Back to resultsEffect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
azithromycin 250 mg tablets
placebo tablets
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring azithromycin, Zithromax
Eligibility Criteria
Inclusion Criteria:
- Male or female, 6-18 years of age at enrollment
- Confirmed diagnosis of CF
- Written informed consent (and assent when applicable)
- Clinically stable at enrollment as assessed by the site investigator
- FEV1 % predicted > 50%
- Ability to comply with medication use, study visits, and study procedures
- Ability to swallow a 250 mg tablet
Exclusion Criteria:
- Weight less than 18.0 kg
- Respiratory culture positive for P. aeruginosa, NTM, or B. cepacia complex within 1 year or at screening, or AFB positive at screening
- Allergy to macrolide antibiotics
- Use of macrolide antibiotics (e.g., azithromycin, clarithromycin) within 60 days of screening
- Use of systemic corticosteroids or intravenous or oral antibiotics within 14 days of screening
- Initiation of high dose ibuprofen, Pulmozyme®, hypertonic saline or aerosolized antibiotics within 30 days of screening
- Chronic therapy with drugs known to have rare but serious interactions with azithromycin: amiodarone, digoxin, disopyramide, lovastatin, pimozide, rifabutin, and nelfinavir
- Investigational drug use within 30 days of screening
- Laboratory abnormalities (creatinine, liver function or neutropenia) at screening and confirmed at follow-up testing prior to randomization
- History of biliary cirrhosis, portal hypertension, or splenomegaly, or splenomegaly on physical exam
- History of ventricular arrhythmia
- Other major organ dysfunction, excluding pancreatic dysfunction
- History of lung transplantation or currently on lung transplant list
- Relative decrease in FEV1 % predicted ≥ 20% between the screening and enrollment visit
- Positive serum pregnancy test at screening
- Pregnant, breastfeeding, or if post-menarche female, unwilling to practice birth control during participation in the study
- History of alcohol, illicit drug or medication abuse within 1 year of screening in the judgment of the site investigator
- Presence of a condition or abnormality that in the opinion of the site investigator would compromise the safety of the subject or the quality of the data
Sites / Locations
- Phoenix Children's Hospital
- Connecticut Children's Medical Center
- Emory University
- Children's Memorial Hospital
- Riley Hospital for Children
- University of Kentucky
- Children's Hospital Boston
- University of Michigan
- University of Minnesota
- Children's Mercy Hospital
- Washington University
- University of Nebraska Medical Center - Pediatric Pulmonary
- Dartmouth Hitchcock Medical Center
- Columbia University
- University of Rochester Medical Center
- SUNY Upstate Medical University
- New York Medical College
- UNC Chapel Hill
- Cincinnati Children's Hospital
- Nationwide Children's Hospital
- The Children's Hospital of Philadelphia
- St. Christopher's Hospital for Children
- Children's Hospital of Pittsburgh, Pulmonary Medicine, Allergy & Immunology
- East Tennessee Children's Hospital, Pediatric Pulmonary & Respiratory Care
- University of Tennessee Health Science Center
- Vanderbilt Children's Hospital
- University of Utah Pediatric Pulmonology
- Vermont Children's Hospital
- University of Virginia at Charlottesville Children's Hospital
- West Virginia University
- Children's Hospital of Wisconsin
- Alberta Children's Hospital
- BC Children's Hospital
- Janeway Children's Health & Rehabilitation Hospital
- McMaster Health Sciences Centre
- Bryan Lyttle, MD, Private Practice
- Children's Hospital of Eastern Ontario
- The Hospital for Sick Children
- CSSS de Chicoutimi
- Montreal Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
Active
Placebo
Arm Description
azithromycin 250 mg tablets
placebo tablets (matched to active drug in appearance)
Outcomes
Primary Outcome Measures
Change in FEV1 From Baseline to End of Treatment at Day 168
Secondary Outcome Measures
Full Information
NCT ID
NCT00431964
First Posted
February 2, 2007
Last Updated
July 24, 2015
Sponsor
CF Therapeutics Development Network Coordinating Center
Collaborators
Cystic Fibrosis Foundation
1. Study Identification
Unique Protocol Identification Number
NCT00431964
Brief Title
Effect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa
Official Title
Multi-center, Multi-national, Randomized, Placebo-Controlled Trial of Azithromycin in Subjects With Cystic Fibrosis 6-18 Years Old, Culture Negative for Pseudomonas Aeruginosa
Study Type
Interventional
2. Study Status
Record Verification Date
July 2015
Overall Recruitment Status
Completed
Study Start Date
February 2007 (undefined)
Primary Completion Date
July 2009 (Actual)
Study Completion Date
November 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CF Therapeutics Development Network Coordinating Center
Collaborators
Cystic Fibrosis Foundation
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a study to examine the safety, effect on lung function, and frequency of symptoms relating to cystic fibrosis during 24 weeks of treatment with the antibiotic azithromycin in 6-18 year-olds with CF who are not infected with Pseudomonas aeruginosa.
