UCLA IPF Clinical Research Network

加州大学洛杉矶分校 IPF 临床研究网络

• 批准号: 7060006
• 负责人: David Abram Zisman
• 金额: $ 19.38万
• 依托单位: UNIVERSITY OF CALIFORNIA LOS ANGELES
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-05-01 至 2010-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7060006
• 关键词: clinical research; clinical trial phase III; combination chemotherapy; cooperative study; human subject; human therapy evaluation; interferon gamma; patient oriented research; phosphodiesterase inhibitors; pulmonary fibrosis /granuloma; pulmonary hypertension; respiratory disorder chemotherapy; sirolimus

DESCRIPTION (provided by applicant): The overall objective of this proposal is to discover novel therapy for the treatment of IPF. We propose two projects. The first project aims to determine the safety and efficacy of sirolimus combined with interferon gamma-1b (IFN-y lb) in 138 patients with idiopathic pulmonary fibrosis (IPF). We propose a two-year multi-center, randomized, double-blinded, controlled study of sirolimus plus IFN-y lb compared to IFN-y lb plus placebo in patients with IPF. Eligible patients will be randomly assigned, in a 2:1 ratio, to receive sirolimus plus IFN-y lb versus IFN-y lb plus placebo, respectively. Randomization will use a randomized permuted block design stratified by clinic with changing block sizes. The primary efficacy outcome is the 6-minute walk distance (6MWD). Secondary efficacy endpoints include pulmonary function, gas exchange, and quality of life (QOL). We hypothesize that patients with mild to moderate IPF (FVC >55% and DLCO >30% of predicted) will demonstrate stabilization or improvement in 6MWD over 96 weeks of treatment with combined therapy. The control group will be expected to demonstrate deterioration in 6MWD. The second project aims to determine the safety and efficacy of sildenafil in 100 patients with IPF and moderate pulmonary arterial hypertension (PAH; defined as meaning pulmonary arterial pressure >30 mm Hg at rest). We propose a 12-week multi-center, randomized, double-blinded, controlled study of sildenafil compared placebo in patients with IPF and PAH. Eligible patients will be equally randomized (1:1) into active or placebo groups. Randomization will use the permuted block design with stratification by clinic. The primary efficacy outcome is the 6MWD. Secondary efficacy endpoints include gas exchange and quality of life (QOL). We anticipate an improvement in 6MWD in patients who receive sildenafil compared to those who receive placebo. (End of Abstract)

描述（由申请人提供）： 本提案的总体目标是发现治疗IPF的新疗法。 我们提出两个项目。 第一个项目旨在确定西罗莫司联合干扰素γ-1b（IFN-γ 1b）治疗138例特发性肺纤维化（IPF）患者的安全性和有效性。 我们提出了在IPF患者中西罗莫司加IFN-γ Ib与IFN-γ Ib加安慰剂相比的两年多中心、随机、双盲、对照研究。 将符合条件的患者以2：1的比例随机分配，分别接受西罗莫司加IFN-γ Ib与IFN-γ Ib加安慰剂。 随机化将使用随机排列区组设计，按诊所分层，改变区组大小。 主要疗效结局是6分钟步行距离（6 MWD）。 次要疗效终点包括肺功能、气体交换和生活质量（QOL）。 我们假设轻度至中度IPF患者（FVC >55%，DLCO >30%预测值）在联合治疗96周后，6 MWD将稳定或改善。 预计对照组将显示6 MWD恶化。 第二个项目旨在确定西地那非在100例IPF和中度肺动脉高压（PAH;定义为静息时肺动脉压>30 mm Hg）患者中的安全性和疗效。 我们提出了一项为期12周的多中心、随机、双盲、对照研究，在IPF和PAH患者中比较西地那非与安慰剂。 合格患者将被随机（1：1）平均分配至活性药物组或安慰剂组。 随机化将采用排列区组设计，并按诊所分层。 主要疗效结局为6 MWD。 次要疗效终点包括气体交换和生活质量（QOL）。 我们预计与接受安慰剂的患者相比，接受西地那非的患者6 MWD有所改善。 (End摘要）