Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01315678
• Date of registration: 14/03/2011
• Prospective Registration: Yes
• Primary sponsor: Insmed Incorporated
• Public title: Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections
• Scientific title: Randomized, Open-Label, Active-Controlled, Multicenter Study to Assess the Efficacy, Safety and Tolerability of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
• Date of first enrolment: February 29, 2012
• Target sample size: 302
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01315678
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Austria, Belgium, Bulgaria, Canada, Denmark, France, Germany, Greece, Hungary, Ireland, Italy, Netherlands, Poland, Serbia, Slovakia, Spain, Sweden, United Kingdom

Contacts

• Name: Gina Eagle, MD
• Affiliation: Insmed Incorporated

Key inclusion & exclusion 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



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pseudomonas Aeruginosa Infection

Intervention(s)

Drug: Tobramycin inhalation solution using a PARI LC® Plus nebulizer.

Drug: Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer.

Primary Outcome(s)

Pulmonary Function Test: Forced Expiratory Volume in 1 Second (FEV1) [Time Frame: Baseline to168 days]

Secondary Outcome(s)

Number of Participants to First All Cause Hospitalization [Time Frame: 168 days]

Relative Percent (%) Change in Respiratory Symptoms as Measured by the CFQ-R [Time Frame: Day 14, Day 28, Day 57, Day 84, Day 113, Day 140 and Day 168]

Pumonary Function Test: Forced Expiratory Volume in 1 Second (FEV1) [Time Frame: Baseline, Day 14, Day 28, Day 57, Day 84, Day 113, Day 140 and Day 168.]

Number of Participants to First Antipseudomonal Antibiotic Treatment for Pulmonary Exacerbation [Time Frame: 168 days]

Number of Participants Experiencing a Pulmonary Exacerbation [Time Frame: 168 days]

Change in Density (Log CFU) in Pseudomonas Aeruginosa in Sputum [Time Frame: Baseline, Day 14, Day 28, Day 57, Day 84, Day 113, Day 140 and Day 168]

Secondary ID(s)

TR02-108

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 24/06/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01315678