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

Completed
Conditions
Cystic Fibrosis
Registration Number
NCT01375036
Lead Sponsor
Gilead Sciences
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
510
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.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
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 Outcome Measures
NameTimeMethod
Annual mean change and mean change from baseline in body mass index (BMI)Baseline to Year 5
Annual number of hospitalizations and the total number of hospitalizations at the end of each yearUp to 5 years
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 CaystonUp to 5 years
Annual mean change and mean change from baseline in FEV1 (liters) and FEV1 % predictedBaseline to Year 5
Annual number of days hospitalized and the total number of hospitalization days at the end of each yearUp to 5 years

Trial Locations

Locations (30)

Providence Alaska Medical Center

🇺🇸

Anchorage, Alaska, United States

University of Arizona

🇺🇸

Tucson, Arizona, United States

Arkansas Children's Hospital Research Institute

🇺🇸

Little Rock, Arkansas, United States

Children's Hospital of Los Angeles

🇺🇸

Los Angeles, California, United States

University of Southern California

🇺🇸

Los Angeles, California, United States

The Children's Hospital

🇺🇸

Denver, Colorado, United States

National Jewish Health

🇺🇸

Denver, Colorado, United States

Central Florida Pulmonary Group

🇺🇸

Altamonte, Florida, United States

Nemours Children's Clinic

🇺🇸

Orlando, Florida, United States

University of Chicago

🇺🇸

Chicago, Illinois, United States

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Providence Alaska Medical Center
🇺🇸Anchorage, Alaska, United States

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