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. Identifier: NCT01316276
Recruitment Status : Completed
First Posted : March 16, 2011
Last Update Posted : October 3, 2016
Information provided by (Responsible Party):
Insmed Incorporated

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Liposomal amikacin for inhalation Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 206 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long Term Safety and Tolerability of Open-Label Liposomal Amikacin for Inhalation (Arikayce™) in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
Study Start Date : October 2012
Actual Primary Completion Date : July 2015

Arm Intervention/treatment
Experimental: Arikayce Drug: Liposomal amikacin for inhalation
  • 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.

Primary Outcome Measures :
  1. 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

  2. Relative change in FEV1 [liters] and FEV1 % predicted [ Time Frame: Day 337 - first extension period ]
  3. Acute tolerability as measured by PFT changes pre- to post-dose [ Time Frame: Day 337 - first extension period ]

Secondary Outcome Measures :
  1. Time to first protocol defined pulmonary exacerbation and proportion of subjects experiencing a protocol defined pulmonary exacerbation [ Time Frame: Day 337 - first extension period ]
  2. 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 ]
  3. Shift in minimum inhibitory concentration for Pseudomonas aeruginosa throughout the study [ Time Frame: Day 337 - first extension period ]
  4. Evaluation of emergent pathogens [ Time Frame: Day 337 - first extension period ]

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01316276

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Sponsors and Collaborators
Insmed Incorporated
Study Director: Gina Eagle, MD Insmed Incorporated

Responsible Party: Insmed Incorporated Identifier: NCT01316276     History of Changes
Other Study ID Numbers: TR02-110
First Posted: March 16, 2011    Key Record Dates
Last Update Posted: October 3, 2016
Last Verified: September 2016

Keywords provided by Insmed Incorporated:
Cystic Fibrosis
Respiratory Infections
Pulmonary Cystic Fibrosis
Anti-bacterial Agent

Additional relevant MeSH terms:
Cystic Fibrosis
Pseudomonas Infections
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Gram-Negative Bacterial Infections
Bacterial Infections
Anti-Bacterial Agents
Anti-Infective Agents