NCT00104520

Brief Summary

The purpose of this study was to evaluate the safety and efficacy of aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
211

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Feb 2005

Geographic Reach
1 country

56 active sites

Status
completed

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

Study Start

First participant enrolled

February 1, 2005

Completed
28 days until next milestone

First Submitted

Initial submission to the registry

March 1, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

March 2, 2005

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2006

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2006

Completed
4.5 years until next milestone

Results Posted

Study results publicly available

March 11, 2011

Completed
Last Updated

March 11, 2011

Status Verified

September 1, 2010

Enrollment Period

1.6 years

First QC Date

March 1, 2005

Results QC Date

September 10, 2010

Last Update Submit

February 16, 2011

Conditions

Keywords

Cystic FibrosisPseudomonas aeruginosaPulmonary Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Time to Need for Inhaled or Intravenous (IV) Antipseudomonal Antibiotics

    The primary endpoint was time to need for a course of inhaled or IV antipseudomonal antibiotics with documented physician assessment of need for antibiotics. Antipseudomonal Antibiotic need was documented based on the presence of at least one of the following four symptoms predictive of pulmonary exacerbation: decreased exercise tolerance, increased cough, increased sputum / chest congestion, decreased appetite, or other.

    Day 0 to Day 84 (end of study)

Secondary Outcomes (4)

  • Change in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Symptoms Scale (RSS) Score

    Day 0 to Day 28

  • Percent Change in Forced Expiratory Volume in 1 Second (FEV1) (L)

    Day 0 to Day 28

  • Number of Hospitalization Days

    Day 0 to Day 84

  • Change From Baseline in Pseudomonas Aeruginosa (PA) Log10 Colony Forming Units (CFU) Per Gram of Sputum

    Day 0 to Day 28

Other Outcomes (2)

  • Number of Participants With Other Pathogens

    Day 0 and Day 28

  • Minimum Concentration of Aztreonam Inhibiting 50% (MIC50) and 90% (MIC90) of All PA Isolates (μg/mL)

    Day 0 to Day 28

Study Arms (2)

Placebo (pooled two times a day [BID]/three times a day [TID])

PLACEBO COMPARATOR
Drug: Placebo two times a day (BID)/three times a day (TID)

AZLI (pooled two times a day [BID]/three times a day [TID])

EXPERIMENTAL
Drug: AZLI 75 mg two times a day (BID)/three times a day (TID)

Interventions

AZLI (pooled two times a day [BID]/three times a day [TID])
Placebo (pooled two times a day [BID]/three times a day [TID])

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • CF as diagnosed by:
  • Documented sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine iontophoresis test; or
  • Two well-characterized genetic mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene; or
  • Abnormal nasal potential difference with accompanying symptoms characteristic of CF.
  • PA present in expectorated sputum or throat swab culture at Screening.
  • Participants must have received three or more courses of TIS within the previous 12 months.
  • Participants on chronic azithromycin must have had no change in regimen in the previous 3 months and must have had a need for TIS and/or additional antipseudomonal therapy since initiation of azithromycin.
  • Forced expiratory volume in 1 second (FEV1) between (and including) 25% and 75% predicted at Screening.
  • Ability to perform reproducible pulmonary function tests.
  • Arterial oxygen saturation (SaO2) greater than or equal to 90% on room air at Screening.

You may not qualify if:

  • Current use of oral corticosteroids in doses exceeding the equivalent of 10 mg prednisone a day or 20 mg prednisone every other day.
  • History of sputum or throat culture swab yielding Burkholderia cepacia in the past 2 years.
  • History of daily continuous oxygen supplementation or requirement for more than 2 liters/minute at night.
  • Administration of any investigational drug or device within 28 days of Screening (Visit 1) or within 6 half-lives of the investigational drug (whichever was longer).
  • Known local or systemic hypersensitivity to monobactam antibiotics.
  • Inability to tolerate inhalation of a short acting Beta-2 agonist.
  • Changes in antimicrobial, bronchodilator, anti-inflammatory, or corticosteroid medications within 7 days before Screening or between Screening and the next visit.
  • Changes in physiotherapy technique or schedule within 7 days before 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 or serum chemistry at Screening (aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\] greater than 5 times the upper limit of normal range; Creatinine greater than 2 times the upper limit of normal range).
  • Positive pregnancy test at Screening.
  • Female of childbearing potential who was lactating or in the opinion of the investigator was not practicing acceptable birth control.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (56)

Phoenix Children's Hospital

Phoenix, Arizona, United States

Location

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Location

University of California, San Diego

La Jolla, California, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, United States

Location

Kaiser Permanente Medical Care Program

Oakland, California, United States

Location

Children's Hospital, Orange Co.

Orange, California, United States

Location

Stanford University Hospital and Medical Center

Palo Alto, California, United States

Location

UC Davis Medical Center

Sacramento, California, United States

Location

Children's Hospital

Denver, Colorado, United States

Location

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Location

University of Florida Health Sciences Center

Gainesville, Florida, United States

Location

Nemours Children's Clinic, Jacksonville

Jacksonville, Florida, United States

Location

University of Miami School of Medicine

Miami, Florida, United States

Location

Nemours Children's Clinic

Orlando, Florida, United States

Location

Pediatric Pulmonary Associates, Florida

St. Petersburg, Florida, United States

Location

Emory Healthcare

Atlanta, Georgia, United States

Location

Medical College of Georgia

Augusta, Georgia, United States

Location

Children's Memorial Hospital/Northwestern University

Chicago, Illinois, United States

Location

Chicago Children's Asthma Respiratory and Exercise Specialists

Glenview, Illinois, United States

Location

Loyola University Medical Center

Maywood, Illinois, United States

Location

North Suburban Pulmonary / Critical Care Consultants

Niles, Illinois, United States

Location

Indiana University

Indianapolis, Indiana, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, United States

Location

Maine Medical Center

Portland, Maine, United States

Location

Children's Hospital, Boston

Boston, Massachusetts, United States

Location

Floating Hospital for Children

Boston, Massachusetts, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, United States

Location

University of Michigan

Ann Arbor, Michigan, United States

Location

Children's Hospital of Michigan/Wayne State University

Detroit, Michigan, United States

Location

University of Minnesota

Minneapolis, Minnesota, United States

Location

Children's Lung Specialists, Ltd.

Las Vegas, Nevada, United States

Location

Morristown Memorial Hospital

Morristown, New Jersey, United States

Location

Albany Medical College

Albany, New York, United States

Location

Long Island College Hospital

Brooklyn, New York, United States

Location

Children's Hospital of Buffalo

Buffalo, New York, United States

Location

Long Island Jewish Medical Center

New Hyde Park, New York, United States

Location

Columbia University Medical Center

New York, New York, United States

Location

State University of New York Stony Brook

Stony Brook, New York, United States

Location

Children's Hospital of Westchester Medical Center/New York Medical College

Valhalla, New York, United States

Location

Akron Children's Hospital

Akron, Ohio, United States

Location

Columbus Children's Hospital, Ohio State University

Columbus, Ohio, United States

Location

Children's Medical Center

Dayton, Ohio, United States

Location

Dr. Santiago Reyes

Oklahoma City, Oklahoma, United States

Location

Oregon Health & Science University

Portland, Oregon, United States

Location

Penn State University Hershey Medical Center

Hershey, Pennsylvania, United States

Location

Drexel University College of Medicine

Philadelphia, Pennsylvania, United States

Location

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, United States

Location

Children's Hospital of Pittsburg

Pittsburgh, Pennsylvania, United States

Location

Rhode Island Hospital

Providence, Rhode Island, United States

Location

Medical University of South Carolina

Charleston, South Carolina, United States

Location

Pediatric Pulmonary Associates, South Carolina

Columbia, South Carolina, United States

Location

Baylor College of Medicine

Houston, Texas, United States

Location

Alamo Clinical Research Associates

San Antonio, Texas, United States

Location

Pediatric Pulmonary Center

Richmond, Virginia, United States

Location

Children's Hospital and Regional Medical Center

Seattle, Washington, United States

Location

West Virginia University

Morgantown, West Virginia, United States

Location

Related Publications (2)

  • Quittner AL, Modi AC, Wainwright C, Otto K, Kirihara J, Montgomery AB. Determination of the minimal clinically important difference scores for the Cystic Fibrosis Questionnaire-Revised respiratory symptom scale in two populations of patients with cystic fibrosis and chronic Pseudomonas aeruginosa airway infection. Chest. 2009 Jun;135(6):1610-1618. doi: 10.1378/chest.08-1190. Epub 2009 May 15.

  • McCoy KS, Quittner AL, Oermann CM, Gibson RL, Retsch-Bogart GZ, Montgomery AB. Inhaled aztreonam lysine for chronic airway Pseudomonas aeruginosa in cystic fibrosis. Am J Respir Crit Care Med. 2008 Nov 1;178(9):921-8. doi: 10.1164/rccm.200712-1804OC. Epub 2008 Jul 24.

MeSH Terms

Conditions

Cystic FibrosisPseudomonas Infections

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesGram-Negative Bacterial InfectionsBacterial InfectionsBacterial Infections and MycosesInfections

Limitations and Caveats

The study was designed such that participants were discontinued from study participation upon meeting the primary endpoint (time to need for inhaled or IV antipseudomonal antibiotics).

Results Point of Contact

Title
Mark Bresnik, MD, Director, Clinical Research
Organization
Gilead Sciences, Inc.

Study Officials

  • Karen McCoy, MD

    Nationwide Children's Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

March 1, 2005

First Posted

March 2, 2005

Study Start

February 1, 2005

Primary Completion

September 1, 2006

Study Completion

September 1, 2006

Last Updated

March 11, 2011

Results First Posted

March 11, 2011

Record last verified: 2010-09

Locations