A Phase 2b, Multicenter, Randomized, Double-blind, Parallel-group, Placebo-controlled, Dose Range Finding Study to Evaluate the Efficacy, Safety, and Tolerability of Nebulized CSL787 in Adults (18 to 85 Years) With Non-cystic Fibrosis Bronchiectasis
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- CSL Behring
- 入组人数
- 450
- 试验地点
- 13
- 主要终点
- Time to first (TTF) Exacerbation
研究概览
简要总结
This study is a phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group, dose range finding study designed to explore the efficacy, safety, and tolerability of 2 active treatment regimens of CSL787 (immunoglobulin G [IgG] inhalation solution) compared with placebo over a period of 6 to 12 months independent of the occurrence of pulmonary exacerbations.
The primary aim of the study is to characterize the overall effect of CSL787 as well as the dose response of 2 active treatment regimens of inhaled CSL787 administered to participants with NCFB toward prolonging the TTF exacerbation.
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
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult between the ages of 18 to 85 years
- •Primary diagnosis of NCFB confirmed by chest computed tomography (CT) scan, where bronchiectasis has been documented by a radiologist. Diagnosis in the medical records based on historical scans is acceptable if the chest CT scan confirming the participant's NCFB diagnosis was performed within 12 months before enrollment. Participants for whom no chest CT scan results are available within the previous 12 months will undergo a chest CT scan during the Screening Period
- •Exacerbation history within the previous 1 year defined as either 1 of the following:
- •>= 2 documented exacerbations requiring oral and/or intravenous (IV) antibiotic therapy to treat a pulmonary infection.
- •1 documented exacerbation requiring oral and/or IV antibiotic therapy to treat a pulmonary infection and a St. George's Respiratory Questionnaire (SGRQ) Symptoms score of > 40 at Screening.
- •Note: Other medications to treat NCFB such as: oral macrolides, or dipeptidyl peptidase-1 (DPP-1) inhibitors are allowed, provided >= 1 historical exacerbation occurred while on the medication for >= 3 months at a stable dose.
- •Postbronchodilator percentage of the predicted normal forced expiratory volume in 1 second of expiration [FEV1% predicted] > 35% and forced expiratory volume in 1 second (FEV1) >= 1 liter (L) obtained in accordance with American Thoracic Society (ATS) / European Respiratory Society (ERS) standards for spirometry during Screening and at Baseline.
排除标准
- •History of bronchospasm in response to inhaled therapies including inhaled antibiotics
- •Known or suspected hypersensitivity, or other severe reactions, to the investigational product (IP), to any excipients of the IP, or to other immunoglobulin.
- •Primary diagnosis of other pulmonary disorders, including chronic obstructive pulmonary disease (COPD) asthma or, diffuse panbronchiolitis (DPB), as determined by the investigator.
- •Pulmonary exacerbation requiring antibiotic therapy within the 4 weeks before Baseline.
研究组 & 干预措施
Placebo
Participants in this arm will receive placebo.
干预措施: Placebo (Drug)
Placebo
Participants in this arm will receive placebo.
干预措施: Nebulizer (Device)
CSL787 High Dose
Participants in this arm will receive a high dose of CSL787.
干预措施: CSL787 (Biological)
CSL787 High Dose
Participants in this arm will receive a high dose of CSL787.
干预措施: Nebulizer (Device)
CSL787 Low Dose
Participants in this arm will receive a low dose of CSL787.
干预措施: CSL787 (Biological)
CSL787 Low Dose
Participants in this arm will receive a low dose of CSL787.
干预措施: Nebulizer (Device)
结局指标
主要结局
Time to first (TTF) Exacerbation
时间窗: Up to Month 12
TTF exacerbation, where an exacerbation is defined as a deterioration in \>= 3 of the following symptoms for \>= 48 hours: cough; sputum volume and / or consistency; sputum purulence; breathlessness and / or exercise tolerance; fatigue and / or malaise; hemoptysis AND a clinician determines that antibiotic therapy is required.
次要结局
- Annualized Exacerbation Rate (AER) (Exacerbation Event Rate Per-participant Year)(Up to Month 12)
- Number of Participants Achieving a Clinically Important Difference in the Quality of Life-Bronchiectasis (QoL-B) Respiratory Symptoms Scale(Up to Month 12)
- Percentage of Participants Achieving a Clinically Important Difference in the QoL-B Respiratory Symptoms Scale(Up to Month 12)
- Change From Baseline in QoL-B Respiratory Symptoms Scale(From Baseline to Months 6 and 12)
- Change From Baseline in Total Colony-forming Unit (CFUs) for Pathogenic Bacteria Isolated from Sputum(From Baseline to Month 1)
- Number of Participants with Treatment-emergent Adverse events (TEAEs) and Serious Adverse Events (SAEs)(Up to Month 13)
- Percentage of Participants with TEAEs and SAEs(Up to Month 13)