Efficacy and Safety of 28 or 56 Day Treatment for Pseudomonas Aeruginosa in Children With Cystic Fibrosis
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Tobramycin solution for inhalation 300 mg
Intervention description: Tobramycin solution for inhalation was supplied in 5 mL liquid-filled low-density polyethylene ampoules containing 300 mg tobramycin. Patients used a nebulizer to inhale the contents of the ampoules.
Intervention MeSH terms: Tobramycin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 123.0
Sex: All
Minimum age: 6 Months
Healthy volunteers: No
Keywords: Cystic fibrosis

Summary:

      This study assessed time to recurrence of infection with Pseudomonas aeruginosa following
      treatment of the initial infection with tobramycin nebuliser solution. The safety profile of
      the initial tobramycin treatment was assessed during the first 3 months of the study and
      patients were followed until the end of the study, month 27.
    

Eligibility criteria:

        Inclusion criteria:

          -  Male or female patients ≥ 6 months old

          -  Diagnosis of cystic fibrosis (CF) based upon the following historical criteria
             performed prior to study participation:

               1. confirmed sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis (at
                  least 2 tests), OR

               2. genotype with two identifiable mutations consistent with CF.

          -  First or early lower respiratory tract infection with Pseudomonas (P.) aeruginosa
             documented by either of the following:

               1. first infection defined by the first P. aeruginosa isolated from sputum or deep
                  throat cough swab culture, OR

               2. P. aeruginosa from sputum or deep throat cough swab culture following at least 1
                  year of negative cultures (documented with at least 4 negative cultures during
                  this year and no positive cultures) and no anti-pseudomonal treatment during this
                  1-year period, OR

               3. P. aeruginosa from sputum or deep throat cough swab culture following at least 2
                  years of negative cultures (documented with at least 2 negative cultures per year
                  and no positive cultures) and no anti-pseudomonal treatment during this 2-year
                  period.

          -  Written informed consent by the patient and/or parent/legal guardian according to
             local country regulations.

        Exclusion criteria:

          -  History of aminoglycoside hypersensitivity or adverse reaction to inhaled
             aminoglycoside.

          -  Signs and symptoms of acute pulmonary disease, eg, pneumonia, pneumothorax.

          -  Administration of any investigational drug within 30 days prior to enrollment.

          -  Administration of loop diuretics within 7 days prior to study drug administration.

          -  Personal/family history of abnormal hearing, other than typical hearing loss
             associated with the aging process.

          -  Abnormal result from an audiology testing (defined as either a unilateral pure-tone
             audiometry test showing a threshold elevation > 20 decibels [dB] at any frequency
             across the frequency range 0.25-8 kHz or the absence of emission at the evoked
             otoacoustic emission test).

          -  Positive urine pregnancy test at Day 1 (Baseline) for all female patients who have
             reached menarche.

          -  Use of macrolide antibiotics as a maintenance therapy for 12 or more days during the
             28 days prior to Baseline.

          -  Antibody titers ≥ 1000 for any of the 3 P. aeruginosa exoenzymes: Exotoxin A, alkaline
             protease, or elastase (status to be determined between Baseline and Day 28).