一项 2×2 析因、随机、开放标签临床试验，旨在确定高渗盐水（HTS 6%）和羧甲司坦用于气道清除相对于常规护理在 52 周内对支气管扩张症成人的临床和成本效益：CLEAR 临床试验的方案。
A 2 × 2 factorial, randomised, open-label trial to determine the clinical and cost-effectiveness of hypertonic saline (HTS 6%) and carbocisteine for airway clearance versus usual care over 52 weeks in adults with bronchiectasis: a protocol for the CLEAR clinical trial.
机构信息
Wellcome-Wolfson Institute For Experimental Medicine, School of Medicine, Dentistry and Biomedical Sciences, Queen's University Belfast, Belfast, UK.
Centre for Health and Rehabilitation Technologies (CHaRT), Institute of Nursing and Health Research, Ulster University, Newtownabbey, UK.
出版信息
Trials. 2019 Dec 19;20(1):747. doi: 10.1186/s13063-019-3766-9.
BACKGROUND
Current guidelines for the management of bronchiectasis (BE) highlight the lack of evidence to recommend mucoactive agents, such as hypertonic saline (HTS) and carbocisteine, to aid sputum removal as part of standard care. We hypothesise that mucoactive agents (HTS or carbocisteine, or a combination) are effective in reducing exacerbations over a 52-week period, compared to usual care.
METHODS
This is a 52-week, 2 × 2 factorial, randomized, open-label trial to determine the clinical effectiveness and cost effectiveness of HTS 6% and carbocisteine for airway clearance versus usual care - the Clinical and cost-effectiveness of hypertonic saline (HTS 6%) and carbocisteine for airway clearance versus usual care (CLEAR) trial. Patients will be randomised to (1) standard care and twice-daily nebulised HTS (6%), (2) standard care and carbocisteine (750 mg three times per day until visit 3, reducing to 750 mg twice per day), (3) standard care and combination of twice-daily nebulised HTS and carbocisteine, or (4) standard care. The primary outcome is the mean number of exacerbations over 52 weeks. Key inclusion criteria are as follows: adults with a diagnosis of BE on computed tomography, BE as the primary respiratory diagnosis, and two or more pulmonary exacerbations in the last year requiring antibiotics and production of daily sputum.
DISCUSSION
This trial's pragmatic research design avoids the significant costs associated with double-blind trials whilst optimising rigour in other areas of trial delivery. The CLEAR trial will provide evidence as to whether HTS, carbocisteine or both are effective and cost effective for patients with BE.
TRIAL REGISTRATION
EudraCT number: 2017-000664-14 (first entered in the database on 20 October 2017). ISRCTN.com, ISRCTN89040295. Registered on 6 July/2018. Funder: National Institute for Health Research, Health Technology Assessment Programme (15/100/01).
SPONSOR
Belfast Health and Social Care Trust. Ethics Reference Number: 17/NE/0339. Protocol version: v3.0 Final_14052018.
背景
目前支气管扩张症（BE）管理指南强调缺乏证据推荐粘液溶解剂，如高渗盐水（HTS）和羧甲司坦，以帮助清除痰作为标准治疗的一部分。我们假设粘液溶解剂（HTS 或羧甲司坦，或两者的组合）在 52 周内比常规治疗更能有效减少恶化。
方法
这是一项为期 52 周的 2×2 析因、随机、开放标签试验，旨在确定 HTS 6%和羧甲司坦用于气道清除与常规治疗相比的临床效果和成本效果——高渗盐水（HTS 6%）和羧甲司坦用于气道清除与常规治疗的临床和成本效果（CLEAR）试验。患者将被随机分配到以下 4 组之一：（1）标准治疗和每日两次雾化 HTS（6%）；（2）标准治疗和羧甲司坦（750mg 每日三次，直到第 3 次就诊，然后减少到每日两次 750mg）；（3）标准治疗和每日两次雾化 HTS 和羧甲司坦联合治疗；或（4）标准治疗。主要结局是 52 周内平均恶化次数。主要纳入标准如下：成人 CT 诊断为 BE、BE 为主要呼吸系统诊断、过去一年中因肺炎恶化需要抗生素治疗和每日产生痰。
讨论
该试验的实用研究设计避免了与双盲试验相关的巨大成本，同时优化了试验实施的其他领域的严谨性。CLEAR 试验将提供证据，证明 HTS、羧甲司坦或两者对 BE 患者是否有效且具有成本效益。
试验注册
EudraCT 编号：2017-000664-14（于 2017 年 10 月 20 日首次在数据库中输入）。ISRCTN.com，ISRCTN89040295。于 2018 年 7 月 6 日注册。资助者：英国国家卫生研究院，卫生技术评估计划（15/100/01）。
主办方
贝尔法斯特健康和社会保健信托。伦理参考号：17/NE/0339。方案版本：v3.0 最终版_14052018。
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