Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections
A major factor in the respiratory health of Cystic Fibrosis (CF) subjects is the prevalence of chronic Pseudomonas aeruginosa infections. The Pseudomonas aeruginosa 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 Pseudomonas aeruginosa in CF patients.
The purpose of this European Registration study is to determine whether Arikayce™ is effective in treating chronic lung infections caused by Pseudomonas aeruginosa in Cystic Fibrosis subjects. The effectiveness, safety and tolerability of Arikayce™ will be compared to TOBI, an inhalation antibiotic already available for use. The study will enroll approximately 300 subjects in clinics in Europe and Canada. Subjects will be required to visit the clinic 9 times (including the Screening visit) over a period of approximately 6 months. No overnight stays at the clinic will be required.
|Pseudomonas Aeruginosa Infection||Drug: Liposomal amikacin for inhalation (Arikayce) using the PARI Investigational eFlow® Nebulizer. Drug: Tobramycin inhalation solution using a PARI LC® Plus nebulizer.||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official 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|
- Relative change in Forced Expiratory Volume in 1 second (FEV1) from baseline to end of study (Day 168) [ Time Frame: 168 days ]
- Changes in pulmonary function throughout the study [ Time Frame: 168 days ]
- Time to and proportion of subjects experiencing a pulmonary exacerbation [ Time Frame: 168 days ]
- Time to first antipseudomonal antibiotic treatment for pulmonary exacerbation [ Time Frame: 168 days ]
- Time to and number of hospitalizations [ Time Frame: 168 days ]
- Change in density in Pseudomonas aeruginosa in sputum [ Time Frame: 168 days ]
- Change in patient reported outcomes/symptoms [ Time Frame: 168 days ]
- Evaluation of safety and tolerability [ Time Frame: 168 days ]
|Study Start Date:||March 2012|
|Study Completion Date:||August 2013|
|Primary Completion Date:||June 2013 (Final data collection date for primary outcome measure)|
Arikayce™ is liposomal amikacin for inhalation
Drug: Liposomal amikacin for inhalation (Arikayce) using the PARI Investigational eFlow® Nebulizer.
Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
Active Comparator: TOBI®
TOBI® is tobramycin inhalation solution
Drug: Tobramycin inhalation solution using a PARI LC® Plus nebulizer.
300 mg tobramycin inhalation solution is administered twice a day using a PARI LC® Plus nebulizer.
Cystic Fibrosis (CF) is a genetic disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients with CF manifest pathological changes in a variety of organs that express CFTR. The lungs are frequently affected often resulting in chronic infections by bacteria such as Pseudomonas aeruginosa and airway inflammation. Treatment of chronic lung infections is one of the principal goals of CF therapy. Arikayce™ (liposomal amikacin for inhalation) is a sustained-release formulation of amikacin encapsulated inside nanoscale liposomal carriers designed for administration via inhalation. It is hypothesized that the sustained-release pulmonary targeting and biofilm penetration properties of this formulation will have several advantages over current therapies in treating CF patients with chronic lung infection caused by Pseudomonas aeruginosa.
This Phase 3 European Registration study has been designed to evaluate the efficacy, safety and tolerability of Arikayce™ in treating CF patients with chronic bronchopulmonary infection compared to a currently available antibiotic, TOBI® (tobramycin) Inhalation Solution. Eligible subjects will be randomized 1:1 to receive 590 mg of Arikayce™ once daily via a PARI Investigational eFlow® Nebulizer or 300 mg TOBI® BID via a PARI LC® PLUS nebulizer. Subjects will receive 3 cycles of treatment with each cycle being comprised of 28 days on treatment followed by 28 days off-treatment. Total study duration is up to 186 days (~6 months) including an up to 18 day Screening period. Subjects will be evaluated for safety, tolerability and efficacy bi-weekly during the first 4 weeks of treatment, and thereafter every 4 weeks for the duration of the study. Pharmacokinetics (PK) of Arikayce™ in blood, sputum and 24-hour urine will be determined in a subgroup of study subjects who consent to PK evaluation.
At the completion of the TR02-108 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikayce™ (under a separate protocol TR02-110).
Please refer to this study by its ClinicalTrials.gov identifier: NCT01315678
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|Study Director:||Gina Eagle, MD||Insmed Incorporated|