Cystic Fibrosis
Conditions
Keywords
Cystic fibrosis, PA, Pseudomonas aeruginosa, CF, aztreonam
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.
Interventions
None listed
Sponsors
Cystic Fibrosis Foundation
Gilead Sciences
Study design
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion 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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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) | Up to 5 years | This proportion will be compared annually over 5 years. |
Secondary
| Measure | 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 |
Countries
United States
Outcome results
None listed