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Extension Study of Arikace™ in CF Patients With Chronic Pseudomonas Aeruginosa Infection

This study is currently recruiting participants.
Verified December 2012 by Insmed
Sponsor:
Information provided by (Responsible Party):
Insmed
ClinicalTrials.gov Identifier:
NCT01316276
First received: March 14, 2011
Last updated: December 11, 2012
Last verified: December 2012
History of Changes
  Purpose

The purpose of this study is to evaluate the long term safety and tolerability of Arikace™ 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 Arikace™ 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 (Arikace™) in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Insmed:

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 ]

Estimated Enrollment: 250
Study Start Date: October 2012
Estimated Study Completion Date: May 2015
Estimated Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arikace™ Drug: Liposomal amikacin for inhalation
  • Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
  • 560 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.

Detailed Description:

Eligible subjects will receive 560 mg Arikace™ once daily via a PARI Investigational eFlow® Nebulizer for 28 days followed by a 28 day off treatment period. This cycle (28 days on-treatment, 28 days off-treatment) will be repeated for up to twelve cycles. The study will be implemented as two consecutive extension periods, each consisting of 48 weeks (approximately 12 months). Subjects will be re-consented for the second extension period at the completion of the first extension period. The total study period will be up to 96 weeks (approximately 2 years). In the first extension period, safety, tolerability and efficacy will be evaluated after 6 cycles. Similar safety and tolerability endpoints will be assessed after 6 more cycles for subjects who consent for the second extension period.

During the first 28 days of treatment, subjects will be evaluated at the clinic bi-weekly for safety, tolerability and efficacy. Thereafter, for the duration of the study, subjects will be evaluated at the clinic on the first and last days of dosing during the on-treatment periods. A final site visit will occur 28 days after last dose of Arikace™.

  Eligibility

Ages Eligible for Study:   6 Years and older
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01316276

Contacts
Contact: Scott Constantine CF@insmed.com

Locations
United Kingdom
Royal Brompton Hospital, Department of Respiratory Medicine Recruiting
London, United Kingdom
Contact: Angela Howard     020 7351 8182     a.howard@rbht.nhs.uk    
Principal Investigator: Diana Bilton, MD FRCP            
Sponsors and Collaborators
Insmed
Investigators
Principal Investigator: Michael Konstan, MD University Hospital Case Medical Center
Principal Investigator: Diana Bilton, MB ChB Department of Cystic Fibrosis, Royal Brompton Hospital
Principal Investigator: John P Clancy, MD Children's Hospital Medical Center, Cincinnati
  More Information

No publications provided

Responsible Party: Insmed
ClinicalTrials.gov Identifier: NCT01316276     History of Changes
Other Study ID Numbers: TR02-110
Study First Received: March 14, 2011
Last Updated: December 11, 2012
Health Authority: European Union: European Medicines Agency
Canada: Health Canada

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

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

ClinicalTrials.gov processed this record on June 17, 2013