International Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa

NCT ID: NCT00112359

Last Updated: 2011-04-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 166 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-05-31
• Study Completion Date: 2007-04-30

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).

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo three times a day (TID)

Group Type: PLACEBO_COMPARATOR

Placebo three times a day (TID)

Intervention Type: DRUG

AZLI 75 mg three times a day (TID)

Group Type: EXPERIMENTAL

AZLI 75 mg three times a day (TID)

Intervention Type: DRUG

Interventions

AZLI 75 mg three times a day (TID)

Intervention Type: DRUG

Placebo three times a day (TID)

Intervention Type: DRUG

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gilead Sciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Gilead Sciences, Inc.

Principal Investigators

Bruce Montgomery, MD

Role: STUDY_DIRECTOR

Corus Pharma, Inc.

Locations

University of Alabama at Birmingham

Birmingham, Alabama, United States

Pediatric Breathing Disorders Clinic

Anchorage, Alaska, United States

Phoenix Children's Hospital

Phoenix, Arizona, United States

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Miller Children's Hospital

Long Beach, California, United States

Children's Hospital, Los Angeles

Los Angeles, California, United States

Children's Hospital of Orange County

Orange, California, United States

Capital Allergy and Respiratory Disease Center

Sacramento, California, United States

Yale New Haven Hospital

New Haven, Connecticut, United States

University of Florida Health Sciences Center

Gainesville, Florida, United States

Nemours Children's Clinic, Orlando

Orlando, Florida, United States

Medical College of Georgia

Augusta, Georgia, United States

Children's Memorial Hospital / Northwestern University

Chicago, Illinois, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

University of Iowa

Iowa City, Iowa, United States

Via Christi - St. Francis Regional Medical Center

Wichita, Kansas, United States

Tulane University Health Sciences Center

New Orleans, Louisiana, United States

Louisiana State University Health Sciences Center

Shreveport, Louisiana, United States

Central Maine Pulmonary Associates

Auburn, Maine, United States

University of Michigan

Ann Arbor, Michigan, United States

University of Mississippi Medical Center

Jackson, Mississippi, United States

University of Missouri

Columbia, Missouri, United States

St. Louis University

St Louis, Missouri, United States

Children's Lung Specialists

Las Vegas, Nevada, United States

St. Barnabas Healthcare System

Livingston, New Jersey, United States

Albany Medical College

Albany, New York, United States

Long Island Jewish Medical Center

New Hyde Park, New York, United States

SUNY Upstate Medical University

Syracuse, New York, United States

University of North Carolina

Chapel Hill, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

University of Pennsylvania Health System

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Pediatric Pulmonary Associates, South Carolina

Columbia, South Carolina, United States

Baylor Martha Foster Lung Care Center

Dallas, Texas, United States

Alamo Clinical Research Associates

San Antonio, Texas, United States

University of Utah

Salt Lake City, Utah, United States

Naval Medical Center

Portsmouth, Virginia, United States

Pediatric Pulmonary Center/Virginia Commonwealth University

Richmond, Virginia, United States

University of Washington Medical Center

Seattle, Washington, United States

University of Wisconsin

Madison, Wisconsin, United States

Children's Hospital at Westmead

Westmead, New South Wales, Australia

Westmead Hospital

Westmead, New South Wales, Australia

Royal Children's Hospital

Herston, Queensland, Australia

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Alfred Hospital

Prahran, Victoria, Australia

Sir Charles Gairdner Hospital

Nedlands, Western Australia, Australia

Princess Margaret Hospital for Children

Perth, Western Australia, Australia

Capital Health and the Governors of the University of Alberta

Edmonton, Alberta, Canada

St. Paul's Hospital

Vancouver, British Columbia, Canada

Queen Elizabeth II Health Sciences Centre

Halifax, Nova Scotia, Canada

Brian Lyttle Professional Corporation

London, Ontario, Canada

Centre Hospitalier de l'Universite de Montreal (CHUM)

Montreal, Quebec, Canada

Auckland District Health Board

Auckland, , New Zealand

Countries

United States; Australia; Canada; New Zealand

References

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.

Reference Type: DERIVED

PMID: 19420195 (View on PubMed)

Other Identifiers

CP-AI-007

Identifier Type: -

Identifier Source: org_study_id