A Prospective Observational Study in Cystic Fibrosis Patients With Chronic Respiratory Pseudomonas Aeruginosa Infection Treated With TOBI® PODHALER® (Tobramycin Inhalation Powder) or Other FDA Approved Inhaled Antipseudomonal Antibacterial Drugs
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Mylan Inc.
- 入组人数
- 409
- 试验地点
- 1
- 主要终点
- Number of non-respiratory related hospitalizations.
研究概览
简要总结
This is a multicenter, prospective, two cohort, observational study over a 5-year period in Cystic Fibrosis (CF) patients with chronic Pseudomonas aeruginosa infection.The study will collect data over 1 year on respiratory function, antibacterial effectiveness, and clinical outcomes of treatment with inhaled antipseudomonal antibiotics and data over 5 years on microbiological and safety assessments.
详细描述
This study will include CF patients chronically colonized with P. aeruginosa enrolled in the Cystic Fibrosis Foundation (CFF) PortCF registry and using TOBI® PODHALER® or another FDA-approved inhaled antipseudomonal antibiotic. No therapeutic intervention will be assigned and physicians will use their discretion in choosing a treatment regimen for their patients.
Sputum samples (primarily collected during routine clinical follow-up) from patients able to spontaneously produce sputum will be sent to a central laboratory for analysis.
In addition, this study will include two optional sub-studies for qualifying patients in the first study year - Sputum microbiology sub-study and TOBI® PODHALER® sputum pharmacokinetics (PK) sub-study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •≥ 6 years of age.
- •Documented FEV1 ≥ 25% predicted in the previous year.
- •Diagnosis of cystic fibrosis.
- •Established diagnosis of chronic P. aeruginosa infection of the lungs defined as two or more positive P. aeruginosa cultures in the previous year as documented in the subject's medical history (this may include a history of one positive culture in the year prior to enrollment and one positive culture from the specimen collected at the baseline visit).
- •Prescribed and initiated chronic treatment with FDA-approved inhaled antipseudomonal antibiotic for chronic P. aeruginosa infection (e.g. TOBI® PODHALER®, TOBI®, Cayston® and Bethkis®).
- •Actively enrolled or willingness to enroll in PortCF registry.
- •Willing and able to provide written informed consent or, parent/guardian consent and where applicable pediatric assent, for participation and use of relevant clinical data previously captured in PortCF.
- •Anticipated to have good adherence to routine visits, defined as the investigator having good knowledge that the patient has been to at least 2-3 routine visits in the previous year.
排除标准
- •Documented FEV1 < 25% predicted in the previous year.
- •Current participation in an interventional clinical study with an inhaled antibiotic treatment.
- •Treatment with compounded tobramycin (e.g. the use of tobramycin IV solution adapted for use by inhalation).
- •Treatment with inhaled antipseudomonal antibacterial drug(s) that are not FDA approved.
- •Patients undergoing an early eradication regimen for CF (first line therapy).
研究组 & 干预措施
TOBI® PODHALER® cohort
干预措施: TOBI® PODHALER® (Drug)
non-TOBI® PODHALER® cohort
Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment
干预措施: TOBI® (Drug)
non-TOBI® PODHALER® cohort
Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment
干预措施: Bethkis® (Drug)
non-TOBI® PODHALER® cohort
Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment
干预措施: Cayston® (Drug)
结局指标
主要结局
Number of non-respiratory related hospitalizations.
时间窗: 1 year
Duration of stay for non-respiratory related hospitalizations.
时间窗: 1 year
Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum.
时间窗: 1 year
Minimum inhibitory concentration (MIC) of tobramycin and the following antipseudomonal antibacterial drugs (meropenem, imipenem, ceftazidime, aztreonam and ciprofloxacin) for P. aeruginosa sputum isolates in both treatment cohorts.
时间窗: Up to 5 years
Frequency of the following treatment emergent pathogens in sputum: S. aureus (MRSA and MSSA), S. maltophilia, A. xylosoxidans, and Burkholderia spp.in both treatment cohorts.
时间窗: Up to 5 years
Number of pulmonary exacerbations and those leading to hospitalization.
时间窗: 1 year
Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization.
时间窗: 1 year
Incidence rate of patients with one or more pulmonary exacerbations.
时间窗: 1 year
Incidence rate of pulmonary exacerbations.
时间窗: 1 year
Time to first pulmonary exacerbation.
时间窗: 1 year
Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations.
时间窗: 1 year
Mortality rate
时间窗: 1 year
Pharmacokinetic properties of TOBI® PODHALER® as measured by sputum specimens collected during the on-treatment cycles.
时间窗: 1 year
Number of respiratory related hospitalizations.
时间窗: 1 year
Duration of stay for respiratory related hospitalizations.
时间窗: 1 year
Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline.
时间窗: 1 year
次要结局
- Relative change in FEV1 % predicted from baseline.(1 year)