A Phase 2, Open-Label, Dose-Escalation Study in Subjects With Pulmonary Arterial Hypertension, (PAH, WHO Group 1) and Pulmonary Hypertension Secondary to Idiopathic Pulmonary Fibrosis, (PH-IPF WHO Group 3) Using Inhaled GeNOsyl.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 31
- 试验地点
- 10
- 主要终点
- Identify the minimally and maximum effective doses of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.
研究概览
简要总结
A Phase 2 open label, dose escalation study to find the minimally and maximum effective dose (dose beyond which no further effect on PVR is seen) of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.
详细描述
Up to 54 subjects undergoing RHC are planned in this study, and shall include subjects meeting eligibility criteria classified as WHO Group 1 PAH or WHO Group 3 IPF-PH. Subjects will receive inhaled nitric oxide from the GeNOsyl® System to characterize the hemodynamic response and evaluate safety and tolerability.
Dose cohorts of approximately 5, 15, and 20 ppm nitric oxide in air will be studied. Different dose levels will be achieved by varying the flow rate of the drug substance (80 ppm NO2 in balance air) delivered to the subject via nasal cannula. Each subject will receive two different doses of inhaled nitric oxide separated by a placebo (medical grade air or supplemental oxygen) washout. Eligible subjects will be assigned to a dosing cohort in an escalating manner to receive study drug (80 ppm nitric oxide in air.)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Dosing Group 1
1 Liter per Minute (LPM)of inhaled nitric oxide via nasal cannula: approximately 5 parts per million (ppm)
干预措施: Inhaled Nitric Oxide (Drug)
Dosing Group 2
2 LPM of inhaled nitric oxide via nasal cannula: approximately 15 ppm
干预措施: Inhaled Nitric Oxide (Drug)
Dosing Group 3
4 LPM of inhaled nitric oxide via nasal cannula: approximately 20 ppm
干预措施: Inhaled Nitric Oxide (Drug)
结局指标
主要结局
Identify the minimally and maximum effective doses of inhaled nitric oxide generated by the GeNOsyl® System compared to placebo.
时间窗: through end of Right Heart Catheterization procedure (Treatment Phase approximately 3 hours)
Assess mean change in pulmonary vascular resistance (PVR) for study drug dose 2 compared to placebo. Assess change from pre-dose to end-of-hemodynamic assessment for study drug dose 1. Assess change from placebo to end-of-hemodynamic assessment for study drug dose 2.
次要结局
- Evaluate the pharmacokinetics of total nitrates/nitrites and methemoglobin produced following inhalation of nitric oxide via the GeNOsyl® System.(up through 24 hrs after treatment period for subjects participating in PK sub-study)
- Assess the safety and tolerability of nitric oxide generated by the GeNOsyl® System in subjects with WHO Group 1 PAH and WHO Group 3 PH-IPF.(through end of study (approximately 30 days after treatment visit))