Study of Aztreonam for Inhalation in Children With Cystic Fibrosis and New Infection of the Airways by Pseudomonas Aeruginosa Bacteria
Condition: Pseudomonas Aeruginosa Respiratory Tract Infection/Colonization; Cystic Fibrosis
MeSH terms: Infections; Communicable Diseases; Respiratory Tract Infections; Pseudomonas Infections; Cystic Fibrosis; Fibrosis
Intervention: AZLI; Placebo
Intervention description: Administered via the PARI Altera® Nebulizer System. Participants < 2 years will receive via the SmartMask® Baby, 2 to < 6 years via the SmartMask Kids® and > 6 years via the nebulizer mouthpiece.; Administered via the PARI Altera® Nebulizer System. Participants < 2 years will receive via the SmartMask® Baby, 2 to < 6 years via the SmartMask Kids® and > 6 years via the nebulizer mouthpiece.
Intervention MeSH terms: Aztreonam
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 149.0
Sex: All
Minimum age: 3 Months
Maximum age: 18 Years
Healthy volunteers: No

Summary:

      The primary objective of this study is to evaluate the safety and efficacy of a 14-day course
      versus a 28-day course of aztreonam for inhalation solution (AZLI) in pediatric participants
      with new onset Pseudomonas aeruginosa respiratory tract infection or colonization.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Diagnosis of cystic fibrosis (CF) as determined by the 2008 CF Consensus Conference
             criteria: Sweat chloride level ≥ 60 milliequivalents per liter (mEq/L) by quantitative
             pilocarpine iontophoresis; or a genotype with 2 identifiable mutations consistent with
             CF; or an abnormal nasal transepithelial potential difference (NPD), and 1 or more
             clinical features consistent with CF

          -  Documented new onset of positive respiratory tract culture for PA within 30 days of
             screening defined as either first lifetime documented PA-positive culture, or PA
             recovered after at least a 2-year history of PA-negative respiratory cultures (at
             least 2 cultures per year)

          -  Forced expiratory volume in one second (FEV1) ≥ 80% predicted (for subjects ≥ 6 years
             of age who can reliably perform spirometry assessments)

          -  Clinically stable with no evidence of acute significant respiratory symptoms that
             would require administration of intravenous (IV) antipseudomonal antibiotics, oxygen
             supplementation, or hospitalization

        Key Exclusion Criteria:

          -  Use of IV or inhaled antipseudomonal antibiotics within 2 years of screening

          -  Use of oral antipseudomonal antibiotics for a respiratory event within 30 days of
             study entry (screening visit)

          -  History of intolerance to inhaled short acting β2 agonists

          -  History of lung transplantation

          -  Current requirement for daily continuous oxygen supplementation or requirement of more
             than 2 L/minute at night

          -  Hospitalization for a respiratory event within 30 days prior to screening

          -  Changes in bronchodilator, corticosteroid, dornase alfa, or hypertonic saline
             medications within 7 days prior to screening.

          -  Significant changes (per investigators discretion) in physiotherapy technique or
             schedule within 7 days prior to screening

          -  Abnormal renal or hepatic function results at most recent test within the previous 12
             months, defined as Aspartate aminotransferase (AST) or alanine aminotransferase (ALT)
             > 5 times upper limit of normal (ULN), or Serum creatinine > 2 times ULN for age

          -  Presence of a condition or abnormality that would compromise the subject's safety or
             the quality of the study data, in the opinion of the Investigator

          -  Known hypersensitivity to aztreonam, its metabolites, or formulation excipients in
             AZLI

          -  Respiratory cultures performed within 24 months prior to screening that are positive
             for ANY Burkholderia spp. or Non-tuberculous mycobacteria (NTM)

        Note: Other protocol defined Inclusion/Exclusion criteria may apply