Extension Study of Arikayce in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa Infection
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The purpose of this study is to evaluate the long term safety and tolerability of Arikayce in Cystic Fibrosis patients. This long-term, open-label, multi-cycle extension study will enroll subjects who have successfully completed study TR02-108, were compliant with the study protocol, and did not meet any of the listed study discontinuation criteria. The safety and tolerability of Arikayce will be evaluated for up to approximately 2 years.
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 in two consecutive extension periods, each consisting of 6 cycles for a total of 12 cycles. Each cycle consists of 28 days on-treatment followed by 28 days off-treatment.
Incidence of treatment emergent adverse events [ Time Frame: Day 337 - first extension period ]
Treatment emergent adverse events including serious adverse events and adverse events leading to permanent discontinuation of study drug
Relative change in FEV1 [liters] and FEV1 % predicted [ Time Frame: Day 337 - first extension period ]
Acute tolerability as measured by PFT changes pre- to post-dose [ Time Frame: Day 337 - first extension period ]
Secondary Outcome Measures
Time to first protocol defined pulmonary exacerbation and proportion of subjects experiencing a protocol defined pulmonary exacerbation [ Time Frame: Day 337 - first extension period ]
Time to first antipseudomonal antibiotic treatment for protocol defined pulmonary exacerbation, proportion of subjects initiating treatment and number of days of treatment [ Time Frame: Day 337 - first extension period ]
Shift in minimum inhibitory concentration for Pseudomonas aeruginosa throughout the study [ Time Frame: Day 337 - first extension period ]
Evaluation of emergent pathogens [ Time Frame: Day 337 - first extension period ]
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Ages Eligible for Study:
6 Years and older (Child, Adult, Senior)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Key Inclusion Criteria:
Written informed consent or assent
Subject has completed study TR02-108, and has been compliant with the study protocol
Women of childbearing potential must agree to use reliable methods of contraception for the duration of the study
Key Exclusion Criteria:
Subject met any of the listed criteria for study drug discontinuation in protocol TR02-108.
Abnormal laboratory assessments including LFT (≥ 3× ULN), serum creatinine (> 2× ULN) and ANC (< 1000).
Psychotic, addictive or other disorder limiting the ability to provide informed consent or to comply with study requirements.
History of alcohol, medication or illicit drug abuse within the 6 months prior to consent.
Smoking tobacco or any substance within 6 months prior to consent or anticipated inability to refrain from smoking throughout the study