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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00112359




Registration number
NCT00112359
Ethics application status
Date submitted
1/06/2005
Date registered
2/06/2005
Date last updated
21/04/2011

Titles & IDs
Public title
International Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
Scientific title
A Phase 3, Double-Blind, Multicenter, Multinational, Randomized, Placebo-Controlled Trial Evaluating Aztreonam Lysinate for Inhalation in Cystic Fibrosis Patients With Pulmonary Pseudomonas Aeruginosa (AIR-CF1)
Secondary ID [1] 0 0
CP-AI-007
Universal Trial Number (UTN)
Trial acronym
AIR-CF1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AZLI 75 mg three times a day (TID)
Treatment: Drugs - Placebo three times a day (TID)

Placebo Comparator: Placebo three times a day (TID) -

Experimental: AZLI 75 mg three times a day (TID) -


Treatment: Drugs: AZLI 75 mg three times a day (TID)


Treatment: Drugs: Placebo three times a day (TID)


Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in CFQ-R Respiratory Symptoms Scale (RSS) Score - The CFQ-R was administered at baseline and every visit thereafter. The endpoint was change in respiratory symptoms from baseline, assessed with the CFQ-R respiratory symptoms scale (RSS; range of scores: 0-100; higher scores indicate fewer symptoms).
Timepoint [1] 0 0
Day 0 to Day 28
Secondary outcome [1] 0 0
Change in CFQ-R RSS Score - The CFQ-R was administered at baseline and every visit thereafter. The endpoint was change in respiratory symptoms from baseline, assessed with the CFQ-R RSS (range of scores: 0-100; higher scores indicate fewer symptoms).
Timepoint [1] 0 0
Day 0 to Day 14
Secondary outcome [2] 0 0
Change in CFQ-R RSS Score - The CFQ-R was administered at baseline and every visit thereafter. The endpoint was change in respiratory symptoms from baseline, assessed with the CFQ-R RSS (range of scores: 0-100; higher scores indicate fewer symptoms).
Timepoint [2] 0 0
Day 0 to Day 42
Secondary outcome [3] 0 0
Percent Change in FEV1 (L) - Spirometry was performed according to American Thoracic Society (ATS) guidelines at each visit. The percent change from baseline in forced expiratory volume (liters) in one second (FEV1) was determined at Day 28.
Timepoint [3] 0 0
Day 0 to Day 28
Secondary outcome [4] 0 0
Change From Baseline in Pseudomonas Aeruginosa (PA) Log10 Colony Forming Units (CFU) Per Gram of Sputum - Sputum samples were collected at all participant visits of the study for analysis of microbiology endpoints. Sputum samples were processed for qualitative and quantitative culture of PA (each morphotype). Due to the skewness of the distribution of CFU data, the data were transformed using the base 10 logarithm, in an attempt to normalize the data and allow for parametric tests, before calculating changes. To account for zero values, 1 was added to each CFU measurement before being transformed. Any CFU data values where PA was not isolated from a valid culture were set to zero.
Timepoint [4] 0 0
Day 0 to Day 28
Secondary outcome [5] 0 0
Number of Participants Receiving Intravenous (IV) or Inhaled Antipseudomonal Antibiotics Other Than Trial Drug - Use of IV and inhaled antipseudomonal antibiotics was compiled from data recorded on the Concomitant Medications eCRF.
Timepoint [5] 0 0
Day 0 to Day 42
Secondary outcome [6] 0 0
Number of Participants Hospitalized at Least Once Between Day 0 and Day 42 - Details of all hospitalizations, including the dates of admission and discharge, were recorded on the SAE eCRF.
Timepoint [6] 0 0
Day 0 to Day 42

Eligibility
Key inclusion criteria
- Documentation of CF diagnosis as evidenced by one or more clinical features consistent
with the CF phenotype and one or more of the following criteria:

- Sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine
iontophoresis test (QPIT);

- Two well-characterized mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene; or

- Abnormal nasal potential difference.

