NCT00469027已完成1 期
Phase I Trial to Establish Safety of Autologous Progenitor Cell-based Gene Therapy Delivery of heNOS in Patients With Severe Pulmonary Arterial Hypertension(PAH)Refractory to Conventional Treatment
Northern Therapeutics4 个研究点 分布在 1 个国家目标入组 7 人开始时间: 2006年5月1日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 7
- 试验地点
- 4
- 主要终点
- The primary endpoints will be related to the tolerability and safety of injection of genetically engineered progenitor cells in patients with severe PAH.
研究概览
简要总结
The primary objective is to establish the safety of autologous progenitor cell-based gene therapy of heNOS in patients with severe Pulmonary Arterial Hypertension(PAH) refractory to conventional treatment.
详细描述
This is a two centre, phase I clinical trial. A total of 18 patients will be studied using an open-label, dose escaling protocol; three patients will be entered into each of the five dosing panels. An additional three patients will be entered into the final dose panel to establish safety at the maximum tolerated dose.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of idiopathic Pulmonary Arterial Hypertension
- •Familial PAH or anorexigen induced PAH
- •Specified 6-minute walk distance
排除标准
- •Intra or extra cardiac communication between the right and left sided circulations
- •Hemodynamic instability
- •Left ventricular ejection fraction < 40%
- •Thromboembolic event or recent hospitalization for worsening right sided heart failure in past 3 months
- •CVP>20mmHg at time of research heart catheterization
- •Pregnancy
- •Concurrent hepatitis or HIV
结局指标
主要结局
The primary endpoints will be related to the tolerability and safety of injection of genetically engineered progenitor cells in patients with severe PAH.
时间窗: 5 years
次要结局
- Potential efficacy of this approach will be assessed by changes in hemodynamic pressures, patient perceived quality of life and exercise capacity(3 month post cell delivery)
研究者
研究点 (4)
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