Observational Study in Cystic Fibrosis Patients Using TOBI® PODHALER® or Other FDA Approved Inhaled Antipseudomonal Antibacterial Drugs
- Conditions
- Pseudomonas Aeruginosa in Cystic Fibrosis
- Interventions
- Registration Number
- NCT02449031
- Lead Sponsor
- Mylan Inc.
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 409
- ≥ 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.
- 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).
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description TOBI® PODHALER® cohort TOBI® PODHALER® - non-TOBI® PODHALER® cohort TOBI® Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment non-TOBI® PODHALER® cohort Bethkis® Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment non-TOBI® PODHALER® cohort Cayston® Approximately 250 patients treated with other FDA-approved inhaled antipseudomonal antibacterial drugs at enrollment
- Primary Outcome Measures
Name Time Method Number of non-respiratory related hospitalizations. 1 year Duration of stay for non-respiratory related hospitalizations. 1 year 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. Up to 5 years Number of pulmonary exacerbations and those leading to hospitalization. 1 year Absolute change from baseline in the number of P. aeruginosa colony forming units in sputum. 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. Up to 5 years Proportion of patients experiencing pulmonary exacerbations including those leading to hospitalization. 1 year Incidence rate of patients with one or more pulmonary exacerbations. 1 year Incidence rate of pulmonary exacerbations. 1 year Time to first pulmonary exacerbation. 1 year Use of additional antipseudomonal antibiotics (overall, IV, oral) to treat pulmonary exacerbations. 1 year Mortality rate 1 year Pharmacokinetic properties of TOBI® PODHALER® as measured by sputum specimens collected during the on-treatment cycles. 1 year Number of respiratory related hospitalizations. 1 year Duration of stay for respiratory related hospitalizations. 1 year Absolute change in forced expiratory volume in one second (FEV1) percent predicted from baseline. 1 year
- Secondary Outcome Measures
Name Time Method Relative change in FEV1 % predicted from baseline. 1 year
Trial Locations
- Locations (1)
Novartis Investigative Site
🇺🇸Spokane, Washington, United States