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Extension Study of Arikayce in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa Infection
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
Read our disclaimer for details.
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 ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
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