Registry to Monitor the Susceptibility to Aztreonam of Pseudomonas Aeruginosa Isolates From Cystic Fibrosis Patients (AIR-CF5)
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This is a prospective, longitudinal, 5-year study that will enroll participants from the existing Cystic Fibrosis Foundation (CFF) patient registry. Each enrolled participant will provide samples for microbiological evaluation, obtained upon enrollment and then once per year thereafter for 5 years.
Proportion of participants whose least susceptible Pseudomonas aeruginosa (PA) isolate has a ≥ 4-fold increase in aztreonam minimum inhibitory concentration over 1 year and is above the parenteral breakpoint (> 8 μg/mL) [ Time Frame: Up to 5 years ]
This proportion will be compared annually over 5 years.
Secondary Outcome Measures
Annual mean change and mean change from baseline in FEV1 (liters) and FEV1 % predicted [ Time Frame: Baseline to Year 5 ]
Annual number of hospitalizations and the total number of hospitalizations at the end of each year [ Time Frame: Up to 5 years ]
Annual number of days hospitalized and the total number of hospitalization days at the end of each year [ Time Frame: Up to 5 years ]
Annual mean change and mean change from baseline in body mass index (BMI) [ Time Frame: Baseline to Year 5 ]
Annual number of Cayston treatment courses per participant and the total number of Cayston treatment courses at the end of each year in participants that used Cayston [ Time Frame: Up to 5 years ]
Biospecimen Retention: Samples Without DNA
Pseudomonas aeruginosa isolates from sputum or throat swab specimens
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Ages Eligible for Study:
6 Years and older (Child, Adult, Senior)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
A representative cross-section of individuals with mild (FEV1 75% to ≤ 90% predicted), moderate (FEV1 40% to 74% predicted), or severe (FEV1 <40% predicted) lung disease and a history of ≥ 2 lower respiratory tract cultures positive for PA (at any time) will be enrolled. This will include approximately 100 individuals with pre-study exposure to Cayston (defined as having received at least one prior 28-day course of Cayston at any time before enrolling into the study). Enrollment will be monitored and potentially modified to ensure that adequate numbers of participants in each disease severity category are included. At the Investigator's discretion, participants enrolled in this study can be allowed to participate in clinical trials with other investigational therapies as appropriate.
Key Inclusion Criteria:
Subjects must meet all of the following inclusion criteria to be eligible for participation in this study.
Current participant or willingness to participate in the CFF patient registry database
≥ 6 years of age
Subject has CF as diagnosed by one of the following
Documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test, or
Two well-characterized genetic mutations in the CFTR gene, or
Abnormal nasal potential difference (NPD) AND accompanying clinical characteristics consistent with CF. For subjects who lack documentation of either a positive sweat chloride test or an abnormal NPD, and who have only one well-characterized genetic mutation of the CFTR gene, the diagnosis of CF is determined by the Investigator.
FEV1 ≥ 25% predicted and ≤ 90% predicted.
≥ 2 lower respiratory tract cultures positive for PA with results documented in the subject's medical history.
Subject must be able to provide written informed consent/assent prior to any study related procedure; parent/guardian must be able to give written informed consent as necessary prior to any study related procedure.
Key Exclusion Criteria:
Subjects who meet the following exclusion criterion are not to be enrolled in this study.
• Any serious active medical or psychiatric illness that, in the opinion of the Investigator, would interfere with subject assessment.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.