Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 340
- 试验地点
- 29
- 主要终点
- To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
研究概览
简要总结
The purpose of this study is to determine the efficacy and safety of chronic treatment with inhaled dry powder mannitol in subjects with cystic fibrosis. Previous studies have demonstrated an improvement in lung function related to small airways obstruction and a significant improvement in respiratory symptoms and quality of life after a 2 week treatment with mannitol. This current study seeks to support these early findings and to extend the evidence to support its use as a mucoactive therapy in cystic fibrosis. In particular, the hypothesis that enhanced mucus clearance will improve the lung function and clinical presentation in this population, will be investigated. We also hypothesize that enhanced mucociliary clearance will result in a sustained reduction in mucus load, thus providing less opportunity for bacteria to proliferate, affording a reduction in antibiotic use and hospitalizations. The initial 6 month blinded phase will be followed with an additional 6 months of open label treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent
- •Confirmed diagnosis of cystic fibrosis
- •Aged > 6 years
- •FEV1 >30 % and < 90% predicted
- •Able to perform all the techniques necessary to measure lung function
排除标准
- •"Terminally ill" or listed for lung transplantation
- •Had a lung transplant
- •Using nebulised hypertonic saline
- •Significant episode of haemoptysis (>60 mL) in the three months prior to enrolment
- •Recent myocardial infarction or cerebral vascular accident
- •Breast feeding or pregnant, or plan to become pregnant while in the study participating in another investigative drug study, parallel to, or within 4 weeks of study entry
- •Allergy or intolerance to mannitol
- •Using beta blockers
- •Have a condition or be in a situation which in the Investigator's opinion may put the subject at significant risk, may confound results or may interfere significantly with the patient's participation in the study
研究组 & 干预措施
1
干预措施: Mannitol (Drug)
2
干预措施: placebo (Drug)
结局指标
主要结局
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
时间窗: 6 months
次要结局
- To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF on existing RhDNase treatment compared to control. (key objective)(6 months)
- Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)(6 months / 12 months)
- Improves quality of life (key objective)(6 months)
- Reduces days on IV antibiotics, rescue oral or inhaled antibiotics(6 months / 12 months)
- Reduces days in hospital due to pulmonary exacerbations(6 months / 12 months)
- Improves other measures of lung function(6 months)
- Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)(6 months / 12 months)
- Reduces hospital and community care costs(6 months / 12 months)