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Evaluation of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) in Patients With Bronchiectasis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences
ClinicalTrials.gov Identifier:
NCT00805025
First received: December 5, 2008
Last updated: February 3, 2014
Last verified: February 2014
  Purpose

Over the 70-day study period, eligible patients visited the study clinic every 2 weeks (total of 6 visits) and received a 28-day course of aztreonam for inhalation solution (AZLI). The Quality of Life-Bronchiectasis (QOL-B) questionnaire was completed at several time points during the study, in additional to pulmonary function testing and other standard procedures.


Condition Intervention Phase
Bronchiectasis
Drug: AZLI
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter Trial to Validate the Quality of Life Questionnaire-Bronchiectasis and to Evaluate Perception of Symptom Improvement Following One Course of Aztreonam for Inhalation Solution (AZLI) in Subjects With Bronchiectasis and Gram-negative Bacteria in the Airways

Resource links provided by NLM:


Further study details as provided by Gilead Sciences:

Primary Outcome Measures:
  • Reliability of the Respiratory Domain of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) [ Time Frame: Day -14 to Day 0 ] [ Designated as safety issue: No ]

    Test-retest reliability is a measure of the stability or reproducibility of a measure over a period of time during which status on the underlying construct has not changed, and is measured by the intraclass correlation of scores obtained at 2 time points within that period. Test-retest reliability of respiratory symptoms was calculated for response at Day -14 and Day 0. Reliability of the participants' QOL-B responses was assessed from an Intraclass Correlation Coefficient (ICC). A score of ≥ 0.70 would indicate strong reliability.

    The QOL-B respiratory symptoms score was transformed onto a scale of 0-100, with higher scores representing a better quality of life.


  • Convergent Validity of the Respiratory Domain of the QOL-B [ Time Frame: Day -14 ] [ Designated as safety issue: No ]
    Convergent validity was assessed at Day -14 by examining the correlations between relevant QOL-B domains and other indicators of health status: a bronchiectasis severity score based on high-resolution computerised tomography (HRCT) scan results, forced expiratory volume in 1 second (FEV1) percent predicted, 6-minute walk test (6MWT) results, and St. George's Respiratory Questionnaire (SGRQ) symptoms scores. Correlations with absolute values of 0.30 to 0.50 indicated moderate evidence of convergent validity.


Secondary Outcome Measures:
  • Responsiveness of the Respiratory Domain of the QOL-B as Assessed by the Anchor-based Minimal Clinically Important Difference (MCID) Following Categorization of Level of Change Using the Global Rating of Change Questionnaire (GRCQ) [ Time Frame: Day 0 to Day 28 ] [ Designated as safety issue: No ]

    The anchor-based method measured the participant's perception of change at Day 28 using the GRCQ, which assesses improving/worsening symptoms. Distribution of ratings was categorized as no change (-1 to 1), minimal change (≥ 1.1 to < 3.1 or ≤ -1.1 to > -3.1), moderate change (≥ 3.1 to < 5.1 or ≤ -3.1 to > -5.1) or large change (≥ 5.1, ≤ -5.1). The GRCQ evaluated change in respiratory symptoms on a visual analog scale from -7 (worsening) to +7 (improvement). The following algorithm was used to obtain the mean change:

    If the corresponding GRCQ score was in the "no change" group, then change from baseline QOL-B = Observed QOL-B change from baseline score; if > 1, then change from baseline QOL-B score = Observed QOL-B change from baseline score; if < -1, the change from baseline QOL-B score = (-1) * Observed QOL-B change from baseline score.

    Then the mean change from baseline of QOL-B respiratory symptoms score of the minimal change category group is the anchor-based MCID.



Enrollment: 89
Study Start Date: December 2008
Study Completion Date: October 2009
Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AZLI
Participants were evaluated beginning 14 days prior to starting a 28-day course of AZLI (Day 0 to Day 28), followed by post-treatment assessments every 14 days through Day 56, for a total of 70 days of participation in the study.
Drug: AZLI
75 mg aztreonam for inhalation solution (AZLI), administered 3 times daily using the PARI Investigational eFlow® Nebulizer System (with a minimum of 4 hours between doses) following administration of a short-acting bronchodilator
Other Name: Cayston®

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written informed consent prior to any study-related procedures
  • Ability to read and understand the English language
  • Bronchiectasis confirmed by CT scan of the chest
  • Previous treatment with antibiotics for bronchiectasis
  • Documented history of positive sputum culture for a gram-negative organism within 5 years
  • Positive sputum culture for a gram-negative organism at first visit (Day -14)

Exclusion Criteria:

  • Hospitalization or hemoptysis > 30 mL within 14 days of first visit (Day -14)
  • Antibiotic use for respiratory symptoms within 14 days of first visit (Day -14), excluding chronic, stable azithromycin use
  • Change in corticosteroid or bronchodilator regimen within 14 days of first visit (Day -14)
  • Forced expiratory volume in 1 second (FEV1) < 25% predicted approximately 15 minutes following use of a bronchodilator at first visit (Day -14)
  • Cigarette smoking within 6 months of first visit (Day -14)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00805025

  Hide Study Locations
Locations
United States, Alabama
Birmingham, Alabama, United States, 35209
United States, Arizona
Peoria, Arizona, United States, 85381
Phoenix, Arizona, United States, 85006
United States, California
Orange, California, United States, 92868
United States, Colorado
Denver, Colorado, United States, 80206
United States, Connecticut
Farmington, Connecticut, United States, 06030
New Haven, Connecticut, United States, 06519
United States, District of Columbia
Washington, District of Columbia, United States, 20007
United States, Florida
Miami, Florida, United States, 33136
United States, Georgia
Atlanta, Georgia, United States, 30342
Marietta, Georgia, United States, 30060
United States, Illinois
Chicago, Illinois, United States, 60637
United States, Kansas
Olathe, Kansas, United States, 66061
United States, Massachusetts
Boston, Massachusetts, United States, 02118
United States, Missouri
Chesterfield, Missouri, United States, 63017
United States, New York
Mineola, New York, United States, 11501
New York, New York, United States, 10032
United States, North Carolina
Chapel Hill, North Carolina, United States, 27599
United States, Oregon
Portland, Oregon, United States, 97239
United States, Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Charleston, South Carolina, United States, 29425
United States, Texas
Tyler, Texas, United States, 75708
United States, Virginia
Charlottesville, Virginia, United States, 22908
United States, Wisconsin
Madison, Wisconsin, United States, 53792
Sponsors and Collaborators
Gilead Sciences
Investigators
Study Director: Steve Lamola, MD Gilead Sciences
  More Information

No publications provided

Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT00805025     History of Changes
Other Study ID Numbers: GS-US-219-0102
Study First Received: December 5, 2008
Results First Received: September 9, 2013
Last Updated: February 3, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Bronchiectasis
Bronchial Diseases
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on November 23, 2014