Cystic Fibrosis Clinical Trial
— AZLI CATOfficial title:
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy (CAT) Regimen of Inhaled Antibiotics for the Treatment of Chronic Pulmonary Pseudomonas Aeruginosa Infection in Subjects With Cystic Fibrosis
Verified date | April 2015 |
Source | Gilead Sciences |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
This study is to investigate whether using a continuous alternating therapy (CAT) regimen of
2 antibiotics of different classes and with different mechanisms of action may provide the
clinical benefits of reducing acute pulmonary exacerbations, maintaining lung function, and
controlling respiratory symptoms for cystic fibrosis (CF) patients while minimizing the risk
of emergence of antibiotic-resistant Pseudomonas aeruginosa (PA) strains.
After screening, eligible participants will be enrolled into the study and begin a 28-day
run in period of tobramycin inhalation solution (TIS) twice daily. After the run-in,
participants will return to the clinic and be randomized to either the Aztreonam for
Inhalation Solution (AZLI) arm or the placebo arm. The AZLI arm has 3 cycles of AZLI three
times daily for 28 days alternating with TIS twice daily for 28 days. The placebo arm has 3
cycles of placebo three times daily for 28 days alternating with 3 cycles of TIS twice daily
for 28 days. Participants will return to the clinic for evaluation after each cycle of
antibiotics for evaluation. There will be 9 scheduled study visits per participant.
Status | Completed |
Enrollment | 90 |
Est. completion date | January 2015 |
Est. primary completion date | January 2015 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 6 Years and older |
Eligibility |
Inclusion Criteria: - Confirmed diagnosis of CF - Presence of PA in 2 lower respiratory tract cultures in the 12 months prior to screening - FEV1 =25 and = 75% predicted - History of 1 hospitalization or 1 course of IV antibiotics for an acute respiratory exacerbation in the 12 months prior to screening Exclusion Criteria: - Concurrent use of oral, IV or inhaled antibiotics at enrollment - Concurrent hospitalization at enrollment - History of local or systemic hypersensitivity to monobactams or aminoglycoside antibiotics or history of aminoglycoside antibiotic associated toxicity |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Akron Children's Hospital | Akron | Ohio |
United States | Albany Medical College | Albany | New York |
United States | UNM Clinical and Translational Center | Albuquerque | New Mexico |
United States | Central Florida Pulmonary Group | Altamonte Springs | Florida |
United States | University of Michigan | Ann Arbor | Michigan |
United States | Emory Cystic Fibrosis Center | Atlanta | Georgia |
United States | Georgia Health | Augusta | Georgia |
United States | Austin Children's Chest Associates | Austin | Texas |
United States | Dartmouth Hitchcock Specialty Care Clinic | Bedford | New Hampshire |
United States | University of Alabama at Birmingham | Birmingham | Alabama |
United States | St. Lukes Medical Center | Boise | Idaho |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | Medical University of South Carolina | Charleston | South Carolina |
United States | University of Virginia | Charlottesville | Virginia |
United States | University of Chicago | Chicago | Illinois |
United States | Cincinnati Children's Hospital | Cincinnati | Ohio |
United States | UC Health - University of Cincinnati | Cincinnati | Ohio |
United States | Vermont Lung Center at the University of Vermont | Colchester | Vermont |
United States | UT Southwestern Medical Center | Dallas | Texas |
United States | Geisinger Clinic | Danville | Pennsylvania |
United States | National Jewish Health | Denver | Colorado |
United States | Harper University Hospital | Detroit | Michigan |
United States | Duke University Medical Center | Durham | North Carolina |
United States | Cook Children's Medical Center | Ft. Worth | Texas |
United States | University of Florida | Gainesville | Florida |
United States | Chicago CF Care Specialists NFP dba Cystic Fibrosis Institure | Glenview | Illinois |
United States | Spectrum Health - Helen DeVos Children's Hospital | Grand Rapids | Michigan |
United States | Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania |
United States | South Broward Hospital dba Memorial Healthcare System | Hollywood | Florida |
United States | Indiana University | Indianapolis | Indiana |
United States | Riley Hospital for Children | Indianapolis | Indiana |
