Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12611000945921
Ethics application status
Approved
Date submitted
13/03/2009
Date registered
2/09/2011
Date last updated
2/09/2011
Type of registration
Retrospectively registered

Titles & IDs
Public title
Early lung disease in young infants and young children with cystic fibrosis
Scientific title
The relationship between the lung clearance index, airway infection and inflammation and systemic inflammatory markers in infants and young children with cystic fibrosis
Secondary ID [1] 262974 0
Nil
Universal Trial Number (UTN)
Trial acronym
N/A
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 4471 0
Early lung disease 4472 0
Condition category
Condition code
Respiratory 4743 4743 0 0
Other respiratory disorders / diseases
Infection 4744 4744 0 0
Other infectious diseases
Human Genetics and Inherited Disorders 270872 270872 0 0
Cystic fibrosis

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Newborn screened infants with cystic fibrosis less than 3 years of age attending the Cystic Fibrosis clinic at Sydney Children's Hospital Randwick will be prospectively recruited for bronchoalveolar lavage, infant lung function testing, a chest xray and venepuncture.
A control group of similarly aged healthy infants undergoing sedated cardiac echo or a DMSA scan will also be recruited for infant lung function testing over the same period thus serving as the normative or reference population for infant lung function.
Healthy in fants and infants with CF will undergo sedated lung function on a single occasion over a duration of approximately one hour.
Intervention code [1] 4215 0
Not applicable
Comparator / control treatment
Healthy infants and young children without previous respiratory, cardiac or neurologic imparirment attending Sydney Children's Hospital RANDWICK for a sedated cardiac echo or DMSA renal scan will be approached to undergo lung function as a follow on procedure whilst sedated for their primary investigation
Control group
Active

Outcomes
Primary outcome [1] 5611 0
Lung Clearance Index measured by a multiple-breath washout (MBW) method using a commercially available Infant Lung Function Testing System (Exhalyzer R)
Timepoint [1] 5611 0
At baseline: One-off measurement in this cross-sectional case-control study
Secondary outcome [1] 9442 0
Bronchoalveolar lavage markers of infection (measured by quantitative microbiologic culture) and inflammation (neutrophils, totol cell count , neutrophil elastase and interleukin-8, and the S100 proteins)
Timepoint [1] 9442 0
At baseline: one off measurement
Secondary outcome [2] 287891 0
Systemic markers of inflammation including the S100 proteins and C-reactive protein and blood neutrophils in additon to urinary desmosine
Timepoint [2] 287891 0
All at baseline as a one off measurement
Secondary outcome [3] 287892 0
Prevalence of an abnormal ph probe indicating gastro-oesophageal reflux (only in children with CF)
Timepoint [3] 287892 0
All at baseline as a one off measurement only in subjects with CF

Eligibility
Key inclusion criteria
Cystic fibrosis infants under age 3 years and healthy control children under 3 years of age will be recruited for sedated lung function testing. CF children up to 5 years of age will also undergo venepuncture and urine analysis for desmosine determination
Minimum age
No limit
Maximum age
5 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Chronic neonatal lung disease
Other respiratory disease
Cardiac disease
Neuurologic disease
Respiratory infection in the 3 weeks prior to infant lung function testing

Study design
Purpose
Screening
Duration
Cross-sectional
Selection
Case control
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
1/06/2004
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
45
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 4662 0
Self funded/Unfunded
Name [1] 4662 0
Country [1] 4662 0
Primary sponsor type
Individual
Name
Dr Yvonne Belessis MBBS(Hons) FRACP MPH PhD
Address
Sydney Children's Hospital
High Street
RANDWICK, NSW 2031
Country
Australia
Secondary sponsor category [1] 4208 0
None
Name [1] 4208 0
Address [1] 4208 0
Country [1] 4208 0
Other collaborator category [1] 252244 0
University
Name [1] 252244 0
University of New South Wales
Address [1] 252244 0
Randwick, NSW 2031
Country [1] 252244 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 6698 0
South Eastern Sydney and Illawarra Health Service- Eastern Section
Ethics committee address [1] 6698 0
Hospital Research and Ethics Committee (HREC) Prince of Wales Hospital High Street
Randwick NSW 2031
Ethics committee country [1] 6698 0
Australia
Date submitted for ethics approval [1] 6698 0
Approval date [1] 6698 0
01/01/2002
Ethics approval number [1] 6698 0
1/02/0098

Summary
Brief summary
Although the lungs in children with cystic fibrosis (CF) are essentially normal at birth, airway infection and inflammation may begin within the first few weeks of life. As postnatal lung development, including significant alveolarisation continues during the first 2-3 years of life, this is a vulnerable period where undiagnosed infection and chronic inflammation may lead to irreversible tissue damage and lung disease. Identifying and treating infection (especially with Pseudomonas), in early childhood may prevent significant lung disease, reduce morbidity and improve survival.
Early infection is often subclinical and conventional screening methods such as sputum microbiology, chest radiology and lung function, are either not possible or insensitive to detect disease in this age group. Upper airway cultures such as throat swabs or cough suction specimens correlate poorly with lower airway pathogens and the currently available systemic markers of inflammation lack sensitivity. Bronchoaclveolar lavage (BAL) is considered the gold standard for identifying airway infection and inflammation in young children with CF. The association between BAL markers of infection and inflammation and sensitive infant lung function testing such as the multiple breath washout test which determines the lung clearance index has not been studied.
This study will investigate early CF lung disease by assessing the relationships between BAL markers of infection/inflammation, the lung clearance index, systemic markers of neutrophil inflammation (S100 proteins) and urinary desmosine. The study will also determine the prevalence of gastro-oesophageal reflux and the relationship between BAL pepsin and airway infection and inflammation.
Trial website
Trial related presentations / publications
Presentations:
1. Early detection of cystic fibrosis lung disease: LCI predicts BAL evidence of infection and inflammation. TSANZ 2009

2. BAL pepsin is associated with infection in infants and young children with cystic fibrosis and is suggestive of concurrent microaspiration in early CF lung disease. TSANZ 2008
Public notes

Contacts
Principal investigator
Name 29392 0
Address 29392 0
Country 29392 0
Phone 29392 0
Fax 29392 0
Email 29392 0
Contact person for public queries
Name 12639 0
Dr Yvonne Belessis
Address 12639 0
Sydney Children's Hospital
High Street
RANDWICK, NSW 2031
Country 12639 0
Australia
Phone 12639 0
61 2 93821246
Fax 12639 0
61 2 93821580
Email 12639 0
[email protected]
Contact person for scientific queries
Name 3567 0
Dr Yvonne Belessis
Address 3567 0
Sydney Children's Hospital
High Street
RANDWICK, NSW 2031
Country 3567 0
Australia
Phone 3567 0
61 2 93821246
Fax 3567 0
61 2 93821580
Email 3567 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
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