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

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ClinicalTrials.gov Identifier: NCT00805025
Recruitment Status : Completed
First Posted : December 9, 2008
Results First Posted : March 20, 2014
Last Update Posted : March 20, 2014
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Bronchiectasis
Intervention Drug: AZLI
Enrollment 89
Recruitment Details Participants were enrolled at a total of 21 study sites in the United States. The first participant was screened on 08 December 2008. The last participant observation was on 14 October 2009.
Pre-assignment Details 131 participants were screened and 89 were enrolled and treated.
Arm/Group Title AZLI
Hide Arm/Group Description Participants were evaluated beginning 14 days prior to starting a 28-day course of aztreonam for inhalation solution (AZLI) 75 mg three times daily via investigational nebulizer (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.
Period Title: Overall Study
Started 131
Enrolled and Treated 89
Completed 82
Not Completed 49
Reason Not Completed
Screened but not enrolled/treated             42
Adverse Event             4
Withdrawal by Subject             2
Physician Decision             1
Arm/Group Title AZLI
Hide Arm/Group Description Participants were evaluated beginning 14 days prior to starting a 28-day course of AZLI 75 mg three times daily via investigational nebulizer (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.
Overall Number of Baseline Participants 89
Hide Baseline Analysis Population Description
Enrolled and treated participants
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 89 participants
64  (14.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 89 participants
Female
62
  69.7%
Male
27
  30.3%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 89 participants
Asian 2
African-American 3
White 84
1.Primary Outcome
Title Reliability of the Respiratory Domain of the Quality of Life Questionnaire-Bronchiectasis (QOL-B)
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who were treated and had any QOL-B measurements at both Screening and Day 0 (Visits 1 and 2) were analyzed.
Arm/Group Title AZLI
Hide Arm/Group Description:
Participants were evaluated beginning 14 days prior to starting a 28-day course of AZLI 75 mg three times daily via investigational nebulizer (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.
Overall Number of Participants Analyzed 86
Measure Type: Number
Unit of Measure: Intraclass Correlation Coefficient
0.80
2.Primary Outcome
Title Convergent Validity of the Respiratory Domain of the QOL-B
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants were analyzed for respiratory domain score at Day 0 against each of the other variables; those with data for both the respiratory domain score and each variable are reported.
Arm/Group Title AZLI
Hide Arm/Group Description:
Participants were evaluated beginning 14 days prior to starting a 28-day course of AZLI 75 mg three times daily via investigational nebulizer (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.
Overall Number of Participants Analyzed 131
Measure Type: Number
Unit of Measure: Pearson correlation coefficient
HRCT overall sentisitivity (n = 118) -0.19
FEV1 % predicted (n = 119) 0.11
6MWT (n = 121) 0.16
SGRQ symptoms (n = 121) -0.75
3.Secondary Outcome
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)
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who were evaluable for MCID and had both GRCQ and QOL-B change values at Day 28 (Visit 4) in any GRCQ domain were analyzed.
Arm/Group Title AZLI
Hide Arm/Group Description:
Participants were evaluated beginning 14 days prior to starting a 28-day course of AZLI 75 mg three times daily via investigational nebulizer (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.
Overall Number of Participants Analyzed 83
Mean (Standard Deviation)
Unit of Measure: units on a scale
2.9  (10.2)
Time Frame Pre-baseline through 70 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title AZLI
Hide Arm/Group Description Participants were evaluated beginning 14 days prior to starting a 28-day course of AZLI 75 mg three times daily via investigational nebulizer (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.
All-Cause Mortality
AZLI
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
AZLI
Affected / at Risk (%)
Total   4/89 (4.49%) 
Injury, poisoning and procedural complications   
Pelvic fracture  1  1/89 (1.12%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/89 (1.12%) 
Respiratory, thoracic and mediastinal disorders   
Bronchiectasis  1  1/89 (1.12%) 
Chronic obstructive pulmonary disease  1  1/89 (1.12%) 
Lung disorder  1  1/89 (1.12%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (11.1)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
AZLI
Affected / at Risk (%)
Total   36/89 (40.45%) 
Gastrointestinal disorders   
Diarrhoea  1  8/89 (8.99%) 
General disorders   
Pyrexia  1  7/89 (7.87%) 
Fatigue  1  6/89 (6.74%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  10/89 (11.24%) 
Dyspnoea  1  9/89 (10.11%) 
Productive cough  1  8/89 (8.99%) 
Wheezing  1  8/89 (8.99%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (11.1)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met:

  • The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or
  • The study has been completed at all study sites for at least 2 years
Results Point of Contact
Name/Title: Clinical Trial Disclosures
Organization: Gilead Sciences, Inc.
Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT00805025     History of Changes
Other Study ID Numbers: GS-US-219-0102
First Submitted: December 5, 2008
First Posted: December 9, 2008
Results First Submitted: September 9, 2013
Results First Posted: March 20, 2014
Last Update Posted: March 20, 2014