Cystic FibrosisPhase 3AIR-CF1NCT00112359
International Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
A Phase 3 study testing AZLI 75 mg three times a day (TID) in people with Cystic Fibrosis, run by Gilead Sciences at 53 sites in 4 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- AZLI 75 mg three times a day (TID)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 166 participants
- Where
- 53 sitesin 4 countries
- Study dates
- May 2005to Apr 2007
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
53 sites in 4 countries.
53 sites in 4 countries
United States40 sites
Albany Medical College
Albany, New York
Pediatric Breathing Disorders Clinic
Anchorage, Alaska
University of Michigan
Ann Arbor, Michigan
Central Maine Pulmonary Associates
Auburn, Maine
Medical College of Georgia
Augusta, Georgia
University of Alabama at Birmingham
Birmingham, Alabama
University of North Carolina
Chapel Hill, North Carolina
Children's Memorial Hospital / Northwestern University
Chicago, Illinois
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio
University of Missouri
Columbia, Missouri
Pediatric Pulmonary Associates, South Carolina
Columbia, South Carolina
Baylor Martha Foster Lung Care Center
Dallas, Texas
University of Florida Health Sciences Center
Gainesville, Florida
Riley Hospital for Children
Indianapolis, Indiana
University of Iowa
Iowa City, Iowa
University of Mississippi Medical Center
Jackson, Mississippi
Children's Lung Specialists
Las Vegas, Nevada
University of Arkansas for Medical Sciences
Little Rock, Arkansas
St. Barnabas Healthcare System
Livingston, New Jersey
Miller Children's Hospital
Long Beach, California
Children's Hospital, Los Angeles
Los Angeles, California
University of Wisconsin
Madison, Wisconsin
Yale New Haven Hospital
New Haven, Connecticut
Long Island Jewish Medical Center
New Hyde Park, New York
Tulane University Health Sciences Center
New Orleans, Louisiana
Children's Hospital of Orange County
Orange, California
Nemours Children's Clinic, Orlando
Orlando, Florida
University of Pennsylvania Health System
Philadelphia, Pennsylvania
Phoenix Children's Hospital
Phoenix, Arizona
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania
Naval Medical Center
Portsmouth, Virginia
Pediatric Pulmonary Center/Virginia Commonwealth University
Richmond, Virginia
Capital Allergy and Respiratory Disease Center
Sacramento, California
University of Utah
Salt Lake City, Utah
Alamo Clinical Research Associates
San Antonio, Texas
University of Washington Medical Center
Seattle, Washington
Louisiana State University Health Sciences Center
Shreveport, Louisiana
St. Louis University
St Louis, Missouri
SUNY Upstate Medical University
Syracuse, New York
Via Christi - St. Francis Regional Medical Center
Wichita, Kansas
Australia7 sites
Royal Adelaide Hospital
Adelaide, South Australia
Royal Children's Hospital
Herston, Queensland
Sir Charles Gairdner Hospital
Nedlands, Western Australia
Princess Margaret Hospital for Children
Perth, Western Australia
Alfred Hospital
Prahran, Victoria
Children's Hospital at Westmead
Westmead, New South Wales
Westmead Hospital
Westmead, New South Wales
Canada5 sites
Capital Health and the Governors of the University of Alberta
Edmonton, Alberta
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia
Brian Lyttle Professional Corporation
London, Ontario
Centre Hospitalier de l'Universite de Montreal (CHUM)
Montreal, Quebec
St. Paul's Hospital
Vancouver, British Columbia
About this study
Official 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)
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).
Read the detailed description
CF patients often have lung infections that occur repeatedly or worsen over time. The lung infections are often caused by a bacteria called Pseudomonas aeruginosa (PA). Treatment with antibiotics can stop or slow down the growth of the bacteria. The antibiotics may be given by mouth, intravenously (IV), or by inhalation as a mist. The purpose of this study was to evaluate the safety and efficacy of AZLI, an investigational formulation of the antibiotic aztreonam and administered TID using the PARI eFlow® electronic nebulizer, in CF patients with PA.
In this study, participant eligibility was assessed at a screening visit 7 to 14 days prior to the baseline visit (Day 0). Those participants who continued to meet eligibility criteria at Day 0 were randomized and began a 28-day course of blinded study treatment (AZLI TID or placebo TID). Participants returned for clinic visits at Day 14, an end of treatment visit at Day 28, and a follow-up visit 14 days after the last dose of study drug (Day 42).
- Conditions
- Cystic Fibrosis
- Being tested
- AZLI 75 mg three times a day (TID), Placebo three times a day (TID)
- Keywords
- Cystic Fibrosis, Pseudomonas aeruginosa, Pulmonary Cystic Fibrosis
- Medical subject headings
- Cystic Fibrosis, Pseudomonas Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AZLI 75 mg three times a day (TID)Drug
Placebo three times a day (TID)Drug
| Study group | Role | What they receive |
|---|---|---|
| Placebo three times a day (TID) | Placebo comparator | Not described Receives: Drug: Placebo three times a day (TID) |
| AZLI 75 mg three times a day (TID) | Experimental | Not described Receives: Drug: AZLI 75 mg three times a day (TID) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Day 0 to Day 28
Secondary outcomes (6)
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).