Detailed Description
Azithromycin is an antibiotic that has been shown to improve lung function in patients with cystic fibrosis (CF) whose lungs are infected with a bacterium called Pseudomonas aeruginosa. Scientists are not sure how azithromycin works in cystic fibrosis. It does not appear to work by killing the bacteria Pseudomonas aeruginosa, but it may make these bacteria and other bacteria less damaging to the lungs by reducing their ability to attach to the lining of the lung, or by reducing the bacteria's ability to make substances that damage the lungs of patients with cystic fibrosis. Azithromycin may also work directly on the cells in the lungs to improve lung function. This could occur by reducing inflammation (swelling) in the lungs, and/or making the mucus less sticky, or by affecting the salt channel that doesn't function correctly in CF. If azithromycin works in one or more of these ways; it may also be effective in improving lung function in cystic fibrosis patients who are not infected with Pseudomonas aeruginosa.
We are conducting this research study to examine the safety, effect on lung function and frequency of symptoms relating to cystic fibrosis during 24 weeks of treatment with the antibiotic azithromycin. This study is designed to determine if patients with cystic fibrosis whose lungs are not infected with the bacteria Pseudomonas aeruginosa will benefit from 24 weeks of treatment with the antibiotic azithromycin. Benefit will be determined as having better pulmonary function tests and getting sick less often compared to a placebo (sugar pill). This study is also designed to determine if azithromycin is safe when administered for 24 weeks to cystic fibrosis patients not infected with Pseudomonas aeruginosa. By doing this study, we hope to learn more about CF and improve the way in which we treat it.
Comparison: Three times weekly azithromycin tablets added to standard care, compared to three times weekly placebo tablets added to standard care.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
azithromycin, Zithromax
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
263 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Active
Arm Type
Active Comparator
Arm Description
azithromycin 250 mg tablets
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
placebo tablets (matched to active drug in appearance)
Intervention Type
Drug
Intervention Name(s)
azithromycin 250 mg tablets
Other Intervention Name(s)
Zithromax
Intervention Description
One (1) tablet three times weekly for patients who weigh 40-79 lbs
Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
Intervention Type
Drug
Intervention Name(s)
placebo tablets
Other Intervention Name(s)
inactive pill
Intervention Description
One (1) tablet three times weekly for patients who weigh 40-79 lbs
Two (2) tablets three times weekly for patients who weigh greater than or equal to 80 lbs
Primary Outcome Measure Information:
Title
Change in FEV1 From Baseline to End of Treatment at Day 168
Time Frame
change from baseline to day 168
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female, 6-18 years of age at enrollment
Confirmed diagnosis of CF
Written informed consent (and assent when applicable)
Clinically stable at enrollment as assessed by the site investigator
FEV1 % predicted > 50%
Ability to comply with medication use, study visits, and study procedures
Ability to swallow a 250 mg tablet
Exclusion Criteria:
Weight less than 18.0 kg
Respiratory culture positive for P. aeruginosa, NTM, or B. cepacia complex within 1 year or at screening, or AFB positive at screening
Allergy to macrolide antibiotics
Use of macrolide antibiotics (e.g., azithromycin, clarithromycin) within 60 days of screening
Use of systemic corticosteroids or intravenous or oral antibiotics within 14 days of screening
Initiation of high dose ibuprofen, Pulmozyme®, hypertonic saline or aerosolized antibiotics within 30 days of screening
Chronic therapy with drugs known to have rare but serious interactions with azithromycin: amiodarone, digoxin, disopyramide, lovastatin, pimozide, rifabutin, and nelfinavir
Investigational drug use within 30 days of screening
Laboratory abnormalities (creatinine, liver function or neutropenia) at screening and confirmed at follow-up testing prior to randomization
History of biliary cirrhosis, portal hypertension, or splenomegaly, or splenomegaly on physical exam
History of ventricular arrhythmia
Other major organ dysfunction, excluding pancreatic dysfunction
History of lung transplantation or currently on lung transplant list
Relative decrease in FEV1 % predicted ≥ 20% between the screening and enrollment visit
Positive serum pregnancy test at screening
Pregnant, breastfeeding, or if post-menarche female, unwilling to practice birth control during participation in the study
