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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AZLI | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AZLI | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Day -14 to Day 0 |
| 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. | Day -14 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Steve Lamola, MD | Gilead Sciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | 35209 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24626872 | Derived | 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. |
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131 participants were screened and 89 were enrolled and treated.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | AZLI | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Day 0 to Day 28 |
| Peoria |
| Arizona |
| 85381 |
| United States |
| Phoenix | Arizona | 85006 | United States |
| Orange | California | 92868 | United States |
| Denver | Colorado | 80206 | United States |
| Farmington | Connecticut | 06030 | United States |
| New Haven | Connecticut | 06519 | United States |
| Washington D.C. | District of Columbia | 20007 | United States |
| Miami | Florida | 33136 | United States |
| Atlanta | Georgia | 30342 | United States |
| Marietta | Georgia | 30060 | United States |
| Chicago | Illinois | 60637 | United States |
| Olathe | Kansas | 66061 | United States |
| Boston | Massachusetts | 02118 | United States |
| Chesterfield | Missouri | 63017 | United States |
| Mineola | New York | 11501 | United States |
| New York | New York | 10032 | United States |
| Chapel Hill | North Carolina | 27599 | United States |
| Portland | Oregon | 97239 | United States |
| Philadelphia | Pennsylvania | 19104 | United States |
| Charleston | South Carolina | 29425 | United States |
| Tyler | Texas | 75708 | United States |
| Charlottesville | Virginia | 22908 | United States |
| Madison | Wisconsin | 53792 | United States |
| Enrolled and Treated |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
Enrolled and treated participants
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| ID | Title | Description |
|---|---|---|
| BG000 | AZLI | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Participants who were treated and had any QOL-B measurements at both Screening and Day 0 (Visits 1 and 2) were analyzed. | Posted | Number | Intraclass Correlation Coefficient | Day -14 to Day 0 |
|
|
| ||||||||||||||||||||||||||
| Primary | 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. | 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. | Posted | Number | Pearson correlation coefficient | Day -14 |
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| Secondary | 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. | 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. | Posted | Mean | Standard Deviation | units on a scale | Day 0 to Day 28 |
|
Pre-baseline through 70 days
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | AZLI | 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. | 4 | 89 | 36 | 89 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | MedDRA (11.1) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (11.1) | Systematic Assessment |
| |
| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (11.1) | Systematic Assessment |
| |
| Pelvic fracture | Injury, poisoning and procedural complications | MedDRA (11.1) | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (11.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (11.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (11.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (11.1) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (11.1) | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (11.1) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (11.1) | Systematic Assessment |
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| Fatigue | General disorders | MedDRA (11.1) | Systematic Assessment |
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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:
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Disclosures | Gilead Sciences, Inc. | ClinicalTrialDisclosures@gilead.com |
| ID | Term |
|---|---|
| D001987 | Bronchiectasis |
| ID | Term |
|---|---|
| D001982 | Bronchial Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| D001398 | Aztreonam |
| ID | Term |
|---|---|
| D008997 | Monobactams |
| D047090 | beta-Lactams |
| D007769 | Lactams |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D013457 | Sulfur Compounds |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
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