- PA present in expectorated sputum or throat swab culture at Screening.

- FEV1 between (and including) 25% and 75% predicted at Screening.

- Negative pregnancy test at Screening.

- Ability to perform reproducible pulmonary function tests.

- Arterial oxygen saturation (SaO2) greater than or equal to 90% on room air at
Screening.

- Ability to provide written informed consent.
Minimum age
6 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Administration of antipseudomonal antibiotics by inhalation, IV, or oral routes
(including azithromycin) within 14 days of Screening.

- Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg
prednisone/day or 20 mg prednisone every other day.

- History of sputum or throat swab culture yielding Burkholderia cepacia in the previous
2 years.

- History of daily continuous oxygen supplementation or requirement for more than 2
liters/minute at night.

- Administration of any investigational drug or use of any investigational device within
28 days of Screening and within 6 half-lives of the investigational drug (whichever
was longer).

- Known local or systemic hypersensitivity to monobactam antibiotics.

- Inability to tolerate short-acting bronchodilator use at least three times daily.

- Changes in protocol-permitted antimicrobial, bronchodilator, anti-inflammatory, or
corticosteroid medications within 7 days prior to Screening or between Screening and
the next visit.

- Changes in physiotherapy technique or schedule within 7 days prior to Screening or
between Screening and the next visit.

- History of lung transplantation.

- A chest x-ray indicating abnormal findings at Screening or within the previous 90
days.

- Abnormal renal or hepatic function at Screening.

- Any serious or active medical or psychiatric illness which, in the opinion of the
investigator, would have interfered with participant treatment, assessment, or
compliance with the protocol.

- Use of aerosolized hypertonic saline (except for sputum induction) during the 14 days
preceding Visit 1.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Westmead Hospital - Westmead
Recruitment hospital [3] 0 0
Royal Children's Hospital - Herston
Recruitment hospital [4] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [5] 0 0
Alfred Hospital - Prahran
Recruitment hospital [6] 0 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [7] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment postcode(s) [3] 0 0
- Adelaide
Recruitment postcode(s) [4] 0 0
- Prahran
Recruitment postcode(s) [5] 0 0
- Nedlands
Recruitment postcode(s) [6] 0 0
- Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Alaska
Country [3] 0 0
United States of America
State/province [3] 0 0
Arizona
Country [4] 0 0
United States of America
State/province [4] 0 0
Arkansas
Country [5] 0 0
United States of America
State/province [5] 0 0
California
Country [6] 0 0
United States of America
State/province [6] 0 0
Connecticut
Country [7] 0 0
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Florida
Country [8] 0 0
United States of America
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Georgia
Country [9] 0 0
United States of America
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Illinois
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United States of America
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Indiana
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United States of America
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Iowa
Country [12] 0 0
United States of America
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Kansas
Country [13] 0 0
United States of America
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Louisiana
Country [14] 0 0
United States of America
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Maine
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Michigan
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Mississippi
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Missouri
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Nevada
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New Jersey
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New York
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North Carolina
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Ohio
Country [23] 0 0
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Pennsylvania
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South Carolina
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Texas
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Utah
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Virginia
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Washington
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Wisconsin
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Canada
State/province [30] 0 0
Alberta
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Canada
State/province [31] 0 0
British Columbia
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Canada
State/province [32] 0 0
Nova Scotia
Country [33] 0 0
Canada
State/province [33] 0 0
Ontario
Country [34] 0 0
Canada
State/province [34] 0 0
Quebec
Country [35] 0 0
New Zealand
State/province [35] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Gilead Sciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study was to evaluate the safety and efficacy of a 28-day course of
aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung
infection due to Pseudomonas aeruginosa (PA).
Trial website
https://clinicaltrials.gov/show/NCT00112359
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Bruce Montgomery, MD
Address 0 0
Corus Pharma, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications