A Phase 3, Double-Blind, Multicenter, Multinational, Randomized, Placebo-Controlled Trial Evaluating Aztreonam Lysinate for Inhalation in Cystic Fibrosis Patients With Pulmonary Pseudomonas Aeruginosa (AIR-CF1)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 166
- 试验地点
- 53
- 主要终点
- Change in CFQ-R Respiratory Symptoms Scale (RSS) Score
研究概览
简要总结
The purpose of this study was to evaluate the safety and efficacy of a 28-day course of aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).
详细描述
CF patients often have lung infections that occur repeatedly or worsen over time. The lung infections are often caused by a bacteria called Pseudomonas aeruginosa (PA). Treatment with antibiotics can stop or slow down the growth of the bacteria. The antibiotics may be given by mouth, intravenously (IV), or by inhalation as a mist. The purpose of this study was to evaluate the safety and efficacy of AZLI, an investigational formulation of the antibiotic aztreonam and administered TID using the PARI eFlow® electronic nebulizer, in CF patients with PA.
In this study, participant eligibility was assessed at a screening visit 7 to 14 days prior to the baseline visit (Day 0). Those participants who continued to meet eligibility criteria at Day 0 were randomized and began a 28-day course of blinded study treatment (AZLI TID or placebo TID). Participants returned for clinic visits at Day 14, an end of treatment visit at Day 28, and a follow-up visit 14 days after the last dose of study drug (Day 42).
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documentation of CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
- •Sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT);
- •Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene; or
- •Abnormal nasal potential difference.
- •PA present in expectorated sputum or throat swab culture at Screening.
- •FEV1 between (and including) 25% and 75% predicted at Screening.
- •Negative pregnancy test at Screening.
- •Ability to perform reproducible pulmonary function tests.
- •Arterial oxygen saturation (SaO2) greater than or equal to 90% on room air at Screening.
- •Ability to provide written informed consent.
排除标准
- •Administration of antipseudomonal antibiotics by inhalation, IV, or oral routes (including azithromycin) within 14 days of Screening.
- •Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg prednisone/day or 20 mg prednisone every other day.
- •History of sputum or throat swab culture yielding Burkholderia cepacia in the previous 2 years.
- •History of daily continuous oxygen supplementation or requirement for more than 2 liters/minute at night.
- •Administration of any investigational drug or use of any investigational device within 28 days of Screening and within 6 half-lives of the investigational drug (whichever was longer).
- •Known local or systemic hypersensitivity to monobactam antibiotics.
- •Inability to tolerate short-acting bronchodilator use at least three times daily.
- •Changes in protocol-permitted antimicrobial, bronchodilator, anti-inflammatory, or corticosteroid medications within 7 days prior to Screening or between Screening and the next visit.
- •Changes in physiotherapy technique or schedule within 7 days prior to Screening or between Screening and the next visit.
- •History of lung transplantation.
- •A chest x-ray indicating abnormal findings at Screening or within the previous 90 days.
- •Abnormal renal or hepatic function at Screening.
- •Any serious or active medical or psychiatric illness which, in the opinion of the investigator, would have interfered with participant treatment, assessment, or compliance with the protocol.
- •Use of aerosolized hypertonic saline (except for sputum induction) during the 14 days preceding Visit 1.
研究组 & 干预措施
Placebo three times a day (TID)
干预措施: Placebo three times a day (TID) (Drug)
AZLI 75 mg three times a day (TID)
干预措施: AZLI 75 mg three times a day (TID) (Drug)
结局指标
主要结局
Change in CFQ-R Respiratory Symptoms Scale (RSS) Score
时间窗: Day 0 to Day 28
The CFQ-R was administered at baseline and every visit thereafter. The endpoint was change in respiratory symptoms from baseline, assessed with the CFQ-R respiratory symptoms scale (RSS; range of scores: 0-100; higher scores indicate fewer symptoms).
次要结局
- Change in CFQ-R RSS Score(Day 0 to Day 42)
- Percent Change in FEV1 (L)(Day 0 to Day 28)
- Change From Baseline in Pseudomonas Aeruginosa (PA) Log10 Colony Forming Units (CFU) Per Gram of Sputum(Day 0 to Day 28)
- Number of Participants Receiving Intravenous (IV) or Inhaled Antipseudomonal Antibiotics Other Than Trial Drug(Day 0 to Day 42)
- Number of Participants Hospitalized at Least Once Between Day 0 and Day 42(Day 0 to Day 42)