Cystic FibrosisPhase 3AZLI CATNCT01641822

Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF

A Phase 3 study testing AZLI and Tobramycin inhalation solution in people with Cystic Fibrosis, run by Gilead Sciences at 71 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 6 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: AZLI and Tobramycin inhalation solution
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 107 participants
Where: 71 sitesUnited States
Study dates: Dec 2012to Jan 2015
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

Confirmed diagnosis of CF
Presence of PA in 2 lower respiratory tract cultures in the 12 months prior to screening
Forced expiratory volume (FEV)1 ≥ 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

Who cannot join

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

Trial locations and contacts

71 sites in 1 country.

City, postcode or hospital

71 sites in 1 country

United States71 sites

Akron Children's Hospital
Akron, Ohio, 44308
Albany Medical College
Albany, New York, 12208
UNM Clinical and Translational Center
Albuquerque, New Mexico, 87131
Central Florida Pulmonary Group
Altamonte Springs, Florida, 32701
University of Michigan
Ann Arbor, Michigan, 48109
Emory Cystic Fibrosis Center
Atlanta, Georgia, 30084
Georgia Health
Augusta, Georgia, 30912
Austin Children's Chest Associates
Austin, Texas, 78723
Dartmouth Hitchcock Specialty Care Clinic
Bedford, New Hampshire, 03110
University of Alabama at Birmingham
Birmingham, Alabama, 35233
St. Lukes Medical Center
Boise, Idaho, 37012
Boston Children's Hospital
Boston, Massachusetts, 02115
Medical University of South Carolina
Charleston, South Carolina, 29425
University of Virginia
Charlottesville, Virginia, 22908
University of Chicago
Chicago, Illinois, 60637
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
UC Health - University of Cincinnati
Cincinnati, Ohio, 45267
Vermont Lung Center at the University of Vermont
Colchester, Vermont, 05446
UT Southwestern Medical Center
Dallas, Texas, 75390
Geisinger Clinic
Danville, Pennsylvania, 17822
National Jewish Health
Denver, Colorado, 80206
Harper University Hospital
Detroit, Michigan, 48201
Duke University Medical Center
Durham, North Carolina, 27710
Cook Children's Medical Center
Fort Worth, Texas, 76104
University of Florida
Gainesville, Florida, 32610
Chicago CF Care Specialists NFP dba Cystic Fibrosis Institure
Glenview, Illinois, 60025
Spectrum Health - Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
South Broward Hospital dba Memorial Healthcare System
Hollywood, Florida, 33021
Indiana University
Indianapolis, Indiana, 46202
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Nemours Children's Clinic - Jacksonville
Jacksonville, Florida, 32207
University of Kansas Medical Center
Kansas City, Kansas, 66160
East Tennessee Children's Hospital
Knoxville, Tennessee, 37916
University of Tennessee Medical Center
Knoxville, Tennessee, 37920
University of California - San Diego
La Jolla, California, 92093
Children's Lung Specialists
Las Vegas, Nevada, 89107
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Children's Hospital Los Angeles
Los Angeles, California, 90027
Children's Foundation Research Institute/UTHSC
Memphis, Tennessee, 28103
University of Miami
Miami, Florida, 33136
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53201
West Virginia University
Morgantown, West Virginia, 26506
Morristown Medical Center
Morristown, New Jersey, 07962
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232
Long Island Jewish Medical Center - Adult CF & Bronchiectasis Center
New Hyde Park, New York, 11042
Gunnar Esiason Adult CF and Lung Program
New York, New York, 10032
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510
Kaiser Permanente Medical Center
Oakland, California, 94611
OU Health Sciences Center
Oklahoma City, Oklahoma, 73104
Santiago Reyes, MD
Oklahoma City, Oklahoma, 73112
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Nemours Children's Clinic - Orlando
Orlando, Florida, 32801
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Nemour's Children's Clinic
Pensacola, Florida, 32504
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19107
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Maine Medical Center
Portland, Maine, 38103
VCU Children's Hospital
Richmond, Virginia, 23298
University of Utah
Salt Lake City, Utah, 84132
Alamo Clinical Research Associates
San Antonio, Texas, 78212
St. Louis University - Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104
All Children's Hospital
St. Petersburg, Florida, 33701
SUNY Upstate University
Syracuse, New York, 13210
Tampa General Hospital
Tampa, Florida, 33606
The Toledo Hospital/Toledo Children's Hospital CF Center
Toledo, Ohio, 43606
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803

Other trials in United States

About this study

Official 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

The primary objective of this study is to evaluate the safety and efficacy of a CAT regimen with aztreonam for inhalation solution (AZLI) and tobramycin inhalation solution (TIS) in adult and pediatric subjects with cystic fibrosis (CF) and pulmonary Pseudomonas aeruginosa (PA) infection. Participants will be enrolled in a 28 day TIS run-in phase, and will be eligible for randomization in the comparative phase if they have not received non-study oral antibiotics for a respiratory event, or IV or inhaled antibiotics for any indication between Visits 2 and 3, have not developed a condition requiring hospitalization or other change in clinical status which, in the opinion of the investigator would preclude their ability to continue in the study, and have demonstrated at least 50% TIS compliance. Participants enrolled in the comparative phase will be randomized to receive 3 cycles of treatment, each cycle consisting alternating regimens: AZLI or placebo for 28 days followed by TIS for 28 days.

