Extension Study of Arikayce in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa Infection

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Insmed Incorporated
ClinicalTrials.gov Identifier:
NCT01316276
First received: March 14, 2011
Last updated: December 8, 2015
Last verified: December 2015
  Purpose
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 Intervention Phase
Cystic Fibrosis
Drug: Liposomal amikacin for inhalation
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Insmed Incorporated:

Primary Outcome Measures:
  • Incidence of treatment emergent adverse events [ Time Frame: Day 337 - first extension period ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
  • Acute tolerability as measured by PFT changes pre- to post-dose [ Time Frame: Day 337 - first extension period ] [ Designated as safety issue: Yes ]

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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]
  • Shift in minimum inhibitory concentration for Pseudomonas aeruginosa throughout the study [ Time Frame: Day 337 - first extension period ] [ Designated as safety issue: Yes ]
  • Evaluation of emergent pathogens [ Time Frame: Day 337 - first extension period ] [ Designated as safety issue: Yes ]

Enrollment: 206
Study Start Date: October 2012
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   6 Years and older   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01316276

  Show 50 Study Locations
Sponsors and Collaborators
Insmed Incorporated
Investigators
Study Director: Gina Eagle, MD Insmed Incorporated
  More Information

Responsible Party: Insmed Incorporated
ClinicalTrials.gov Identifier: NCT01316276     History of Changes
Other Study ID Numbers: TR02-110 
Study First Received: March 14, 2011
Last Updated: December 8, 2015
Health Authority: European Union: European Medicines Agency
Canada: Health Canada

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

Additional relevant MeSH terms:
Infection
Fibrosis
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
Amikacin
Anti-Bacterial Agents
Anti-Infective Agents

ClinicalTrials.gov processed this record on July 24, 2016