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A Study to Assess the Safety and Efficacy of Treprostinil to Facilitate Liver Transplantation in Patients With Portopulmonary Hypertension

2017年1月5日 更新者：United Therapeutics

An Open-Label Study to Assess the Safety and Efficacy of Treprostinil to Facilitate Liver Transplantation in Patients With Portopulmonary Hypertension

This was a multicenter, prospective, observational, open-label study. Patients meeting inclusion/exclusion criteria received treatment with treprostinil as recommended by their treating physicians and were followed according to standard of care. This observational study collected clinical data and biologic specimens from patients who were treated for portopulmonary hypertension (PoPH), with a goal of achieving hemodynamic parameters appropriate for orthotopic liver transplantation candidacy, including mean pulmonary arterial pressure (mPAP) less than 35 mmHg and pulmonary vascular resistance (PVR) less than 3 Wood-units (WU) at Week 24 in patients with severe PoPH.

研究概览

地位

完全的

条件

干预/治疗

详细说明

Treprostinil is approved as a continuous subcutaneous (SC) or intravenous (IV) infusion by the FDA for the treatment of WHO group I PAH with New York Heart Association (NYHA) Functional Class II, III or IV symptomatology. To date, treprostinil has not been studied in the setting of PoPH; however, it is commonly prescribed in this setting. This was an observational, open-label, multicenter study which documented the safety and efficacy profile of this agent in PoPH to facilitate orthotopic liver transplantation (OLT).

研究类型

观察性的

注册 (实际的)

13

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 美国
    • California
      • Los Angeles、California、美国、90024
        • University of California, Los Angeles
    • Georgia
      • Atlanta、Georgia、美国、30322
        • Emory Univeristy
    • Massachusetts
      • Boston、Massachusetts、美国、02115
        • Brigham and Women's Hospital
    • Texas
      • Dallas、Texas、美国、75235
        • University of Texas, Southwestern Medical Center

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 孩子
  • 成人
  • 年长者

接受健康志愿者

不

有资格学习的性别

全部

取样方法

概率样本

研究人群

Subjects were recruited from 4 liver transplantation centers in the US, referred for portopulmonary hypertension.

描述

Inclusion Criteria:

  • Patients must:

    1. Had portal hypertension.
    2. Be otherwise suitable candidates for OLT.
    3. Had severe pulmonary arterial hypertension (PAH) defined as a resting mean pulmonary arterial pressure (mPAP) >35 mmHg and pulmonary vascular resistance (PVR) ≥3 Wood Units (WU) by right heart catheterization (RHC) performed as part of standard of care evaluation within 90 days of enrollment.
    4. Treprostinil therapy must be recommended by the treating physician per standard of care.
    5. Be NYHA Functional Class II, III, or IV.
    6. Had pulmonary capillary wedge (PCW) pressure ≤18 mmHg and transpulmonary gradient (TPG) ≥15 mmHg.

Exclusion Criteria:

  • Patients must not:

    1. Had received any any investigational therapy as part of a clinical trial for any indication within 30 days prior to enrollment.
    2. Had a change in dose of treatment for PAH (bosentan [Tracleer], ambrisentan [Letairis], tadalafil [Adcirca], or sildenafil [Revatio]), within 30 days prior to enrollment. That is, subjects may have been treated with any of these agents provided the dose was stable for at least 30 days prior to enrollment.
    3. Had renal failure requiring hemodialysis.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

队列和干预

团体/队列
干预/治疗
Portopulmonary hypertension
药品：Treprostinil

Remodulin is supplied in concentrations of 1, 2.5 , 5, and 10 mg/mL and can be administered as supplied or diluted for intravenous (IV) infusion prior to administration.

Remodulin is indicated for subcutaneous (SC) or IV use only as a continuous infusion. Remodulin is preferably infused subcutaneously, but can be administered by a central IV line if the SC route is not tolerated. The infusion rate is initiated at 1.25 ng/kg/min. If this initial dose cannot be tolerated because of systemic effects, the infusion rate should be reduced to 0.625 ng/kg/min.

其他名称：
  • 重组蛋白

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Number of Subjects Who Achieved Hemodynamic Parameters Appropriate for Orthotopic Liver Transplantation Candidacy at Week 24.
大体时间：24 Weeks
The primary efficacy endpoint was the number of subjects who achieved a mean pulmonary arterial pressure (mPAP) less than 35 mmHg and a pulmonary vascular resistance (PVR) less than 3 Wood units (WU) at Week 24 in patients with severe portopulmonary hypertension (PoPH).
24 Weeks

