A Pilot Trial of Intravenous Iron for the Treatment of Iron Deficiency in Adult Patients With Cystic Fibrosis
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron
研究概览
简要总结
This pilot interventional cohort study will examine the effects of intravenous iron in adults with cystic fibrosis and iron deficiency.
详细描述
Iron deficiency is common in adults with cystic fibrosis, and is associated with adverse outcomes. Oral iron supplementation is poorly tolerated and may be ineffective. In some centres, intravenous iron is used to correct iron deficiency, but concerns have been raised about the safety of this treatment in the setting of chronic airways infection. The investigators are therefore planning a pilot interventional cohort study examining the effects of intravenous iron in a group of adults with cystic fibrosis. Patients will be recruited in Oxford and studied prospectively over 16 weeks, with iron given at week 4. The primary focus of this single-centre pilot/feasibility study is safety, specifically in relation to infection.
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
- •Age ≥18 years with established diagnosis of cystic fibrosis
- •Iron deficiency (transferrin saturation ≤16 % or ferritin <15 μg/l, within last 4 months)
排除标准
- •Urgent (<6 weeks) need for iron supplementation
- •Active infection (currently requiring IV antibiotics)
- •Previous intravenous iron supplementation (within last 4 months)
- •Current oral iron supplementation
- •Hypersensitivity to ferric carboxymaltose
- •Active non-tuberculous mycobacterial pulmonary disease (as defined by ATS-IDSA criteria)
- •Liver failure
- •Ferritin >300 μg/l or transferrin saturation >45%
- •Pregnancy or breast feeding
- •Previous transplantation
- •Judged by member of trial team to be unlikely to comply with safety aspects of trial
研究组 & 干预措施
Intravenous iron
All participants will receive a single dose of intravenous ferric carboxymaltose
干预措施: Ferric carboxymaltose (Drug)
结局指标
主要结局
Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron
时间窗: 8 weeks
New infective events are defined as any of: 1. New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study) 2. Clinical infection requiring IV antibiotics (as determined by clinical team) 3. Admission to hospital for infection-related reason (as determined by clinical team) 4. Significant deterioration in lung function (\>10% fall in FEV1), not otherwise explained (as determined by clinical team)
次要结局
- Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron(16 weeks (plus 8 weeks of retrospective data collection from notes))
- Change in number of antibiotic days(16 weeks)
- Change in body mass index(16 weeks)
- Change in abundance of sputum Pseudomonas(16 weeks)
- Change in exercise capacity (shuttle walk test)(16 weeks)
- Change in lung function (FEV1)(16 weeks)
- Change in arterial oxygen saturation(16 weeks)
- Change in sputum microbiological diversity(16 weeks)
- Change in quality of life (SF-36 questionnaire)(16 weeks)
- Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)(16 weeks)
- Percentage of patient in whom each outcome is successfully measured(16 weeks)
- Change in quality of life (CFQ-R questionnaire)(16 weeks)
- Percentage of eligible patients entering and completing the study(16 weeks)