Randomized, Controlled Crossover Trial to Evaluate the Effects of Ambulatory Oxygen on Health Status in Patients With Fibrotic Lung Disease (FLD).
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- change in K-BILD The King's Brief Interstitial Lung Disease Questionnaire health status score
研究概览
简要总结
The main aim of this project is to establish whether ambulatory oxygen in patients with fibrotic ILD whose oxygen saturation falls ≤ 88% on a 6MWT, leads to a significant improvement in health status. The core of the project will be a four week randomised, crossover controlled trial of ambulatory oxygen used during daily activities. The optimal Oxygen flow rate is determined by titration at screening visit and administered during activity for a two-week period, compared to two weeks off oxygen.
详细描述
The planned non-commercial study is a randomised, controlled crossover trial of ambulatory oxygen against no ambulatory oxygen over a four week period (two weeks on ambulatory oxygen and two weeks on air, with no portable devices), to evaluate the effects of ambulatory oxygen on health status in patients with ILD. A short crossover study in this context has many advantages, since ambulatory oxygen has immediate effect, with no wash-out period needed after use. The primary outcome of the study will be the change in health status, as measured by the K-BILD questionnaire (Thorax 2012 67: 804-810).
At the start of the trial, the effects of ambulatory oxygen on 6 Minute Walk Test (6MWT) parameters will be evaluated on oxygen and on air-filled canisters, with the patient blind to the contents of the canister, to assess whether oxygen-induced improvements in 6MWT parameters can predict its effectiveness in day to day life. The 6MWT is a well established and highly reproducible test validated in ILD patients, with significant prognostic implications.
The study design does not include a placebo arm because:
- The intervention is a combination of possible benefits from oxygen and the disadvantage of canister weight. These cannot be separated. Placebo control is impossible because there is no means of providing placebo weight. Attempts to control solely for oxygen use without taking canister weight into account are clinically meaningless. In a recent COPD, chronic obstructive pulmonary disease study, cylinder weight was reported as a barrier to use by 93% of study participants.
- A positive result against an air-filled canister arm would be clinically uninterpretable. This is a study design in which the 'placebo' would be actively harmful to study participants. Carriage of an air-filled cylinder would be expected to lead to earlier desaturation and reduced exercise tolerance. Such a design would not inform the real life comparison between oxygen plus cylinder and no intervention.
- All this aside, blinding is legally impossible (UK health and safety regulations require that oxygen cylinders for home use must be clearly labelled).
It should also be stressed that objective measures of change are evaluated as secondary end-points: the investigators expect to explore correlations between these variables and the primary end-point to exclude the possibility that an observed treatment benefit on the primary end-point might be confounded by a placebo effect.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Single (Participant)
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
- •IPF or another fibrotic ILD (including fibrotic NSIP, fibrotic organising pneumoni, and fibrotic hypersensitivity pneumonitis)
- •patients aged 18 - 99 yrs
- •Desaturation ≤ 88% on a 6MWT on room air
- •Stable respiratory symptoms in the 4 weeks preceding the trial including the run in period
排除标准
- •Patients meeting criteria for long term oxygen therapy, including hypercapnic patients
- •Patients expected to change treatment during the course of the study
- •Significant locomotor or communication difficulties and/or severe co-morbidities
- •Patients with sarcoidosis or connective tissue disease affecting the musculoskeletal system
- •Current smokers
- •Pregnancy
研究组 & 干预措施
ambulatory oxygen cylinders
Subjects randomised to have the intervention of portable oxygen for the first two weeks. Only patients with stable symptoms at the end of the 'run in' period and reproducible 6-minute walk distance on the 6MWT during the baseline visit, as a marker of clinical stability of the disease will be randomized. They will be a portable oxygen cylinder during 2 weeks when they realize activities.
干预措施: Ambulatory Oxygen (Drug)
结局指标
主要结局
change in K-BILD The King's Brief Interstitial Lung Disease Questionnaire health status score
时间窗: February 2017 (up to 3 years)
The mean change vs baseline in the K-BILD health status score on and off ambulatory oxygen will be compared by using either a paired t-test or Wilcoxon signed rank test, as appropriate to the distribution of the data. To allow assessment of the effect of baseline values on treatment and any other potential confounders, regression with robust variances or linear mixed models will be used. STATA software will be used for statistical analysis.
次要结局
- Sense wear activity monitor measures of daily number of steps walked, daily expenditure and time spent at different levels of activity.(1 week)
- Continuous oxygen saturation for 48 hours at weekly intervals(48 hours)
- Quality of life as assessed by SGRQ and the hospital anxiety and depression score(2 weeks)
- Individual questions specifically related to activity from both the k-BILD and the SOBQ questionnaires(2 weeks)
- Dyspnoea scores(2 weeks)
- Global assessment of change in walking ability and exertional breathlessness as better, the same or worse.(2 weeks)
- Qualitative assessment of the impact of ambulatory oxygen(2 weeks of treatment)