AMBER: Azithromycin to Modify Bronchiectasis Exacerbation Risk - A Double-Blind Placebo-Controlled Randomized Trial in Adults With Non-Cystic Fibrosis Bronchiectasis
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 500
- 试验地点
- 1
研究概览
简要总结
The Azithromycin to Modify Bronchiectasis Exacerbation Risk (AMBER) trial is a prospective, randomized, double-blind, placebo-controlled, parallel-group clinical trial in adults with clinically and radiologically confirmed non-cystic fibrosis bronchiectasis (NCFB).
The trial evaluates whether azithromycin 250 mg orally once daily for 12 months, added to standard bronchiectasis care, reduces the occurrence of at least one bronchiectasis exacerbation during 12-month follow-up compared with matching placebo added to standard bronchiectasis care.
Participants will be randomized in a 1:1 allocation ratio to standard care plus matching placebo or standard care plus azithromycin. The primary analysis will follow the intention-to-treat (ITT) principle.
The AMBER trial is embedded within the Assiut University bronchiectasis translational research platform and is linked to the Bronchiectasis Assessment of Severity and Exacerbations (BASE) framework and the Bronchiectasis Phenotype Identification Model (BPIM). BASE and BPIM are not used for randomization stratification and will not modify the primary randomized comparison.
The locked Version 1.0 methodological disclosure document, protocol, and statistical analysis plan (SAP), primary sample-size source code, and endpoint-level sample-size support matrix are archived in Zenodo: https://doi.org/10.5281/zenodo.20178963.
The AMBER public preregistration is also available through the Open Science Framework (OSF) under Digital Object Identifier (DOI) 10.17605/OSF.IO/RE54V.
详细描述
The Azithromycin to Modify Bronchiectasis Exacerbation Risk (AMBER) trial is a prospective, randomized, double-blind, placebo-controlled, parallel-group superiority clinical trial conducted within the Assiut University bronchiectasis translational research platform.
The trial enrolls adults with clinically and radiologically confirmed non-cystic fibrosis bronchiectasis (NCFB). Participants will be randomized at the individual-patient level in a 1:1 allocation ratio to one of two treatment groups:
- Standard care plus matching placebo once daily for 12 months.
- Standard care plus azithromycin 250 mg orally once daily for 12 months.
Standard bronchiectasis care may include mucolytics, bronchodilators, chest physiotherapy, patient education, airway-clearance support, and treatment of acute exacerbations with antibiotics with or without systemic corticosteroids according to clinical indication.
The primary objective is to determine whether azithromycin added to standard care reduces the occurrence of at least one bronchiectasis exacerbation during 12-month follow-up compared with matching placebo added to standard care.
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)
盲法说明
AMBER is a double-blind, placebo-controlled randomized trial. Participants, care providers, investigators, and outcomes assessors will remain unaware of participant-level treatment assignment until database lock unless emergency unblinding is required for participant safety. Matching placebo will be prepared to resemble active azithromycin as closely as feasible in appearance, packaging, and administration schedule. Study medication will be dispensed using concealed allocation and sequentially numbered containers.
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
- •Adult patients aged 18 years or older.
- •Clinically and radiologically confirmed non-cystic fibrosis bronchiectasis (NCFB).
- •Diagnosis supported by clinical assessment and high-resolution computed tomography (HRCT).
- •Patients attending outpatient clinics, inpatient wards, respiratory follow-up services, or routine bronchiectasis care pathways at Assiut University Hospitals during the enrollment period.
- •Suitable for baseline disease-signature assessment.
- •Able to undergo protocol-defined clinical, functional, radiological, oxygenation, inflammatory, symptom, and safety assessment.
- •Able to complete planned 12-month follow-up.
- •Eligible for randomization after baseline safety evaluation.
- •Written informed consent obtained from the participant or legal representative.
排除标准
- •Cystic fibrosis-related bronchiectasis.
- •Traction bronchiectasis due to advanced fibrotic interstitial lung disease as the dominant respiratory diagnosis.
- •Active pulmonary tuberculosis at enrollment.
- •Active nontuberculous mycobacterial pulmonary disease requiring specific treatment at enrollment.
- •Active malignancy or terminal non-respiratory illness expected to prevent planned follow-up.
- •Acute life-threatening illness preventing safe enrollment, baseline assessment, or randomization.
- •Recent major thoracic surgery or acute thoracic trauma interfering with baseline respiratory assessment.
- •Known hypersensitivity, contraindication, or serious intolerance to azithromycin or macrolide therapy.
- •Baseline cardiac findings judged by the investigator to make long-term azithromycin unsafe.
- •Requirement for long-term maintenance macrolide therapy at enrollment.
- •Current long-term maintenance macrolide use that cannot be safely discontinued before enrollment.
- •Inability to complete required baseline disease-signature assessment.
- •Inability or unwillingness to complete planned 12-month follow-up.
- •Refusal to participate.
研究者
Ahmad Shaddad
Associate Professor
Assiut University