Measured: Day 0 to Day 14
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).
Measured: Day 0 to Day 42
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.
Measured: Day 0 to Day 28
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.
Measured: Day 0 to Day 28
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.
Measured: Day 0 to Day 42
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.
Measured: Day 0 to Day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2005 (estimated)
- Study startMay 2005
- Primary completionApr 2007
- Study completionApr 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Gilead SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 166 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gilead Sciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Bruce Montgomery, MD, Study director, Corus Pharma, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00112359
- Sponsor's protocol ID
- CP-AI-007
- Acronym
- AIR-CF1
- Status last verified
- Mar 2011
- First submitted
- 1 Jun 2005
- Last updated
- 21 Apr 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Retsch-Bogart GZ, Quittner AL, Gibson RL, Oermann CM, McCoy KS, Montgomery AB, Cooper PJ. Efficacy and safety of inhaled aztreonam lysine for airway pseudomonas in cystic fibrosis. Chest. 2009 May;135(5):1223-1232. doi: 10.1378/chest.08-1421.
PubMed 19420195
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT00112359
- Acronym / sponsor ID
- AIR-CF1 / CP-AI-007
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Pseudomonas aeruginosa, Pulmonary Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 166 participants (actual)
- Lead sponsor
- Gilead SciencesIndustry (pharma, biotech, devices)
- Study start
- May 2005
- Primary completion
- Apr 2007 (actual)
- Study completion
- Apr 2007 (actual)
- First posted
- 2 Jun 2005 (estimated)
- Results
- Posted 21 Apr 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
CF patients often have lung infections that occur repeatedly or worsen over time. The lung infections are often caused by a bacteria called Pseudomonas aeruginosa (PA). Treatment with antibiotics can stop or slow down the growth of the bacteria. The antibiotics may be given by mouth, intravenously (IV), or by inhalation as a mist. The purpose of this study was to evaluate the safety and efficacy of AZLI, an investigational formulation of the antibiotic aztreonam and administered TID using the PARI eFlow® electronic nebulizer, in CF patients with PA.
In this study, participant eligibility was assessed at a screening visit 7 to 14 days prior to the baseline visit (Day 0). Those participants who continued to meet eligibility criteria at Day 0 were randomized and began a 28-day course of blinded study treatment (AZLI TID or placebo TID). Participants returned for clinic visits at Day 14, an end of treatment visit at Day 28, and a follow-up visit 14 days after the last dose of study drug (Day 42).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo three times a day (TID) | Placebo comparator | Drug: Placebo three times a day (TID) |
| AZLI 75 mg three times a day (TID) | Experimental | Drug: AZLI 75 mg three times a day (TID) |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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:
- 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.
Key exclusion criteria (14 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AZLI 75 mg three times a day (TID) | Drug | AZLI 75 mg three times a day (TID) |
| Placebo three times a day (TID) | Drug | Placebo three times a day (TID) |
Primary outcome
Change in CFQ-R Respiratory Symptoms Scale (RSS) Score
Time frame: Day 0 to Day 28
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).
6 secondary outcomes
Change in CFQ-R RSS Score
Time frame: Day 0 to Day 14
Change in CFQ-R RSS Score
Time frame: Day 0 to Day 42
Percent Change in FEV1 (L)
Time frame: Day 0 to Day 28
Change From Baseline in Pseudomonas Aeruginosa (PA) Log10 Colony Forming Units (CFU) Per Gram of Sputum
Time frame: Day 0 to Day 28
Number of Participants Receiving Intravenous (IV) or Inhaled Antipseudomonal Antibiotics Other Than Trial Drug
Time frame: Day 0 to Day 42
Number of Participants Hospitalized at Least Once Between Day 0 and Day 42
Time frame: Day 0 to Day 42
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 19420195Retsch-Bogart GZ, Quittner AL, Gibson RL, Oermann CM, McCoy KS, Montgomery AB, Cooper PJ. Efficacy and safety of inhaled aztreonam lysine for airway pseudomonas in cystic fibrosis. Chest. 2009 May;135(5):1223-1232. doi: 10.1378/chest.08-1421.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bruce Montgomery, MD study director
Corus Pharma, Inc.
53 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AZLI 75 mg three times a day (TID) in people with Cystic Fibrosis, run by Gilead Sciences at 53 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Apr 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 53 sites in United States, Australia, Canada, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Apr 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in CFQ-R Respiratory Symptoms Scale (RSS) Score (time frame: Day 0 to Day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00112359, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Apr 2011.