Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections
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ClinicalTrials.gov Identifier: NCT01315678 |
Recruitment Status
:
Completed
First Posted
: March 15, 2011
Last Update Posted
: April 17, 2015
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Tracking Information | ||||
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First Submitted Date ICMJE | March 14, 2011 | |||
First Posted Date ICMJE | March 15, 2011 | |||
Last Update Posted Date | April 17, 2015 | |||
Study Start Date ICMJE | March 2012 | |||
Actual Primary Completion Date | June 2013 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
Relative change in Forced Expiratory Volume in 1 second (FEV1) from baseline to end of study (Day 168) [ Time Frame: 168 days ] | |||
Original Primary Outcome Measures ICMJE | Same as current | |||
Change History | Complete list of historical versions of study NCT01315678 on ClinicalTrials.gov Archive Site | |||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Same as current | |||
Current Other Outcome Measures ICMJE | Not Provided | |||
Original Other Outcome Measures ICMJE | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections | |||
Official Title ICMJE | Randomized, Open-Label, Active-Controlled, Multicenter Study to Assess the Efficacy, Safety and Tolerability of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa | |||
Brief Summary | A major factor in the respiratory health of Cystic Fibrosis (CF) subjects is the prevalence of chronic Pseudomonas aeruginosa infections. The Pseudomonas aeruginosa infection rate in CF patients increases with age and by age 18 years approximately 85% of CF patients in the US are infected. Liposomal amikacin for inhalation (Arikayce™) was developed as a possible treatment for chronic infection due to Pseudomonas aeruginosa in CF patients. The purpose of this European Registration study is to determine whether Arikayce™ is effective in treating chronic lung infections caused by Pseudomonas aeruginosa in Cystic Fibrosis subjects. The effectiveness, safety and tolerability of Arikayce™ will be compared to TOBI, an inhalation antibiotic already available for use. The study will enroll approximately 300 subjects in clinics in Europe and Canada. Subjects will be required to visit the clinic 9 times (including the Screening visit) over a period of approximately 6 months. No overnight stays at the clinic will be required. |
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Detailed Description | Cystic Fibrosis (CF) is a genetic disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients with CF manifest pathological changes in a variety of organs that express CFTR. The lungs are frequently affected often resulting in chronic infections by bacteria such as Pseudomonas aeruginosa and airway inflammation. Treatment of chronic lung infections is one of the principal goals of CF therapy. Arikayce™ (liposomal amikacin for inhalation) is a sustained-release formulation of amikacin encapsulated inside nanoscale liposomal carriers designed for administration via inhalation. It is hypothesized that the sustained-release pulmonary targeting and biofilm penetration properties of this formulation will have several advantages over current therapies in treating CF patients with chronic lung infection caused by Pseudomonas aeruginosa. This Phase 3 European Registration study has been designed to evaluate the efficacy, safety and tolerability of Arikayce™ in treating CF patients with chronic bronchopulmonary infection compared to a currently available antibiotic, TOBI® (tobramycin) Inhalation Solution. Eligible subjects will be randomized 1:1 to receive 590 mg of Arikayce™ once daily via a PARI Investigational eFlow® Nebulizer or 300 mg TOBI® BID via a PARI LC® PLUS nebulizer. Subjects will receive 3 cycles of treatment with each cycle being comprised of 28 days on treatment followed by 28 days off-treatment. Total study duration is up to 186 days (~6 months) including an up to 18 day Screening period. Subjects will be evaluated for safety, tolerability and efficacy bi-weekly during the first 4 weeks of treatment, and thereafter every 4 weeks for the duration of the study. Pharmacokinetics (PK) of Arikayce™ in blood, sputum and 24-hour urine will be determined in a subgroup of study subjects who consent to PK evaluation. At the completion of the TR02-108 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikayce™ (under a separate protocol TR02-110). |
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Study Type ICMJE | Interventional | |||
Study Phase | Phase 3 | |||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Pseudomonas Aeruginosa Infection | |||
Intervention ICMJE |
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Study Arms |
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
302 | |||
Original Estimated Enrollment ICMJE |
300 | |||
Actual Study Completion Date | August 2013 | |||
Actual Primary Completion Date | June 2013 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Key Inclusion Criteria:
Key Exclusion Criteria:
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Sex/Gender |
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Ages | 6 Years and older (Child, Adult, Senior) | |||
Accepts Healthy Volunteers | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | Austria, Belgium, Bulgaria, Canada, Denmark, France, Germany, Greece, Hungary, Ireland, Italy, Netherlands, Poland, Serbia, Slovakia, Spain, Sweden, United Kingdom | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01315678 | |||
Other Study ID Numbers ICMJE | TR02-108 | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement | Not Provided | |||
Responsible Party | Insmed Incorporated | |||
Study Sponsor ICMJE | Insmed Incorporated | |||
Collaborators ICMJE | Not Provided | |||
Investigators ICMJE |
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PRS Account | Insmed Incorporated | |||
Verification Date | March 2015 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |