DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Patients With Cystic Fibrosis and Chronic Burkholderia Species Infection

Information source: Gilead Sciences
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Cystic Fibrosis; Burkholderia Infections

Intervention: AZLI (Drug); Placebo (Drug)

Phase: Phase 3

Status: Completed

Sponsored by: Gilead Sciences

Official(s) and/or principal investigator(s):
Mark Bresnik, MD, Study Director, Affiliation: Gilead Sciences

Summary

The purpose of this research study was to determine if an experimental drug called Aztreonam for Inhalation Solution (AZLI) was safe and effective to treat Burkholderia lung infections in patients with cystic fibrosis (CF). Spirometry was used to assess pulmonary function, and the revised Cystic Fibrosis Questionnaire (CFQ-R) was used to assess quality of life. The CFQ-R is a validated, patient-reported outcome tool used to measure health-related quality of life for children and adults with CF. The study consisted of a 24-week randomized phase, and a 24-week open-label phase. Primary and secondary efficacy analyses were conducted for the 24-week randomized phase only. Safety data were collected for both the randomized and open-label phases.

Clinical Details

Official title: Phase 3b Randomized, Double-Blind, Placebo-Controlled Two-Part Trial to Assess the Safety and Efficacy of Continuous Aztreonam for Inhalation Solution (AZLI) in Subjects With Cystic Fibrosis (CF) and Chronic Burkholderia Species Infection

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Primary outcome: AUCave of Relative Change in FEV1 % Predicted From Baseline to Week 24

Secondary outcome:

Total Number of Systemic and/or Inhaled Antibiotic Courses for Respiratory Events

AUCave of Change in CFQ-R RSS Scores From Baseline to Week 24

AUCave of Relative Change From Baseline to Week 24 in FEV1

AUCave of Relative Change From Baseline to Week 24 in FVC

AUCave of Relative Change From Baseline to Week 24 in FEF25-75

AUCave of the Change From Baseline to Week 24 in Physical Functioning Score as Assessed by the CFQ-R

AUCave of the Change From Baseline to Week 24 in Weight Score as Assessed by the CFQ-R

AUCave of the Change From Baseline to Week 24 in Treatment Burden Score as Assessed by the CFQ-R

Change in BMI From Baseline to Week 24

Change in Burkholderia Spp. CFU in Sputum From Baseline to Week 24

Percentage of Days Participants Used Antibiotics

Percent of Days Hospitalized

Percentage of Missed School or Work Days

Eligibility

Minimum age: 6 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria: 1. Male or female ≥ 6 years of age 2. Subjects with CF as diagnosed by one of the following:

- Documented sweat chloride ≥ 60 milliequivalent (mEq)/L by quantitative

pilocarpine iontophoresis test

- Documented sweat sodium ≥ 60 mmol/L

- Two well-characterized genetic mutations in the CF transmembrane conductance

regulator (CFTR) gene

- Abnormal nasal potential difference (NPD) with accompanying symptoms

characteristic of CF 3. Chronic infection with Burkholderia spp. defined by:

- One sputum (or bronchoalveolar lavage) culture positive for Burkholderia spp.

within 6 months prior to baseline assessment,

- At least 50% of sputum (or bronchoalveolar lavage) cultures collected at least

one month apart over the previous 12 months prior to baseline assessment positive for Burkholderia spp. (minimum of 2 positive cultures), and

- At least one positive sputum (or bronchoalveolar lavage) culture (obtained at

any point in time) confirmed to be Burkholderia spp. by the Cystic Fibrosis Foundation (CFF) Burkholderia cepacia Research Laboratory and Repository at the University of Michigan (or equivalent Canadian reference laboratory). 4. Concomitant aerosolized antibiotic treatment: subjects receiving intermittent (alternating month on/month off) aerosolized antibiotic treatment were eligible, but must have been at least 1 week into their off-treatment cycle at the time of baseline assessment. Subjects receiving continuous aerosolized antibiotic treatment were eligible without restriction on their aerosolized antibiotic treatment. 5. Chest radiograph, computed tomography (CT), or magnetic resonance imaging (MRI) (most recent, obtained within 90 days of screening) without significant acute findings (eg, infiltrates [lobar or diffuse interstitial], pleural effusion, pneumothorax), and no significant intercurrent illness; chronic, stable findings (eg, chronic scarring or atelectasis) were allowed. 6. Subjects (and parent/guardian as required) must have been able to provide written informed consent/assent prior to any study-related procedures, 7. Ability to perform reproducible pulmonary function tests 8. Sexually active females of childbearing potential must have agreed to use a highly effective method of contraception during heterosexual intercourse throughout the study period and for 30 days following discontinuation of study drug. A highly effective method of birth control was defined as a method that would result in a low failure rate (ie, less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), or a vasectomized partner. Exclusion Criteria: 1. Administration of any investigational drug or use of any investigational device within 28 days of randomization/baseline and within six half-lives of the investigational drug (whichever is longer) 2. Administration of AZLI treatment within the 28 days prior to randomization/baseline 3. Known local or systemic hypersensitivity to monobactam antibiotics 4. History of lung transplantation 5. Abnormal renal or hepatic function results at most recent test within the previous 90 days, defined as:

- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 5 times the

upper limit of the normal range (ULN)

- Serum creatinine > 2 times ULN

6. Known portal hypertension or complications of CF hepatopathy 7. Positive urine pregnancy test (confirmed by serum pregnancy test) at screening; all women of childbearing potential were tested 8. Any female of childbearing potential who was lactating or not practicing a highly effective method of birth control as defined in the protocol 9. Any serious or active medical or psychiatric illness which, in the opinion of the investigator, would have interfered with subject treatment, assessment or compliance with the protocol

Locations and Contacts

Mobile, Alabama 36608, United States

Phoenix, Arizona 85016, United States

Little Rock, Arkansas 72205, United States

Denver, Colorado 80206, United States

Hartford, Connecticut 06102, United States

Wilmington, Delaware 19803, United States

Jacksonville, Florida 32207, United States

Miami, Florida 33136, United States

Tampa, Florida 33606, United States

Glenview, Illinois 60025, United States

Boston, Massachusetts 02115, United States

Worcester, Massachusetts 01605, United States

Detroit, Michigan 48201, United States

Minneapolis, Minnesota 55455, United States

St. Louis, Missouri 63110, United States

Las Vegas, Nevada 89107, United States

Morristown, New Jersey 07962, United States

New Brunswick, New Jersey 08903, United States

Albuquerque, New Mexico 87131, United States

New Hyde Park, New York 11040, United States

Chapel Hill, North Carolina 27599, United States

Akron, Ohio 44308, United States

Columbus, Ohio 43205, United States

Toledo, Ohio 43606, United States

Oklahoma City, Oklahoma 73112, United States

Toronto, Ontario M5B 1W8, Canada

Portland, Oregon 97239, United States

Hershey, Pennsylvania 17033, United States

Philadelphia, Pennsylvania 19104, United States

Pittsburgh, Pennsylvania 15201, United States

Charleston, South Carolina 29425, United States

Columbia, South Carolina 29203, United States

Richmond, Virginia 23298, United States

Morgantown, West Virginia 26506, United States

Milwaukee, Wisconsin 53201, United States

Additional Information

Starting date: February 2010
Last updated: February 7, 2014

Page last updated: August 20, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017