Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections
Condition: Pseudomonas Aeruginosa Infection
MeSH terms: Infections; Communicable Diseases; Pseudomonas Infections; Cystic Fibrosis
Intervention: Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer.; Tobramycin inhalation solution using a PARI LC® Plus nebulizer.
Intervention description: Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
Administration time is approximately 13 minutes.
liposomal amikacin for inhalation will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment.; 300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
Nebulization time is approximately 20 minutes for each administration.
Tobramycin inhalation solution will be administered for 3 cycles where each cycle consists of 28 days on-treatment followed by 28 days off-treatment
Intervention MeSH terms: Tobramycin; Amikacin
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 302.0
Sex: All
Minimum age: 6 Years
Healthy volunteers: No
Keywords: Cystic Fibrosis; Respiratory Infections; Pulmonary Cystic Fibrosis; CFTR; Amikacin; Anti-bacterial Agents; Amikacin liposome inhalation suspension (ALIS)

Summary:

      A major factor in the respiratory health of Cystic Fibrosis (CF) participants is the
      prevalence of chronic Pseudomonas aeruginosa (Pa) infections. The Pa infection rate in CF
      patients increases with age and by age 18 years approximately 85% of CF patients in the US
      are infected. Liposomal amikacin for inhalation (Arikayce™) was developed as a possible
      treatment for chronic infection due to Pa in CF patients.

      The purpose of this study is to determine whether Arikayce™ is effective in treating chronic
      lung infections caused by Pa in CF participants. The effectiveness, safety, and tolerability
      of Arikayce™ will be compared to Tobramycin TOBI®, an inhalation antibiotic already available
      for use.
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Written informed consent or assent

          -  Confirmed diagnosis of CF

          -  History of chronic infection with Pseudomonas aeruginosa

          -  Sputum culture positive for Pseudomonas aeruginosa at Screening

          -  FEV1 ≥ 25% of predicted value at Screening

        Key Exclusion Criteria:

          -  FEV1 <25% of predicted at Screening

          -  History of major complications of lung disease within 8 weeks prior to Screening

          -  Hemoptysis of ≥60 mL in a 24-hour period within 4 weeks prior to Screening

          -  History of positive culture for Burkholderia cepacia within 2 years prior to Screening

          -  History of pulmonary tuberculosis or non-tuberculous mycobacterial lung disease
             treated within 2 years prior to Screening or requiring treatment at the time of
             screening

          -  History of Allergic Broncho-Pulmonary Aspergillosis or any other condition requiring
             systemic steroids at a dose ≥ equivalent of 10 mg/day of prednisone within 3 months
             prior to Screening

          -  Presence of any clinically significant cardiac disease

          -  History of lung transplantation

          -  Daily, continuous oxygen supplementation or nighttime supplemental oxygen requirement
             of greater than 2 L/min

          -  Administration of any investigational products within 8 weeks prior to study Day 1

          -  Smoking tobacco or any substance within 6 months prior to screening or anticipated
             inability to refrain from smoking throughout the study