次要结果测量

结果测量
措施说明
大体时间
Change in Hemodynamic Parameters (Via Right Heart Catheterization [RHC]) at Rest From Baseline to Week 24
大体时间：24 weeks
The change in hemodynamic parameters (including systolic pulmonary arterial pressure [PAPs], diastolic pulmonary arterial pressure [PAPd], mean pulmonary arterial pressure [mPAP], and transpulmonary gradient [TPG]) was evaluated at rest from Baseline to Week 24. The median change in hemodynamic parameters from Baseline to Week 24 via right-heart catheterization (RHC) is presented.
24 weeks
Change in Heart Rate at Rest From Baseline to Week 24
大体时间：24 weeks
The change in heart rate was evaluated at rest from Baseline to Week 24.
24 weeks
Change in Cardiac Output at Rest From Baseline to Week 24
大体时间：24 weeks
The change in cardiac output was evaluated at rest from Baseline to Week 24. The median change in cardiac output from Baseline to Week 24 is presented.
24 weeks
Change in Arterial and Venous Oxygen Saturation at Rest From Baseline to Week 24
大体时间：24 weeks
The change in arterial and venous oxygen saturation was evaluated at rest from Baseline to Week 24.
24 weeks
Change in Pulmonary Vascular Resistance (PVR) at Rest From Baseline to Week 24
大体时间：24 weeks
The change in pulmonary vascular resistance (PVR) was evaluated at rest from Baseline to Week 24.
24 weeks
Change in 6-minute Walk Distance (6MWD) From Baseline to Weeks 12 and 24.
大体时间：Baseline and Weeks 12 and 24
The 6-Minute Walk Test was conducted at Screening, Baseline prior to starting study drug and at least 24 hours after the Screening test, and during the Treatment Phase at Weeks 12 and 24.
Baseline and Weeks 12 and 24
Change in Echocardiogram Parameters (Right Atrium and Right Ventricle Area) From Baseline to Weeks 12 and 24
大体时间：Baseline and Weeks 12 and 24
Standard transthoracic echocardiogram with continuous wave Doppler and color flow imaging was completed at Screening. All patients who were enrolled in this study underwent an echocardiogram within 30 days of enrollment as well as repeat echocardiograms on Weeks 12 and 24.
Baseline and Weeks 12 and 24
Change in Echocardiogram Parameters (Right Ventricle Diameter) From Baseline to Weeks 12 and 24
大体时间：Baseline and Weeks 12 and 24
Standard transthoracic echocardiogram with continuous wave Doppler and color flow imaging was completed at Screening. All patients who were enrolled in this study underwent an echocardiogram within 30 days of enrollment as well as repeat echocardiograms on Weeks 12 and 24.
Baseline and Weeks 12 and 24
Change in Echocardiogram Parameters (Right Ventricular Systolic Pressure) From Baseline to Weeks 12 and 24
大体时间：Baseline and Weeks 12 and 24
Standard transthoracic echocardiogram with continuous wave Doppler and color flow imaging was completed at Screening. All patients who were enrolled in this study underwent an echocardiogram within 30 days of enrollment as well as repeat echocardiograms on Weeks 12 and 24.
Baseline and Weeks 12 and 24
Change in Echocardiogram Parameters (Tricuspid Annular Plane Systolic Excursion) From Baseline to Weeks 12 and 24
大体时间：Baseline and Weeks 12 and 24
Standard transthoracic echocardiogram with continuous wave Doppler and color flow imaging was completed at Screening. All patients who were enrolled in this study underwent an echocardiogram within 30 days of enrollment as well as repeat echocardiograms on Weeks 12 and 24.
Baseline and Weeks 12 and 24
Change in Quality of Life From Baseline to Weeks 12 and 24
大体时间：Baseline and Weeks 12 and 24
The 36-item Short Form Survey (SF-36) is a health related quality of life instrument, which measures dimensions of physical and social roles and functioning, mental health, vitality, and pain. Items are scored on a 0 to 100 range so that the lowest scores represent the highest disability. The quality of life assessment was conducted at Baseline and Weeks 12 and 24 and the change from Baseline to Weeks 12 and 24 is presented.
Baseline and Weeks 12 and 24
Change in Plasma Brain N-terminal Pro-brain Natriuretic Peptide (NT-proBNP) From Baseline to Weeks 12 and 24
大体时间：Baseline to Weeks 12 and 24
NT-proBNP was assessed at Baseline, Weeks 12 and 24.
Baseline to Weeks 12 and 24

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

United Therapeutics

合作者

University of California, Los Angeles
Emory University
Brigham and Women's Hospital
University of Texas

调查人员

  • 研究主任：Rajan Saggar, MD、University of California, Los Angeles
  • 研究主任：Micah Fisher, MD、Emory University
  • 研究主任：Aaron Waxman, MD, PhD、Brigham and Women's Hospital
  • 研究主任：Sonja Bartolome, MD、UT Southwestern Medical Center

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始

2011年1月1日

初级完成 (实际的)

2013年4月1日

研究完成 (实际的)

2013年4月1日

研究注册日期

首次提交

2009年12月8日

首先提交符合 QC 标准的

2009年12月8日

首次发布 (估计)

2009年12月9日

研究记录更新

最后更新发布 (实际的)

2017年2月24日

上次提交的符合 QC 标准的更新

2017年1月5日

最后验证

2017年1月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • RIV-PH-414

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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条件

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