An 8 Week Open-label Interventional Multicenter Study to Evaluate the Lung Clearance Index as Endpoint for Clinical Trials in Cystic Fibrosis Patients ≥ 6 Years of Age, Chronically Infected With Pseudomonas Aeruginosa
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- Change From Baseline in Lung Clearance Index (LCI) After 4 Weeks Following Onset of Study
研究概览
简要总结
The purpose of the study was to evaluate lung clearance index (LCI) by a standardized procedure in a well characterized study setting and to assess feasibility of LCI as a more sensitive method than forced expiratory volume at 1 second (FEV1) to measure effectiveness of antibiotic therapy in patients with CF aged 6 years and older with mild to moderate lung disease.
详细描述
This study was terminated prematurely. The reason for trial termination was challenge with enrollment and patient recruitment. A significant decrease in the eligible patient population had been identified as main driver.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
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
- •Confirmed diagnosis of CF
- •Patients with elevated LCI of ≥ 7.5 at screening
- •Patients with FEV1 of ≥ 50% predicted at screening
- •Use of inhaled Tobramycin in a 28 days on / off regimen in the past 3 months before screening
- •chronic lung Infection with Pseudomonas aeruginosa
排除标准
- •Patients who are regularly receiving more than one class of inhaled anti-pseudomonal antibiotic
- •Patients who have used oral or intravenous anti-pseudomonal antibiotics within 28 days prior to on-phase of study drug
- •Pregnant or nursing (lactating) women
- •Change in dose, formulation or strength of the study drug in the past treatment cycle before screening
- •History of hearing loss or chronic tinnitus
- •Infection with Burkholderia cenocepacia complex
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Tobramycin ALL
300mg nebulized Tobramycin (Tobramycin inhalation solution(TIS)) or TOBI Podhaler (Tobramycin inhalation powder(TIP), equivalent dry powder)twice a day (BID) 28days on / 28 days off
干预措施: TIS or TIP (Drug)
结局指标
主要结局
Change From Baseline in Lung Clearance Index (LCI) After 4 Weeks Following Onset of Study
时间窗: Baseline, week 4
The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.
次要结局
- Change From Baseline of Forced Expiratory Volume at 1 Second (FEV1) After 4 Weeks Following Onset of Study(Baseline, week 4)
- Change From Baseline of Colony-forming Units (CFU) After 4 Weeks Following Onset of Study(Baseline, week 4)
- Change From Baseline in Lung Clearance Index (LCI) After 1 Week(Baseline, week 1)
- Change of Lung Clearance Index (LCI) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)(week 4, week 8)
- Change of Forced Expiratory Volume at 1 Second(FEV1) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)(week 4, week 8)
- Change of Colony-forming Units (CFU) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)(week 4, week 8)