Long Term Administration of Inhaled 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
- 318
- 试验地点
- 53
- 主要终点
- Change in Absolute FEV1 From Baseline Over 26 Weeks
研究概览
简要总结
The purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis. Previous studies have demonstrated improvements in lung function, mucociliary clearance, changes in physical properties of mucus, 24 hour sputum weight and quality of life. The results of this study are to further investigate and confirm these findings in addition to examine the effect on antibiotic use and chest infections. It is hypothesised that inhaled mannitol will have beneficial effects compared to a control treatment. An open label phase of 26 weeks duration will follow the blinded 26 week phase. During the open label phase all subjects will receive active 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
- •Have given written informed consent to participate in this study in accordance with local regulations
- •Have a confirmed diagnosis of cystic fibrosis (positive sweat chloride value ≥ 60 mEq/L) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype)
- •Be aged > 6 years old
- •Have FEV1 >40 % and < 90% predicted
- •Be able to perform all the techniques necessary to measure lung function
排除标准
- •Investigators, site personnel directly affiliated with this study, or their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted.
- •Be considered "terminally ill" or eligible for lung transplantation
- •Have had a lung transplant
- •Be using nebulized hypertonic saline in the 4 weeks prior to visit 1
- •Have had a significant episode of hemoptysis (>60 mL) in the three months prior to enrolment
- •Have had a myocardial infarction in the three months prior to enrolment
- •Have had a cerebral vascular accident in the three months prior to enrolment
- •Have had major ocular surgery in the three months prior to enrolment
- •Have had major abdominal, chest or brain surgery in the three months prior to enrolment
- •Have a known cerebral, aortic or abdominal aneurysm
- •Be breast feeding or pregnant, or plan to become pregnant while in the study
- •Be using an unreliable form of contraception (female subjects at risk of pregnancy only)
- •Be participating in another investigative drug study, parallel to, or within 4 weeks of visit 0
- •Have a known allergy to mannitol
- •Be using beta blockers
- •Have uncontrolled hypertension - systolic BP > 190 and / or diastolic BP > 100
- •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
- •Be 'Mannitol Tolerance Test positive'
研究组 & 干预措施
A
active treatment
干预措施: inhaled mannitol (Drug)
B
干预措施: Placebo comparator (Drug)
结局指标
主要结局
Change in Absolute FEV1 From Baseline Over 26 Weeks
时间窗: 26 weeks
Change from baseline in forced expiratory volume at one second (FEV1) averaged over 26 weeks (measured at 6,14 and 26 weeks) The mean absolute change from baseline FEV1 (mL) over 26 weeks (measured at week 6, 14 and 26) will be compared between the two treatment groups with a REML (restricted maximum likelihood) based repeated measures approach.Least square means presented are for the average change over the 6, 14, and 26 week visits.
次要结局
- Change From Baseline FEF25-75 (mL/s) Over 26 Weeks(26 weeks)
- Change in FEV1 From Baseline Over 26 Weeks - Dornase Users(26 weeks)
- Rate of Protocol Defined Pulmonary Exacerbations (PDPE)(26 weeks)
- Hospitalisations Associated With Protocol Defined Pulmonary Exacerbations (PDPEs)(26 weeks)
- Absolute Change in FEV1 Percent Predicted at 26 Weeks(26 weeks)
- Antibiotic Use Associated With PDPEs(26 weeks)
- Change in FVC (mL) Across 26 Weeks(26 weeks)
- Sputum Weight at Baseline in Response to First Dose of Treatment(up to 30 mins after first dose of trial treatment)