An Open-label, Pilot Study to Assess Safety, Tolerability, Pharmacokinetics and Effects of Inhaled PC945 in the Pre-emptive Treatment of Aspergillus Fumigatus Colonisation in Lung Transplant Recipients
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 3
- 主要终点
- Proportion of participants receiving PC945 who meet the markedly abnormal criteria for 12-lead ECG assessment at lease once post dose
研究概览
简要总结
This study tests the effects of pre-emptive treatment with an experimental drug PC945 in lung transplant recipients whose lungs are infected by the fungus Aspergillus fumigatus.
PC945 may be useful in treating patients infected with Aspergillus fumigatus as, unlike the usual treatments, it is inhaled into the lung and has been designed to stay there and treat the infection. Participants will be monitored for up-to 12 weeks for the presence of Aspergillus in their lungs. Suitable participants will receive PC945 for an initial 28 days (Pre-emptive treatment phase) and, if needed, a further 8-weeks (Extended treatment phase).The amount of fungus in the patients' lungs will be measured over the course of the study. Participants with lung infections but not eligible for PC945 will be followed-up for 16-weeks on standard of care treatment. The study will take place at multiple sites in UK and 10 participants will receive PC945. The maximum study duration will be about 28 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 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
PC945
干预措施: PC945 (Drug)
Standard of Care
Standard of care anti-fungal medication
干预措施: Standard of Care (Drug)
结局指标
主要结局
Proportion of participants receiving PC945 who meet the markedly abnormal criteria for 12-lead ECG assessment at lease once post dose
时间窗: Baseline to Week 16
Forced vital capacity (FVC) values from participants receiving PC945
时间窗: Baseline to Week 16
Proportion of participants receiving PC945 who meet the markedly abnormal criteria for safety laboratory assessment at lease once post dose
时间窗: Baseline to Week 16
Predicted post bronchodilator forced expiratory volume in 1 second (FEV1) values from participants receiving PC945
时间窗: Baseline to Week 16
Adverse events (AEs)
时间窗: 48 hours Post Transplant to Week 16 post treatment
Maximum plasma concentration
时间窗: Baseline to Week 16
Derived pharmacokinetic parameters for PC945 and the potential circulating metabolite(s), if detectable
Proportion of participants receiving PC945 who meet the markedly abnormal criteria for vital signs assessment at lease once post dose
时间窗: Baseline to Week 16
Area under the curve from time 0 to 2 h post-dose (AUC0-2)
时间窗: Baseline to Week 16
Derived pharmacokinetic parameters for PC945 and the potential circulating metabolite(s), if detectable
Concentration at the end of the dosage interval (Ctrough)
时间窗: Baseline to Week 16
Derived pharmacokinetic parameters for PC945 and the potential circulating metabolite(s), if detectable
次要结局
- Lung Concentration of PC945(Baseline to Week 16)
- A. fumigatus fungal culture status (presence or absence) in subjects with a baseline A. fumigatus-positive bronchoalveolar lavage (BAL)(Baseline to Week 16)
- Change in BAL A. fumigatus measured by quantitative polymerase chain reaction (qPCR)(Baseline to Week 16)
- Aspergillus status (Presence or absence) of BAL using an Aspergillus immunochromatographic lateral flow device (AspLFD).(Baseline to Week 16)
- Subject experience of inhaled amphotericin B(Baseline to Week 16)
- Change in the number of sputum A. fumigatus colony forming units (CFU) in cultured BAL.(Baseline to Week 16)
- Galactomannan levels in BAL(Baseline to Week 16)
- Status of fungal hyphae or pseudohyphae (presence or absence) on cytological examination(Baseline to Week 16)
- Subject experience of inhaled PC945(Baseline to Week 16)