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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]

Description

Pseudomonas aeruginosa (PA) is the most significant bacteria in patients with cystic fibrosis (CF). Once the PA is in the lungs it is very difficult to get rid of the bacteria. Therefore, CF patients must take medication either by oral or inhaled antibiotics for a very long time. The antibiotic called tobramycin inhalation solution (TOBI; TIS) has been on the market since 1997 and is widely used for management of CF in patients with PA;up to 69% of PA-positive CF patients were prescribed at least one course of TIS in 2008. In addition to TIS, IV tobramycin is also commonly used to treat acute lung infections of CF lung disease. Therefore, exposure in the CF population is extensive.A second aerosolized antibiotic is now available in the US. Cayston (aztreonam forinhalation solution; AZLI) is an inhaled monobactam antibacterial approved by the FDA in February 2010. It is indicated to improve respiratory symptoms in CF patients with PA. Undoubtedly, the introduction of a new inhaled antibiotic will change the profile of PA that is seen in CF patients with CF. The goal of this study is to track these changes over five years in a representational cohort of the US patient population. The main endpoint will be to evaluate the proportion of subjects who have sputum sample with PA. The secondary endpoints include:1) Annual change from baseline in lung function measured by the spirometry test. 2) The annual number of hospitalizations and the total number of hospitalizations at theend of each year3) The annual number of days hospitalized and the total number of hospitalization daysat the end of each year4) Annual change from the start of the study the patient's body weight change.5) Annual number of Cayston treatment courses per patient and the total number ofCayston treatment courses at the end of each year in patients that used CaystonNo formal hypotheses will be tested in this study. A sample size of 500 subjects was selected in order to provide a representative cross-section of subjects and to ensure that at least 300 subjects will have available data at the end of 5 years. Assuming the highest variability estimate of 0.25, the proportion of subjects whose least susceptible PA isolate has a 4-fold increase in aztreonam MIC over 1 year and is above the parenteral breakpoint (> 8 g/mL) can be estimated within the following ranges using a 95% confidence interval (CI).

Phase

N/A

Inclusion and Exclusion 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

  • Subjects who meet the following exclusion criterion are not to be enrolled in this study.

Sites

  • California

    • Children's Hospital of Los Angeles, Los Angeles, California, 90027
  • Nevada

    • Children's Lung Specialists, Las Vegas, Nevada, 89107
  • Utah

    • University of Utah, Salt Lake City, Utah, 84132
  • Colorado

    • National Jewish Health, Denver, Colorado, 80206
    • The Children's Hospital, Denver, Colorado, 80045
  • Arkansas

    • Arkansas Children's Hospital Research Institute, Little Rock, Arkansas, 72202
  • Minnesota

    • Children's Hospitals & Clinics of Minnesota, Minneapolis, Minnesota, 55404
  • Wisconsin

    • Children's Hospital of Wisconsin, Milwaukee, Wisconsin, 53201
  • Indiana

    • Riley Hospital for Children, Indianapolis, Indiana, 46202
  • Ohio

    • University of Cincinnati Medical Center, Cincinnati, Ohio, 45267
    • Cincinati Children's Hosptial Medical Center, Cincinnati, Ohio, 45229
    • The Children's Medical Center of Dayton, Dayton, Ohio, 45404
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