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

Bronchiectasis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00805025
• Other study ID #: GS-US-219-0102
• Status: Completed
• Phase: Phase 2
• First received: December 5, 2008
• Last updated: February 3, 2014
• Start date: December 2008
• Est. completion date: October 2009

Study information

• Verified date: February 2014
• Source: Gilead Sciences
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 89
• Est. completion date: October 2009
• Est. primary completion date: October 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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)

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Bronchiectasis

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Gilead Sciences

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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.	Day -14 to Day 0	No
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.	Day -14	No
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.	Day 0 to Day 28	No