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Trial registered on ANZCTR


Registration number
ACTRN12610001072000
Ethics application status
Approved
Date submitted
2/12/2010
Date registered
6/12/2010
Date last updated
1/02/2019
Date data sharing statement initially provided
1/02/2019
Date results information initially provided
1/02/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
Prevention of bronchiectasis in infants with cystic fibrosis
Scientific title
A Phase 3 multi-centre randomised placebo-controlled study of azithromycin in the primary prevention of radiologically-defined bronchiectasis in infants with cystic fibrosis
Secondary ID [1] 253230 0
STICK10K0
Universal Trial Number (UTN)
U1111-1118-3843
Trial acronym
COMBAT CF
Linked study record

Health condition
Health condition(s) or problem(s) studied:
bronchiectasis in cystic fibrosis 258763 0
Condition category
Condition code
Respiratory 258909 258909 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
azithromycin liquid preparation given by oral administration 10 mg/kg three times a week from 3 months of age until 3 years of age
Intervention code [1] 257698 0
Prevention
Comparator / control treatment
inert liquid preparation, identical in taste and colour, given by oral administration three times a week from 3 months of age until 3 years of age
Control group
Placebo

Outcomes
Primary outcome [1] 259767 0
Proportion of children with radiologically-defined bronchiectasis
Timepoint [1] 259767 0
three years of age
Secondary outcome [1] 268533 0
extent and severity of bronchiectasis
Timepoint [1] 268533 0
three years of age
Secondary outcome [2] 268534 0
CF-related quality of life
Timepoint [2] 268534 0
three years of age
Secondary outcome [3] 268535 0
time to first pulmonary exacerbation
Timepoint [3] 268535 0
3 years of age
Secondary outcome [4] 268536 0
proportion of participants experiencing a pulmonary exacerbation
Timepoint [4] 268536 0
3 years of age
Secondary outcome [5] 268537 0
number of courses of inhaled or oral antibiotics
Timepoint [5] 268537 0
3 years of age
Secondary outcome [6] 268538 0
number of days of inhaled antibiotics
Timepoint [6] 268538 0
3 years of age
Secondary outcome [7] 268539 0
incidence of hospitalizations/Accident & Emergency visitsfor acute respiratory exacerbations
Timepoint [7] 268539 0
3 years of age
Secondary outcome [8] 268540 0
number of days of hospitalization and number of days of intravenous antibiotics for acute respiratory exacerbations
Timepoint [8] 268540 0
3 years of age
Secondary outcome [9] 279410 0
volume of trapped gas at age 3 years assessed using a low dose volumetric computed tomography scan
Timepoint [9] 279410 0
3 years of age
Secondary outcome [10] 279411 0
Body mass index
Timepoint [10] 279411 0
3 years of age

Eligibility
Key inclusion criteria
1. Children of either sex with a diagnosis of CF following detection via New Born Screening (NBS);
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
Minimum age
6 Weeks
Maximum age
6 Months
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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

Study design
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Participants are enrolled following confirmation of cystic fibrosis via new born screening. Randomisation by IWRS.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Block randomization stratified by study site
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA,VIC
Recruitment hospital [1] 7759 0
Princess Margaret Hospital - Subiaco
Recruitment hospital [2] 7760 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [3] 7761 0
Lady Cilento Children's Hospital - South Brisbane
Recruitment hospital [4] 7762 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [5] 7763 0
Sydney Children's Hospital - Randwick
Recruitment hospital [6] 7764 0
Womens and Childrens Hospital - North Adelaide
Recruitment postcode(s) [1] 3494 0
6008
Recruitment postcode(s) [2] 3495 0
4101
Recruitment postcode(s) [3] 3496 0
4029
Recruitment postcode(s) [4] 3497 0
2145
Recruitment postcode(s) [5] 3498 0
2031
Recruitment postcode(s) [6] 3500 0
3052
Recruitment postcode(s) [7] 3501 0
3168
Recruitment postcode(s) [8] 3502 0
5006
Recruitment postcode(s) [9] 15693 0
6008 - Subiaco
Recruitment postcode(s) [10] 15694 0
3052 - Parkville
Recruitment postcode(s) [11] 15695 0
4101 - South Brisbane
Recruitment postcode(s) [12] 15696 0
2145 - Westmead
Recruitment postcode(s) [13] 15697 0
2031 - Randwick
Recruitment postcode(s) [14] 15698 0
5006 - North Adelaide
Recruitment outside Australia
Country [1] 3072 0
New Zealand
State/province [1] 3072 0
Country [2] 5309 0
New Zealand
State/province [2] 5309 0
Christchurch

Funding & Sponsors
Funding source category [1] 258170 0
Charities/Societies/Foundations
Name [1] 258170 0
Cystic Fibrosis Foundation Therapeutics Inc
Address [1] 258170 0
Grants and Contracts office
6931 Arlington Rd
Bethesda Maryland 20814
Country [1] 258170 0
United States of America
Primary sponsor type
Other
Name
Telethon Kids Institute
Address
100 Roberts Rd
Subiaco WA 6008
Country
Australia
Secondary sponsor category [1] 257344 0
Other
Name [1] 257344 0
Queensland Children's Medical Research Institute
Address [1] 257344 0
Level 4, Foundation Building
Royal Children's Hospital
Herston Rd, Herston Qld 4029
Country [1] 257344 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 260158 0
Princess Margaret Hospital for Children Human Ethics Committee
Ethics committee address [1] 260158 0
Roberts Road
Subiaco WA 6008
Ethics committee country [1] 260158 0
Australia
Date submitted for ethics approval [1] 260158 0
Approval date [1] 260158 0
15/04/2010
Ethics approval number [1] 260158 0
1795/EP

Summary
Brief summary
The general aim of this project is to conduct a randomized, double-blind, placebo, placebo-controlled clinical trial of azithromycin to determine whether treatment from infancy is safe and will prevent the onset of bronchiectasis. One hundred and thirty infants will be recruited from CF clinics in Australia and New Zealand and treated from 3 months to three years of age. The primary outcome will be the proportion with radiologically-defined bronchiectasis at 3 years of age. Safety and mechanistic evaluations will also be undertaken.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 31984 0
Prof Stephen Stick
Address 31984 0
Department of Respiratory Medicine
Princess Margaret Hospital for Children
Roberts Rd
Subiaco WA 6008
Country 31984 0
Australia
Phone 31984 0
+61 8 9340 8830
Fax 31984 0
+61 8 9340 8181
Email 31984 0
stephen.stick@health.wa.gov.au
Contact person for public queries
Name 15231 0
Ms Nat Eiffler
Address 15231 0
Telethon Kids Institute
100 Roberts Rd
SUBIACO WA 6008
Country 15231 0
Australia
Phone 15231 0
+61 8 94897814
Fax 15231 0
+61 7 3636 5578
Email 15231 0
nat.eiffler@telethonkids.org.au
Contact person for scientific queries
Name 6159 0
Prof Professor Stephen Stick
Address 6159 0
Department of Respiratory Medicine
Princess Margaret Hospital for Children
Ropberts Rd
Subiaco WA 6008
Country 6159 0
Australia
Phone 6159 0
+61893408830
Fax 6159 0
+61893408181
Email 6159 0
stephen.stick@health.wa.gov.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No/undecided IPD sharing reason/comment
What supporting documents are/will be available?
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary