Prevention of Bronchiectasis in Infants With Cystic Fibrosis
NCT ID: NCT01270074
Last Updated: 2021-09-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
130 participants
INTERVENTIONAL
2012-04-30
2021-03-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Effect of Azithromycin on Lung Function in 6-18 Year-olds With Cystic Fibrosis (CF) Not Infected With P. Aeruginosa
NCT00431964
Continuous Azithromycin in Cystic Fibrosis Patients Beyond Two Years
NCT02803944
Study to Assess Efficacy of AZD1236 in Patients With Cystic Fibrosis
NCT00812045
Scandinavian Cystic Fibrosis Azithromycin Study
NCT00411736
Improving Treatment of Nontuberculous Mycobacterial Infection in Cystic Fibrosis
NCT02372383
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Title Multi-centre randomized placebo-controlled study of azithromycin in the primary prevention of radiologically-defined bronchiectasis in infants with Cystic Fibrosis
Clinical Phase Phase 3
Protocol Number: AZI001
TGA Reference Number:
Protocol Co-Chairs: Peter D. Sly \& Stephen M. Stick Microbiology Consultant: Lisa Saiman CT Consultant: Harm Tiddens Statistical Consultant: Robert S Ware
Study Design Randomized, double-blind parallel groups. Participants will be randomized into one of the following 2 groups on a 1:1 ratio with 65 participants per group;
Group A: 10 mg/kg (as 200mg/5ml) azithromycin three times weekly for three years added to standard CF therapy.
Group B: matched placebo three times weekly for three years added to standard CF therapy.
Accrual Objective 130 children
Accrual Period 24 months
Study Duration 36 months
Countries: Australia and New Zealand
Sites: Brisbane Au, Sydney, Au, Melbourne Au, Adelaide Au, Perth Au,Auckland Nz, Christchurch Nz.
Primary Endpoint The primary endpoints are the proportion of children with radiologically-defined bronchiectasis at age 3 years, and the proportion of lung tissue affected by disease at age 3 years.
Secondary Endpoints
* The extent and severity of bronchiectasis at age 3 years
* The volume of trapped gas at age 3 years
* CF-related quality of life
* Time to first pulmonary exacerbation
* Proportion of participants experiencing a pulmonary exacerbation
* Number of courses of inhaled or oral antibiotics
* Number of days of inhaled antibiotics
* Incidence of hospitalizations/Accident and Emergency department (A\&E) visits for an acute respiratory exacerbation
* Number of days hospitalized for an acute respiratory exacerbation
* Number of days if intravenous antibiotics
* Body mass index at 3 years of age.
Exploratory Endpoints
* Markers of neutrophilic inflammation
* Markers of oxidative stress
* Composition of airway flora
Safety Endpoints
* Proportion of participants growing P. aeruginosa in BAL
* Age of acquisition of P. aeruginosa in BAL
* Emergence of macrolide-resistant S. aureus, small colony variant S. aureus and non-tuberculous mycobacteria (NTM)
* Treatment-related adverse events
* Haematology and clinical chemistry
Inclusion Criteria Participants who meet all of the following criteria are eligible for enrolment as study participants:
1. Children of either sex with a diagnosis of CF following detection via New Born Screening (NBS) for cystic fibrosis
2. Participants who, in the opinion of the Investigator, are able to comply with the protocol for its duration
3. Written informed consent signed and dated by parent/legal guardian according to local regulations
Exclusion Criteria Participants who meet any of these criteria are not eligible for enrolment as trial participants:
1. Born \<30 weeks gestation
2. Prolonged mechanical ventilation in the first 3 months of life
3. Participation in another randomized controlled trial within the 3 months preceding inclusion in this study
4. A significant medical disease or condition other than CF that is likely to interfere with the child's ability to complete the entire protocol
5. Previous major surgery except for meconium ileus
6. Macrolide hypersensitivity
Treatment Description ZITHROMAX® (azithromycin)
Study Procedures The study participants will be stratified by investigational site and randomly assigned to either azithromycin or placebo for three years.
Statistical Considerations Participants will be randomized in blocks to the treatment group or the placebo group using a one-to-one ratio. Randomization will be stratified by study site. This will ensure an approximately equal allocation to each group within each site.
Interim Analyses Interim analyses will occur when the first 50% of children (n=33 per group have completed the 12 month CT and when all subjects have completed the 12 month CT. Interim analyses will determine safety or success (unethical to continue).
