Safety/Tolerability Study of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
Phase 1b/2a Multidose Safety and Tolerability Study of Liposomal Amikacin for Inhalation (Arikayce™) in Cystic Fibrosis Patient With Chronic Infections Due to Pseudomonas Aeruginosa.
1 other identifier
interventional
41
1 country
19
Brief Summary
This is a study to determine the safety and tolerability of 28 days of daily dosing of 560 mg of Arikayce™ versus placebo and daily dosing of 70 mg and 140 mg of Arikayce™ versus placebo in patients who have Cystic fibrosis (CF) and chronic infection due to pseudomonas aeruginosa.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Jan 2008
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 13, 2007
CompletedFirst Posted
Study publicly available on registry
November 15, 2007
CompletedStudy Start
First participant enrolled
January 1, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2009
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2009
CompletedResults Posted
Study results publicly available
June 4, 2019
CompletedJune 4, 2019
May 1, 2019
1.4 years
November 13, 2007
April 3, 2019
May 14, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Participants With Treatment-Emergent Adverse Events
To evaluate the safety and tolerability of 28 days of daily dosing of nebulized Arikayce™, liposomal amikacin for inhalation.
56 days
Secondary Outcomes (8)
Pharmacokinetics of Arikayce™ in Serum
Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikayce™ in Sputum
Day 1 post-dose, Day 14 pre- and post-dose, Day 28 pre- and post-dose
Pharmacokinetics (PK) of Arikayce™ in Urine
Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikayce™ in Serum
Day 1, Day 14 and Day 28
Pulmonary Function: Pre-Dose FEV1 (%-Predicted)
Baseline, Day 28, Day 56, Day 70 and Day 84
- +3 more secondary outcomes
Study Arms (5)
A
ACTIVE COMPARATORArikayce™ at 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo.
B
PLACEBO COMPARATORMatching placebo for 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo.
C
ACTIVE COMPARATORArikayce™ at 70 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
D
ACTIVE COMPARATORArikayce™ at 140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
E
PLACEBO COMPARATORMatching placebo for 70 mg/140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
Interventions
Arikayce™ at 560 mg Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Matching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Matching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Eligibility Criteria
You may qualify if:
- Male or female study subjects must be adults (≥ 6 years of age)
- Confirmed diagnosis of CF
- History of chronic infection with P.aeruginosa
- FEV1 ≥40% of predicted at Screening
- Ability to comply with study medication use, study visits and procedures
- Ability to produce 0.5 grams of sputum
You may not qualify if:
- Administration of any investigational drug within 8 weeks to Study Day 1
- Emergency room visit or hospitalization for CF or respiratory-related illness within 4 weeks prior to screening
- History of alcohol, medication or illicit drug abuse within 1 yr. to screening
- History of lung transplantation
- Female of childbearing potential who are not practicing an acceptable method of birth control or who are lactating
- Positive Pregnancy test
- Use of any anti-pseudomonal antibiotics within 28 days prior to Study Day 1
- Initiation of chronic therapy within 28 days prior to Study Day 1
- History of sputum or throat swab culture yielding Burkholderia cepacia within 2 years prior to screening
- History of mycobacterial and/or Aspergillus infection requiring treatment within 2 years prior to screening
- History of biliary cirrhosis with portal hypertension, or splenomegaly
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (19)
Unknown Facility
Birmingham, Alabama, United States
Unknown Facility
Los Angeles, California, United States
Unknown Facility
Miami, Florida, United States
Unknown Facility
Orlando, Florida, United States
Unknown Facility
Indianapolis, Indiana, United States
Unknown Facility
Iowa City, Iowa, United States
Unknown Facility
Baltimore, Maryland, United States
Unknown Facility
Boston, Massachusetts, United States
Unknown Facility
Ann Arbor, Michigan, United States
Unknown Facility
Minneapolis, Minnesota, United States
Unknown Facility
Jackson, Mississippi, United States
Unknown Facility
St Louis, Missouri, United States
Unknown Facility
Morristown, New Jersey, United States
Unknown Facility
New Brunswick, New Jersey, United States
Unknown Facility
Albuquerque, New Mexico, United States
Unknown Facility
Rochester, New York, United States
Unknown Facility
Philadelphia, Pennsylvania, United States
Unknown Facility
Sioux Falls, South Dakota, United States
Unknown Facility
Seattle, Washington, United States
Related Publications (2)
Okusanya OO, Bhavnani SM, Hammel JP, Forrest A, Bulik CC, Ambrose PG, Gupta R. Evaluation of the pharmacokinetics and pharmacodynamics of liposomal amikacin for inhalation in cystic fibrosis patients with chronic pseudomonal infections using data from two phase 2 clinical studies. Antimicrob Agents Chemother. 2014 Sep;58(9):5005-15. doi: 10.1128/AAC.02421-13. Epub 2014 Mar 31.
PMID: 24687506BACKGROUNDClancy JP, Dupont L, Konstan MW, Billings J, Fustik S, Goss CH, Lymp J, Minic P, Quittner AL, Rubenstein RC, Young KR, Saiman L, Burns JL, Govan JR, Ramsey B, Gupta R; Arikace Study Group. Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection. Thorax. 2013 Sep;68(9):818-25. doi: 10.1136/thoraxjnl-2012-202230. Epub 2013 Jun 8.
PMID: 23749840DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Kevin Mange (Senior VP, Clinical Development and Medical Affairs)
- Organization
- Insmed Incorporated
Study Officials
- STUDY DIRECTOR
Gina Eagle, MD
Insmed Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 13, 2007
First Posted
November 15, 2007
Study Start
January 1, 2008
Primary Completion
June 1, 2009
Study Completion
June 1, 2009
Last Updated
June 4, 2019
Results First Posted
June 4, 2019
Record last verified: 2019-05