multi-center, randomized, placebo-controlled clinical trial. Finally, we will participate fully as a clinical center in all of the multi-center, randomized, placebo-controlled trials as directed by the Steering Committee of the IPF Clinical Research Network. (End of Abstract)