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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 (AZLI CAT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01641822
Recruitment Status : Completed
First Posted : July 17, 2012
Results First Posted : May 9, 2016
Last Update Posted : May 9, 2016
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: AZLI Drug: Placebo to match AZLI Drug: Tobramycin inhalation solution Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 107 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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
Study Start Date : December 2012
Actual Primary Completion Date : January 2015
Actual Study Completion Date : January 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
Active Comparator: AZLI
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.
 Drug: AZLI
Aztreonam for Inhalation Solution (AZLI) 75 mg 3 times daily combined with diluent administered using an eFlow nebulizer
Other Name: Cayston®

Drug: Tobramycin inhalation solution
Tobramycin inhalation solution (TIS) 300 mg 2 times daily using a PARI® LC Plus nebulizer and DeVilbiss Pulmo-Aide® air compressor
Other Name: TOBI®
Placebo Comparator: Placebo
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.
 Drug: Placebo to match AZLI
Placebo to match AZLI 3 times daily combined with diluent administered using an eFlow nebulizer

Drug: Tobramycin inhalation solution
Tobramycin inhalation solution (TIS) 300 mg 2 times daily using a PARI® LC Plus nebulizer and DeVilbiss Pulmo-Aide® air compressor
Other Name: TOBI®


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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 ]
    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.

  2. 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) ]
  3. 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) ]
    The time to first protocol-defined pulmonary exacerbation was calculated using the Kaplan-Meier method.

  4. 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) ]
    The rate of hospitalizations for a respiratory event per participant year was calculated using negative binomial regression analysis.

  5. 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 ]
    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.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01641822


Locations
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Sponsors and Collaborators
Gilead Sciences
Investigators
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Study Director: Mark Bresnik, MD Gilead Sciences
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT01641822    
Other Study ID Numbers: GS-US-205-0170
2015-000398-11 ( EudraCT Number )
First Posted: July 17, 2012    Key Record Dates
Results First Posted: May 9, 2016
Last Update Posted: May 9, 2016
Last Verified: April 2016
Additional relevant MeSH terms:
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Pseudomonas Infections
Cystic Fibrosis
Fibrosis
Infections
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Infant, Newborn, Diseases
Gram-Negative Bacterial Infections
Bacterial Infections
Bacterial Infections and Mycoses
Tobramycin
Anti-Bacterial Agents
Anti-Infective Agents