United States | University of Mississippi Medical Center | Jackson | Mississippi |
United States | Nemours Children's Clinic - Jacksonville | Jacksonville | Florida |
United States | University of Kansas Medical Center | Kansas City | Kansas |
United States | East Tennessee Children's Hospital | Knoxville | Tennessee |
United States | University of Tennessee Medical Center | Knoxville | Tennessee |
United States | University of California - San Diego | La Jolla | California |
United States | Children's Lung Specialists | Las Vegas | Nevada |
United States | Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire |
United States | Children's Hospital Los Angeles | Los Angeles | California |
United States | Children's Foundation Research Institute/UTHSC | Memphis | Tennessee |
United States | University of Miami | Miami | Florida |
United States | Children's Hospital of Wisconsin | Milwaukee | Wisconsin |
United States | West Virginia University | Morgantown | West Virginia |
United States | Morristown Medical Center | Morristown | New Jersey |
United States | Vanderbilt Children's Hospital | Nashville | Tennessee |
United States | Long Island Jewish Medical Center - Adult CF & Bronchiectasis Center | New Hyde Park | New York |
United States | Gunnar Esiason Adult CF and Lung Program | New York | New York |
United States | Children's Hospital of the King's Daughters | Norfolk | Virginia |
United States | Kaiser Permanente Medical Center | Oakland | California |
United States | OU Health Sciences Center | Oklahoma City | Oklahoma |
United States | Santiago Reyes, MD | Oklahoma City | Oklahoma |
United States | University of Nebraska Medical Center | Omaha | Nebraska |
United States | Arnold Palmer Hospital for Children | Orlando | Florida |
United States | Nemours Children's Clinic - Orlando | Orlando | Florida |
United States | Nemour's Children's Clinic | Pensacola | Florida |
United States | Drexel University College of Medicine | Philadelphia | Pennsylvania |
United States | University of Pennsylvania | Philadelphia | Pennsylvania |
United States | Phoenix Children's Hospital | Phoenix | Arizona |
United States | Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Maine Medical Center | Portland | Maine |
United States | VCU Children's Hospital | Richmond | Virginia |
United States | University of Utah | Salt Lake City | Utah |
United States | Alamo Clinical Research Associates | San Antonio | Texas |
United States | St. Louis University - Cardinal Glennon Children's Hospital | St. Louis | Missouri |
United States | All Children's Hospital | St. Petersburg | Florida |
United States | SUNY Upstate University | Syracuse | New York |
United States | Tampa General Hospital | Tampa | Florida |
United States | The Toledo Hospital/Toledo Children's Hospital CF Center | Toledo | Ohio |
United States | Children's National Medical Center | Washington DC | District of Columbia |
United States | Alfred I. duPont Hospital for Children | Wilmington | Delaware |
Lead Sponsor | Collaborator |
---|---|
Gilead Sciences |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Rate of protocol-defined exacerbations from baseline through Week 24 | 24 weeks | No | |
Secondary | Average actual change from baseline in forced expiratory volume in 1 second (FEV1) % predicted at the end of each course of study drug | FEV1 % predicted is defined as FEV1 % of the patient divided by the average FEV1 % in the population for any person of similar age, sex and body composition. | Weeks 4, 12 and 20 | No |
Secondary | Time to first protocol-defined pulmonary exacerbation | The time to first protocol-defined pulmonary exacerbation will be summarized using Kaplan-Meier (KM) summary statistics and analyzed using the log-rank test. | Baseline to Week 24 | No |
Secondary | Rate of hospitalizations for a respiratory event | The rate of respiratory hospitalizations will be summarized and analyzed using negative binomial regression methods similar to the primary endpoint analysis. Total number of events will also be presented. | Baseline to Week 24 | No |
Secondary | Average change from baseline in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Symptom Scale (RSS) score at the end of each course of study drug (Weeks 4, 12, and 20) | Baseline to Weeks 4, 12, and 20 | No | |
Secondary | Percent of subjects who use non-study IV or inhaled antibiotics for protocol-defined pulmonary exacerbations through Week 24 | Baseline to Week 24 | No |
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