Evaluation of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) in Patients With Bronchiectasis
Primary Purpose
Bronchiectasis
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
AZLI
Sponsored by
About this trial
This is an interventional treatment trial for Bronchiectasis
Eligibility 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)
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
AZLI
Arm Description
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.
Outcomes
Primary Outcome Measures
Reliability of the Respiratory Domain of the Quality of Life Questionnaire-Bronchiectasis (QOL-B)
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
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)
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00805025
Brief Title
Evaluation of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) in Patients With Bronchiectasis
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2014
Overall Recruitment Status
Completed
Study Start Date
December 2008 (undefined)
Primary Completion Date
October 2009 (Actual)
Study Completion Date
October 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gilead Sciences
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Bronchiectasis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
89 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AZLI
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
AZLI
Other Intervention Name(s)
Cayston®
Intervention Description
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
Primary Outcome Measure Information:
Title
Reliability of the Respiratory Domain of the Quality of Life Questionnaire-Bronchiectasis (QOL-B)
Description
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.
Time Frame
Day -14 to Day 0
Title
Convergent Validity of the Respiratory Domain of the QOL-B
Description
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.
Time Frame
Day -14
Secondary Outcome Measure Information:
Title
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)
Description
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.
Time Frame
Day 0 to Day 28
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Steve Lamola, MD
Organizational Affiliation
Gilead Sciences
Official's Role
Study Director
Facility Information:
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35209
Country
United States
City
Peoria
State/Province
Arizona
ZIP/Postal Code
85381
Country
United States
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
City
Farmington
State/Province
Connecticut
ZIP/Postal Code
06030
Country
United States
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06519
Country
United States
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
City
Marietta
State/Province
Georgia
ZIP/Postal Code
30060
Country
United States
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
City
Olathe
State/Province
Kansas
ZIP/Postal Code
66061
Country
United States
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
City
Chesterfield
State/Province
Missouri
ZIP/Postal Code
63017
Country
United States
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
City
Tyler
State/Province
Texas
ZIP/Postal Code
75708
Country
United States
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
24626872
Citation
Quittner AL, Marciel KK, Salathe MA, O'Donnell AE, Gotfried MH, Ilowite JS, Metersky ML, Flume PA, Lewis SA, McKevitt M, Montgomery AB, O'Riordan TG, Barker AF. A preliminary quality of life questionnaire-bronchiectasis: a patient-reported outcome measure for bronchiectasis. Chest. 2014 Aug;146(2):437-448. doi: 10.1378/chest.13-1891. Erratum In: Chest. 2014 Nov;146(5):1422.
Results Reference
derived
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Evaluation of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) in Patients With Bronchiectasis
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