Registry to Monitor the Susceptibility to Aztreonam of Pseudomonas Aeruginosa Isolates From Cystic Fibrosis Patients

Study Overview
=================
Brief Summary
-----------------
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.

Official Title
-----------------
A Prospective, 5-year Registry Study to Monitor the Susceptibility to Aztreonam of Pseudomonas Aeruginosa (PA) Isolates From Patients With Cystic Fibrosis in the United States [AIR-CF5]

Conditions
-----------------
Cystic Fibrosis

Participation Criteria
=================
Eligibility Criteria
-----------------
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.

Ages Eligible for Study
-----------------
Minimum Age: 6 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------
What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) | This proportion will be compared annually over 5 years. | Up to 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annual mean change and mean change from baseline in FEV1 (liters) and FEV1 % predicted |  | Baseline to Year 5 | 
| Annual number of hospitalizations and the total number of hospitalizations at the end of each year |  | Up to 5 years | 
| Annual number of days hospitalized and the total number of hospitalization days at the end of each year |  | Up to 5 years | 
| Annual mean change and mean change from baseline in body mass index (BMI) |  | 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 |  | Up to 5 years | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Cystic fibrosis, PA, Pseudomonas aeruginosa, CF, aztreonam