Effect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa
Multi-center, Multi-national, Randomized, Placebo-Controlled Trial of Azithromycin in Subjects With Cystic Fibrosis 6-18 Years Old, Culture Negative for Pseudomonas Aeruginosa
1 other identifier
interventional
263
2 countries
40
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_4
Started Feb 2007
Typical duration for phase_4
40 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2007
CompletedFirst Submitted
Initial submission to the registry
February 2, 2007
CompletedFirst Posted
Study publicly available on registry
February 6, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2009
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2009
CompletedResults Posted
Study results publicly available
August 19, 2015
CompletedAugust 19, 2015
July 1, 2015
2.4 years
February 2, 2007
July 24, 2015
July 24, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change in FEV1 From Baseline to End of Treatment at Day 168
change from baseline to day 168
Study Arms (2)
Active
ACTIVE COMPARATORazithromycin 250 mg tablets
Placebo
PLACEBO COMPARATORplacebo tablets (matched to active drug in appearance)
Interventions
* 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
* 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
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
- +3 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (40)
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
Emory University
Atlanta, Georgia, 30322, United States
Children's Memorial Hospital
Chicago, Illinois, 60614, United States
Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
University of Kentucky
Lexington, Kentucky, 40536, United States
Children's Hospital Boston
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Washington University
St Louis, Missouri, 63110, United States
University of Nebraska Medical Center - Pediatric Pulmonary
Omaha, Nebraska, 68198, United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756, United States
Columbia University
New York, New York, 10032, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
New York Medical College
Valhalla, New York, 10595, United States
UNC Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134, United States
Children's Hospital of Pittsburgh, Pulmonary Medicine, Allergy & Immunology
Pittsburgh, Pennsylvania, 15213, United States
East Tennessee Children's Hospital, Pediatric Pulmonary & Respiratory Care
Knoxville, Tennessee, 37916, United States
University of Tennessee Health Science Center
Memphis, Tennessee, 38105, United States
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232, United States
University of Utah Pediatric Pulmonology
Salt Lake City, Utah, 84108, United States
Vermont Children's Hospital
Burlington, Vermont, 05401, United States
University of Virginia at Charlottesville Children's Hospital
Charlottesville, Virginia, 22908, United States
West Virginia University
Morgantown, West Virginia, 26506, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
BC Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
Janeway Children's Health & Rehabilitation Hospital
St. John's, Newfoundland and Labrador, A1B 3V6, Canada
McMaster Health Sciences Centre
Hamilton, Ontario, L8N 3Z5, Canada
Bryan Lyttle, MD, Private Practice
London, Ontario, N6A 5B8, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
CSSS de Chicoutimi
Chicoutimi, Quebec, G7H 5H6, Canada
Montreal Children's Hospital
Montreal, Quebec, H3H 1P3, Canada
Related Publications (2)
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.
PMID: 20442386RESULTRatjen 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.
PMID: 22595153DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Nicole Hamblett
- Organization
- Seattle Children's Hospital
Study Officials
- PRINCIPAL INVESTIGATOR
Lisa Saiman, MD, MPH
Columbia University
- PRINCIPAL INVESTIGATOR
Michael Anstead, MD
University of Kentucky
- PRINCIPAL INVESTIGATOR
Felix Ratjen, MD
The Hospital for Sick Children, Toronto, Ontario
- PRINCIPAL INVESTIGATOR
Larry Lands, MD
Montreal Children's Hospital of the MUHC
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 2, 2007
First Posted
February 6, 2007
Study Start
February 1, 2007
Primary Completion
July 1, 2009
Study Completion
November 1, 2009
Last Updated
August 19, 2015
Results First Posted
August 19, 2015
Record last verified: 2015-07