A Phase 1b, Multi-center Study of Intravenous (IV) Gallium Nitrate in Patients With Cystic Fibrosis (CF) Who Are Colonized With Nontuberculous Mycobacteria (NTM) (The ABATE Study)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 10
- 主要终点
- Adverse Events of Special Interest
研究概览
简要总结
The purpose of this study is to assess the safety and tolerability of two 5-day infusion cycles of IV gallium in adult patients with CF who are infected with NTM.
Funding Source - FDA Office of Orphan Products Development (OOPD)
详细描述
This is a prospective, multicenter open-label study in adults with CF who are colonized with M. avium complex and/or M. abscessus complex.
Participants are enrolled into one of the two cohorts:
- Treatment-Naïve NTM Cohort: participants with persistently positive NTM culture results (either M. avium complex, M. abscessus complex, or both M. avium and M. abscessus) who had never been treated for current NTM species or previous treatment was associated with clearance of NTM.
- Treatment-Refractory NTM Cohort: participants with persistently positive NTM culture results (either M. avium complex, M. abscessus complex, or both M. avium and M. abscessus) whose treatment failed to respond to 12 months of NTM guideline-based therapy or currently receiving treatment with guideline-based antibiotics for > 12 months.
Subjects will receive two 5-day infusion cycles of IV gallium. The study will evaluate the safety and antimycobacterial effect of two 5-day infusions of IV gallium.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 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 —（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
Infusion of IV Gallium
Gallium nitrate will be infused continuously over 5 days at 200 mg/m2/day. Study drug will be administered via a peripheral IV catheter, a peripherally inserted central catheter (PICC) line, midline catheter, or a chronic indwelling vascular access device using an ambulatory infusion pump infused over 24 hours for 5 sequential days for each cycle. There is a maximum of 2 cycles.
干预措施: Gallium nitrate (Drug)
结局指标
主要结局
Adverse Events of Special Interest
时间窗: Baseline to Day 57
Proportion of patients experiencing one or more Adverse Events of Special Interest (AESI). AESIs include the occurrence of either (1) a serious adverse event (SAE) of grade 3 or higher including hospitalizations or (2) study drug discontinuation because of an AE.
次要结局
- NTM clearance (efficacy)(Day 6 to Day 111)
- Clinically significant abnormal laboratory measures. (safety)(Baseline to Day 57)
研究者
Chris Goss
Professor of Medicine and Pediatrics, University of Washington
Seattle Children's Hospital