A Randomized Controlled Trial on Hypertonic Saline Inhalation in Patients With Nodular-bronchiectatic Mycobacterium Avium Complex Pulmonary Disease
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Change in health-related quality of life
研究概览
简要总结
The SALINE trial will investigate the effect of Hypertonic Saline inhalation plus best supportive care on burden of symptoms, clearance of mycobacteria and functional capacities in participants with Mycobacterium avium complex lung disease and compare the effect to treatment with best supportive care alone.
详细描述
The treatment of Mycobacterium avium complex (MAC) lung disease consists of best supportive care, often accompanied by long-lasting multi-drug antibiotic regimens. Two major radiologic patterns exist: nodular-bronchiectatic and fibrocavitary disease, characterized by slow and rapid progression of disease, respectively.
SALINE is an open-label, randomized, two-arm controlled study that investigates the effect of Hypertonic Saline inhalation (HSi) plus best supportive care versus best supportive care alone for 12 weeks in participants with nodular-bronchiectatic MAC lung disease. The investigators hypothesize that HSi added to best supportive care will improve health-related quality of life and reduce mycobacterial load more than best supportive care alone Participants will be randomized 1:1 to a study arm. Best supportive care comprises of management of a predisposing (lung) condition, guidance in smoking cessation, respiratory physiotherapy (e.g. airway clearance) and nutritional guidance. HSi will be administered two times daily. Antibacterial therapy against other bacterial infections and inhaled corticosteroids are allowed during the study period.
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
- 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
- •International guideline criteria for nodular-bronchiectatic MAC lung disease, i.e. symptomatic, nodular bronchiectatic lesions seen on thorax radiography and ≥2 positive cultures of the same MAC species or one positive culture from a bronchoalveolar lavage;
- •≥1 positive MAC sputum cultures must be collected in the previous 4 months;
- •Signed and dated patient informed consent.
排除标准
- •Fibrocavitary MAC lung disease;
- •Antimycobacterial treatment in the last 6 months;
- •Previous MAC lung disease treatment failure, defined as persistent culture positivity despite >6 months of guideline-recommended treatment;
- •Current clinical relevant asthma or otherwise bronchial hyperresponsiveness that is judged to be a contra-indication for HSi.
- •Current HSi use
- •Former adverse reaction to HSi (note: former HSi use that was stopped due to a lack of clinical improvement is not an exclusion criterium);
- •Hypertonic saline intolerability during the screening test inhalation
- •Diagnosis of HIV;
- •Diagnosis of Cystic fibrosis (CF);
- •Active pulmonary malignancy (primary or metastatic) or any other malignancy requiring chemotherapy or radiotherapy within 6 months before screening or anticipated during the study period;
- •Active pulmonary tuberculosis, fungal or nocardial disease requiring treatment
- •Current use of chronic systemic corticosteroids at doses of 15 mg/day for more than 3 months
- •Prior lung or other solid organ transplant
- •Known or suspected current drug or alcohol abuse, that is, in the opinion of the Investigator, sufficient to compromise the safety or cooperation of the patient.
研究组 & 干预措施
Hypertonic Saline inhalation
Participants randomized to the Hypertonic Saline inhalation arm will be prescribed a nebulizer for Hypertonic Saline Inhalation (5ml, 5.8%) two times a day for 12 weeks. Participants will also receive best supportive care for 12 weeks (see below).
干预措施: Hypertonic Saline inhalation (Device)
Best supportive care
Participants randomized to the best supportive care arm will receive standard of care including management of predisposing (lung) disease, guidance in smoking cessation, respiratory physiotherapy (e.g. airway clearance), nutritional guidance, but no antimycobacterial treatment.
结局指标
主要结局
Change in health-related quality of life
时间窗: 12 weeks
Measured by the Quality of Life - Bronchiectasis (QOL-B) with NTM module questionnaire at baseline, after 4, 8 and 12 weeks. The QOL-B asks the participant to subjectively rank their symptoms using a 4 scale base ranging from "a lot of difficulty" to "no difficulty", "always" to "never", "completely true" to "not at all true" and "a lot" to "not at all" for 8 domains: physical/role/emotional/social functioning, vitality, treatment burden, health perception and respiratory symptoms. The NTM-module also asks participants to subjectively rank eating problems, body image, digestive symptoms, and NTM symptoms on a 4 scale base.
次要结局
- (Serious) Adverse Events as assessed by CTCAE v5.0(12 weeks)
- Change in physical function capacity(12 weeks)
- Therapy adherence(12 weeks)
- Sputum culture conversion(12 weeks)
- Treatment failure(12 weeks)
- Change in semi-quantitative culture results(12 weeks)
- Change in self-reported health status(12 weeks)
- Change in inflammatory serum biomarkers(12 weeks)
- Change in pulmonary function parameters(12 weeks)