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

• 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: Gilead Sciences
• Information provided by (Responsible Party): Gilead Sciences

Tracking Information

• First Submitted Date: December 5, 2008
• First Posted Date: December 9, 2008
• Results First Submitted Date: September 9, 2013
• Results First Posted Date: March 20, 2014
• Last Update Posted Date: March 20, 2014
• Study Start Date: December 2008
• Actual Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 3, 2014)

• Reliability of the Respiratory Domain of the Quality of Life Questionnaire-Bronchiectasis (QOL-B) [ Time Frame: Day -14 to Day 0 ]
  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 ]
  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.

• Original Primary Outcome Measures (submitted: December 8, 2008): The primary objective is to determine the reliability and validity of the QOL-B. [ Time Frame: 70 days ]

Current Secondary Outcome Measures (submitted: February 3, 2014)

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 ]

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.

• Original Secondary Outcome Measures (submitted: December 8, 2008): The secondary objective is to evaluate the responsiveness of the QOL-B to perceived changes in respiratory symptoms. [ Time Frame: 70 days ]
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: 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

Other Name: Cayston®

Study Arms

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.

Intervention: Drug: AZLI

• Publications *: 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. Review. Erratum in: Chest. 2014 Nov;146(5):1422.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 4, 2011): 89
• Original Estimated Enrollment (submitted: December 8, 2008): 76
• Actual Study Completion Date: October 2009
• Actual Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)

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)

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00805025
• Other Study ID Numbers: GS-US-219-0102
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Gilead Sciences
• Study Sponsor: Gilead Sciences
• Collaborators: Not Provided

Investigators

• Study Director: Steve Lamola, MD; Gilead Sciences

• PRS Account: Gilead Sciences
• Verification Date: February 2014