Stopping Rules Study enrolment may be stopped if any of the following events occur:
* Death of a participant that is related to study treatment.
* The trial meets the definition of futility or success at either of the planned interim analyses
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
PREVENTION
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
azithromycin liquid preparation
azithromycin will be given at a dose of 10mg/kg given three times per week from three months of age to three years of age
Azithromycin
azithromycin will be given as a liquid preparation at a dose of 10 mg/kg three times per week from three months of age until three years of age
inert liquid preparation
inert liquid preparation will be given three times per week from three months of age to three years of age
Placebo control
inert liquid preparation will be given three times per week from three months of age to three years of age
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Azithromycin
azithromycin will be given as a liquid preparation at a dose of 10 mg/kg three times per week from three months of age until three years of age
Placebo control
inert liquid preparation will be given three times per week from three months of age to three years of age
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
2. Participants who, in the opinion of the Investigator, are able to comply with the protocol for its duration
3. Written informed consent signed and dated by parent/legal guardian according to local regulations
Exclusion Criteria
2. Prolonged mechanical ventilation in the first 3 months of life
3. Participation in another randomized controlled trial within the 3 months preceding inclusion in this study
4. A significant medical disease or condition other than CF that is likely to interfere with the child's ability to complete the entire protocol
5. Previous major surgery except for meconium ileus
6. Macrolide hypersensitivity
6 Weeks
6 Months
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Telethon Kids Institute
OTHER
The University of Queensland
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Peter D Sly, MMBS MD DSc
Role: STUDY_CHAIR
The University of Queensland
Stephen M Stick, MBBChir PhD
Role: STUDY_CHAIR
Telethon Kids Institute
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Sydney Children's Hospital
Sydney, New South Wales, Australia
Westmead Children's Hospital
Sydney, New South Wales, Australia
Queensland Children's Hospital
Brisbane, Queensland, Australia
Mater Children's Hospital
Brisbane, Queensland, Australia
Royal Children's Hospital
Brisbane, Queensland, Australia
Women's and Children's Hospital
Adelaide, South Australia, Australia
Monash Medical Centre
Melbourne, Victoria, Australia
Royal Children's Hospital
Melbourne, Victoria, Australia
Perth Children's Hospital
Perth, Western Australia, Australia
Starship Hospital
Auckland, , New Zealand
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Douglas TA, Brennan S, Gard S, Berry L, Gangell C, Stick SM, Clements BS, Sly PD. Acquisition and eradication of P. aeruginosa in young children with cystic fibrosis. Eur Respir J. 2009 Feb;33(2):305-11. doi: 10.1183/09031936.00043108. Epub 2008 Nov 14.
Sly PD, Brennan S, Gangell C, de Klerk N, Murray C, Mott L, Stick SM, Robinson PJ, Robertson CF, Ranganathan SC; Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST-CF). Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. Am J Respir Crit Care Med. 2009 Jul 15;180(2):146-52. doi: 10.1164/rccm.200901-0069OC. Epub 2009 Apr 16.
Stick SM, Brennan S, Murray C, Douglas T, von Ungern-Sternberg BS, Garratt LW, Gangell CL, De Klerk N, Linnane B, Ranganathan S, Robinson P, Robertson C, Sly PD; Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF). Bronchiectasis in infants and preschool children diagnosed with cystic fibrosis after newborn screening. J Pediatr. 2009 Nov;155(5):623-8.e1. doi: 10.1016/j.jpeds.2009.05.005. Epub 2009 Jul 19.
Stick SM, Foti A, Ware RS, Tiddens HAWM, Clements BS, Armstrong DS, Selvadurai H, Tai A, Cooper PJ, Byrnes CA, Belessis Y, Wainwright C, Jaffe A, Robinson P, Saiman L, Sly PD; COMBAT CF Study Group. The effect of azithromycin on structural lung disease in infants with cystic fibrosis (COMBAT CF): a phase 3, randomised, double-blind, placebo-controlled clinical trial. Lancet Respir Med. 2022 Aug;10(8):776-784. doi: 10.1016/S2213-2600(22)00165-5. Epub 2022 Jun 2.
Related Links
Access external resources that provide additional context or updates about the study.
The Australia Respiratory Early Surveillance Team for Cystic Fibrosis
Telethon Institute for Child Health Research
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
STICK10K0
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
AZI001
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.