History of alcohol, illicit drug or medication abuse within 1 year of screening in the judgment of the site investigator
Presence of a condition or abnormality that in the opinion of the site investigator would compromise the safety of the subject or the quality of the data
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lisa Saiman, MD, MPH
Organizational Affiliation
Columbia University
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Michael Anstead, MD
Organizational Affiliation
University of Kentucky
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Felix Ratjen, MD
Organizational Affiliation
The Hospital for Sick Children, Toronto, Ontario
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Larry Lands, MD
Organizational Affiliation
Montreal Children's Hospital of the MUHC
Official's Role
Principal Investigator
Facility Information:
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Connecticut Children's Medical Center
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06106
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Children's Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
Riley Hospital for Children
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Kentucky
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
Children's Hospital Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Washington University
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
University of Nebraska Medical Center - Pediatric Pulmonary
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
Dartmouth Hitchcock Medical Center
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
Columbia University
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
SUNY Upstate Medical University
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
New York Medical College
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Facility Name
UNC Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
St. Christopher's Hospital for Children
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19134
Country
United States
Facility Name
Children's Hospital of Pittsburgh, Pulmonary Medicine, Allergy & Immunology
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
East Tennessee Children's Hospital, Pediatric Pulmonary & Respiratory Care
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37916
Country
United States
Facility Name
University of Tennessee Health Science Center
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Facility Name
Vanderbilt Children's Hospital
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
University of Utah Pediatric Pulmonology
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Vermont Children's Hospital
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05401
Country
United States
Facility Name
University of Virginia at Charlottesville Children's Hospital
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
West Virginia University
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
Children's Hospital of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Alberta Children's Hospital
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B 6A8
Country
Canada
Facility Name
BC Children's Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
Janeway Children's Health & Rehabilitation Hospital
City
St. John's
State/Province
Newfoundland and Labrador
ZIP/Postal Code
A1B 3V6
Country
Canada
Facility Name
McMaster Health Sciences Centre
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Bryan Lyttle, MD, Private Practice
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5B8
Country
Canada
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
CSSS de Chicoutimi
City
Chicoutimi
State/Province
Quebec
ZIP/Postal Code
G7H 5H6
Country
Canada
Facility Name
Montreal Children's Hospital
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3H 1P3
Country
Canada
12. IPD Sharing Statement
Citations:
PubMed Identifier
20442386
Citation
Saiman L, Anstead M, Mayer-Hamblett N, Lands LC, Kloster M, Hocevar-Trnka J, Goss CH, Rose LM, Burns JL, Marshall BC, Ratjen F; AZ0004 Azithromycin Study Group. Effect of azithromycin on pulmonary function in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA. 2010 May 5;303(17):1707-15. doi: 10.1001/jama.2010.563.
Results Reference
result
PubMed Identifier
22595153
Citation
Ratjen F, Saiman L, Mayer-Hamblett N, Lands LC, Kloster M, Thompson V, Emmett P, Marshall B, Accurso F, Sagel S, Anstead M. Effect of azithromycin on systemic markers of inflammation in patients with cystic fibrosis uninfected with Pseudomonas aeruginosa. Chest. 2012 Nov;142(5):1259-1266. doi: 10.1378/chest.12-0628.
Results Reference
derived
Learn more about this trial
Effect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa
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