Conditions: Cystic Fibrosis
Being tested: AZLI, Placebo to match AZLI, Tobramycin inhalation solution
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AZLIDrug
Also called Cayston®
Aztreonam for Inhalation Solution (AZLI) 75 mg 3 times daily combined with diluent administered using an eFlow nebulizer
Placebo to match AZLIDrug
Placebo to match AZLI 3 times daily combined with diluent administered using an eFlow nebulizer
Tobramycin inhalation solutionDrug
Also called TOBI®
Tobramycin inhalation solution (TIS) 300 mg 2 times daily using a PARI® LC Plus nebulizer and DeVilbiss Pulmo-Aide® air compressor

Study groups in NCT01641822
Study group	Role	What they receive
AZLI	Active comparator	Participants will be randomized to receive 3 cycles of treatment, each cycle consisting alternating regimens: AZLI for 28 days followed by TIS for 28 days. Receives: Drug: AZLI; Drug: Tobramycin inhalation solution
Placebo	Placebo comparator	Participants will be randomized to receive 3 cycles of treatment, each cycle consisting alternating regimens: placebo to match AZLI for 28 days followed by TIS for 28 days. Receives: Drug: Placebo to match AZLI; Drug: Tobramycin inhalation solution

What is being measured

The primary outcome is the main question the study is designed to answer.

Rate of Protocol-defined Exacerbations (PDE) From Baseline Through Week 24
PDEs were characterized by a change or worsening from baseline of 1 or more documented signs or symptoms (decreased exercise tolerance, increased cough, increased sputum or chest congestion, decreased appetite, or other signs or symptoms) associated with the use of non-study IV or inhaled antibiotics and be verified by a blinded independent adjudication committee.
Measured: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)

Secondary outcomes (5)

Average Actual Change From Baseline in FEV1 % Predicted Across All Courses of AZLI/Placebo Treatment (Weeks 4, 12 and 20)
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. The adjusted mean is from a mixed-effect model repeated measures (MMRM) analysis. The model includes terms for baseline value, previous exacerbations (1, 2, ≥ 3), treatment, visit (categorical), and treatment by visit interaction.
Measured: Comparative Phase: Baseline and Weeks 4, 12 and 20
Percentage of Participants Who Used Non-study IV or Inhaled Antibiotics for PDEs
Measured: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)
Time to First Protocol-defined Pulmonary Exacerbation
The time to first protocol-defined pulmonary exacerbation was calculated using the Kaplan-Meier method.
Measured: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)
Rate of Hospitalizations for a Respiratory Event
The rate of hospitalizations for a respiratory event per participant year was calculated using negative binomial regression analysis.
Measured: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)
Average Change From Baseline in the CFQ-R Respiratory Symptom Scale (RSS) Score Across All Courses of AZLI/Placebo Treatment (Weeks 4, 12 and 20)
Respiratory symptoms (eg, coughing, congestion, wheezing) were assessed with the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Symptoms Scale (RSS). The range of scores (units) was 0 to 100 with higher scores indicating fewer symptoms. The adjusted mean is from a mixed-effect model repeated measures (MMRM) analysis. The model includes terms for baseline value, previous exacerbations (1, 2, ≥ 3), treatment, visit (categorical), and treatment by visit interaction.
Measured: Comparative Phase: Baseline and Weeks 4, 12 and 20

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2012Jan 2015

First posted17 Jul 2012 (estimated)
Study startDec 2012
Primary completionJan 2015
Study completionJan 2015

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: Double blind
Who is masked: Participant, Investigator
Enrolment: 107 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01641822
Sponsor's protocol ID: GS-US-205-0170
Other IDs: 2015-000398-11, 2015
Acronym: AZLI CAT
Status last verified: Apr 2016
First submitted: 13 Jul 2012
Last updated: 9 May 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Flume PA, Clancy JP, Retsch-Bogart GZ, Tullis DE, Bresnik M, Derchak PA, Lewis SA, Ramsey BW. Continuous alternating inhaled antibiotics for chronic pseudomonal infection in cystic fibrosis. J Cyst Fibros. 2016 Nov;15(6):809-815. doi: 10.1016/j.jcf.2016.05.001. Epub 2016 May 24.
PubMed 27233377

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 9 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official 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
ClinicalTrials.gov ID: NCT01641822
Acronym / sponsor ID: AZLI CAT / GS-US-205-0170
Conditions: Cystic Fibrosis
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 107 participants (actual)
Lead sponsor: Gilead SciencesIndustry (pharma, biotech, devices)
Study start: Dec 2012
Primary completion: Jan 2015 (actual)
Study completion: Jan 2015 (actual)
First posted: 17 Jul 2012 (estimated)
Results: Posted 9 May 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
AZLI	Active comparator	Drug: AZLI, Drug: Tobramycin inhalation solutionParticipants will be randomized to receive 3 cycles of treatment, each cycle consisting alternating regimens: AZLI for 28 days followed by TIS for 28 days.
Placebo	Placebo comparator	Drug: Placebo to match AZLI, Drug: Tobramycin inhalation solutionParticipants will be randomized to receive 3 cycles of treatment, each cycle consisting alternating regimens: placebo to match AZLI for 28 days followed by TIS for 28 days.

