International Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: AZLI 75 mg three times a day (TID); Placebo three times a day (TID)
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 166.0
Sex: All
Minimum age: 6 Years
Healthy volunteers: No
Keywords: Cystic Fibrosis; Pseudomonas aeruginosa; Pulmonary Cystic Fibrosis

Summary:

      The purpose of this study was to evaluate the safety and efficacy of a 28-day course of
      aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung
      infection due to Pseudomonas aeruginosa (PA).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Documentation of CF diagnosis as evidenced by one or more clinical features consistent
             with the CF phenotype and one or more of the following criteria:

               -  Sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine
                  iontophoresis test (QPIT);

               -  Two well-characterized mutations in the cystic fibrosis transmembrane conductance
                  regulator (CFTR) gene; or

               -  Abnormal nasal potential difference.

          -  PA present in expectorated sputum or throat swab culture at Screening.

          -  FEV1 between (and including) 25% and 75% predicted at Screening.

          -  Negative pregnancy test at Screening.

          -  Ability to perform reproducible pulmonary function tests.

          -  Arterial oxygen saturation (SaO2) greater than or equal to 90% on room air at
             Screening.

          -  Ability to provide written informed consent.

        Exclusion Criteria:

          -  Administration of antipseudomonal antibiotics by inhalation, IV, or oral routes
             (including azithromycin) within 14 days of Screening.

          -  Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg
             prednisone/day or 20 mg prednisone every other day.

          -  History of sputum or throat swab culture yielding Burkholderia cepacia in the previous
             2 years.

          -  History of daily continuous oxygen supplementation or requirement for more than 2
             liters/minute at night.

          -  Administration of any investigational drug or use of any investigational device within
             28 days of Screening and within 6 half-lives of the investigational drug (whichever
             was longer).

          -  Known local or systemic hypersensitivity to monobactam antibiotics.

          -  Inability to tolerate short-acting bronchodilator use at least three times daily.

          -  Changes in protocol-permitted antimicrobial, bronchodilator, anti-inflammatory, or
             corticosteroid medications within 7 days prior to Screening or between Screening and
             the next visit.

          -  Changes in physiotherapy technique or schedule within 7 days prior to Screening or
             between Screening and the next visit.

          -  History of lung transplantation.

          -  A chest x-ray indicating abnormal findings at Screening or within the previous 90
             days.

          -  Abnormal renal or hepatic function at Screening.

          -  Any serious or active medical or psychiatric illness which, in the opinion of the
             investigator, would have interfered with participant treatment, assessment, or
             compliance with the protocol.

          -  Use of aerosolized hypertonic saline (except for sputum induction) during the 14 days
             preceding Visit 1.