Who may qualify

Ages: 6 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

Confirmed diagnosis of CF
Presence of PA in 2 lower respiratory tract cultures in the 12 months prior to screening
Forced expiratory volume (FEV)1 ≥ 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

Key exclusion criteria (3 listed)

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

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
AZLIAlso: Cayston®Aztreonam for Inhalation Solution (AZLI) 75 mg 3 times daily combined with diluent administered using an eFlow nebulizer	Drug	AZLI
Placebo to match AZLIPlacebo to match AZLI 3 times daily combined with diluent administered using an eFlow nebulizer	Drug	Placebo
Tobramycin inhalation solutionAlso: TOBI®Tobramycin inhalation solution (TIS) 300 mg 2 times daily using a PARI® LC Plus nebulizer and DeVilbiss Pulmo-Aide® air compressor	Drug	AZLI, Placebo

Primary outcome

Rate of Protocol-defined Exacerbations (PDE) From Baseline Through Week 24
Time frame: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)
PDEs were characterized by a change or worsening from baseline of 1 or more documented signs or symptoms (decreased exercise tolerance, increased cough, increased sputum or chest congestion, decreased appetite, or other signs or symptoms) associated with the use of non-study IV or inhaled antibiotics and be verified by a blinded independent adjudication committee.

5 secondary outcomes

Average Actual Change From Baseline in FEV1 % Predicted Across All Courses of AZLI/Placebo Treatment (Weeks 4, 12 and 20)
Time frame: Comparative Phase: Baseline and Weeks 4, 12 and 20
Percentage of Participants Who Used Non-study IV or Inhaled Antibiotics for PDEs
Time frame: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)
Time to First Protocol-defined Pulmonary Exacerbation
Time frame: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)
Rate of Hospitalizations for a Respiratory Event
Time frame: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)
Average Change From Baseline in the CFQ-R Respiratory Symptom Scale (RSS) Score Across All Courses of AZLI/Placebo Treatment (Weeks 4, 12 and 20)
Time frame: Comparative Phase: Baseline and Weeks 4, 12 and 20

Results and safety

Results posted to the registry on 9 May 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
TIS Run-In Treatment Group	107	93
AZLI	0	0
Placebo	0	0

Primary outcome: Rate of Protocol-defined Exacerbations (PDE) From Baseline Through Week 24

Time frame: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days). Number. Unit: PDEs per participant year

Measure	AZLI n = 43	Placebo n = 47
Value	1.309	1.762

AZLI vs Placebo: Risk Ratio (RR): 0.743 (95% CI 0.446 to 1.238), p = 0.25, Negative binomial regression

Population analysed: Intent-to-Treat (ITT) Analysis Set: all randomized participants

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: TIS Run-In Treatment Group: up to 28 days (plus 30 days if not continuing to the comparative phase). Comparative phase: from first dose of AZLI or Placebo through 30 days after last dose (average 155.4 days).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
TIS Run-In Treatment Group	4 of 107 (3.7%)	27 of 107 (25.2%)	Not reported
AZLI	21 of 42 (50%)	38 of 42 (90.5%)	Not reported
Placebo	24 of 46 (52.2%)	44 of 46 (95.7%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 27233377Flume PA, Clancy JP, Retsch-Bogart GZ, Tullis DE, Bresnik M, Derchak PA, Lewis SA, Ramsey BW. Continuous alternating inhaled antibiotics for chronic pseudomonal infection in cystic fibrosis. J Cyst Fibros. 2016 Nov;15(6):809-815. doi: 10.1016/j.jcf.2016.05.001. Epub 2016 May 24.

Related trials for Cystic Fibrosis

RecruitingPhase 2
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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.

Mark Bresnik, MD study director

Gilead Sciences

71 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing AZLI and Tobramycin inhalation solution in people with Cystic Fibrosis, run by Gilead Sciences at 71 sites in United States.

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 4 inclusion and 3 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 9 May 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 71 sites in United States. 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 9 May 2016). 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?

Rate of Protocol-defined Exacerbations (PDE) From Baseline Through Week 24 (time frame: Baseline in the comparative phase to the end of study (average time on study during the Comparative Phase: 155.4 days)). 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 NCT01641822, 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 9 May 2016.