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Trial registered on ANZCTR


Registration number
ACTRN12612001167853
Ethics application status
Approved
Date submitted
31/10/2012
Date registered
2/11/2012
Date last updated
2/11/2012
Type of registration
Retrospectively registered

Titles & IDs
Public title
A study examining the utility of inhaled mannitol as an additional therapy during an admission to hospital for an acute pulmonary exacerbation in children with cystic fibrosis
Scientific title
A double blind, placebo-controlled, randomised study of inhaled mannitol during acute pulmonary exacerbation in children with cystic fibrosis – a pilot study
Secondary ID [1] 281475 0
Nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 287734 0
Condition category
Condition code
Respiratory 288076 288076 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 288097 288097 0 0
Cystic fibrosis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Inhaled dry powder mannitol 400mg (10 x 40mg capsules) twice daily for 12 days as an adjunctive treatment during hospitalisation for an acute pulmonary exacerbation. Capsules were placed in a special delivery device which pierced the capsule and the contents were inhaled.
Intervention code [1] 285979 0
Treatment: Drugs
Comparator / control treatment
Inhaled Placebo. The placebo used was large particle mannitol which is not respirable and therefore simply lodges in the upper respiratory tract on inhalation. This was also given as 10 x 40mg capsules twice daily for 12 days. As with thr active drug, capsules were placed in a special delivery device which pierced the capsule and the contents were inhaled.
Control group
Placebo

Outcomes
Primary outcome [1] 288282 0
Improvement in clinical status at the end of the treatment period (as assessed using a CF specific clinical score)
Timepoint [1] 288282 0
day 14 of the admission
Secondary outcome [1] 299746 0
Improvement in lung function as assessed by spirometry
Timepoint [1] 299746 0
day 14 of the admission
Secondary outcome [2] 299747 0
Improvement in lung function as assessed by multiple breath washout
Timepoint [2] 299747 0
day 14 of the admission
Secondary outcome [3] 299748 0
Improvement in lung function as assessed by forced oscillation technique
Timepoint [3] 299748 0
day 14 of the admission
Secondary outcome [4] 299749 0
Improvement in lung function as assessed by cardiorespiratory exercise testing
Timepoint [4] 299749 0
day 14 of the admission
Secondary outcome [5] 299752 0
Improvement in lung function as assessed by plethysmography
Timepoint [5] 299752 0
day 14 of the admission

Eligibility
Key inclusion criteria
admission to hospital with a pulmonary exacerbation as defined by the Fuch’s Criteria and requiring IV antibiotic treatment.
at least 6 years of age
FEV1 at least 40% predicted
Minimum age
6 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
commencement of oral steroids (on admission) or new mucolytic (within 3 months of admission)
oxygen requirement or planned surgery during admission
significant haemoptysis during current exacerbation

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
1:1 randomisation by study pharmacist, in conjunction with the hospital statistician. Simple randomisation using computer generated sequence performed by statistician and boxes labelled as study drug. The Pharmacist was not an investigator and held the randomisation list.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
The randomization will be 1:1 active to placebo. Simple randomisation using a randomisation table created by computer software (i.e. computerised sequence generation).
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
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)

Funding & Sponsors
Funding source category [1] 286241 0
Hospital
Name [1] 286241 0
The Children's Hospital at Westmead
Address [1] 286241 0
Locked Bag 4001
Westmead
NSW 2145
Country [1] 286241 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Locked Bag 4001
Westmead
NSW 2145
Country
Australia
Secondary sponsor category [1] 285048 0
Commercial sector/Industry
Name [1] 285048 0
Pharmaxis
Address [1] 285048 0
Locked Bag 5015, Frenchs Forest NSW 2086
Country [1] 285048 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 288320 0
Royal Alexandria Hospital for Children
Ethics committee address [1] 288320 0
Locked Bag 4001
Westmead
NSW 2145
Ethics committee country [1] 288320 0
Australia
Date submitted for ethics approval [1] 288320 0
Approval date [1] 288320 0
13/04/2007
Ethics approval number [1] 288320 0
2007/016

Summary
Brief summary
To see if the addition of dry powder mannitol as an extra therapy during hospitalisation for an acute pulmonary exacerbation in young people with Cystic Fibrosis improves outcomes from that admission. Our hypothesis is that it will improve not only clinical status at the time of discharge but also measures of lung function.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 34898 0
Address 34898 0
Country 34898 0
Phone 34898 0
Fax 34898 0
Email 34898 0
Contact person for public queries
Name 18145 0
Dr Hiran Selvadurai
Address 18145 0
Department of Respiratory Medicine
Corner of Hawkesbury Road and Hainsworth Street
Locked Bag 4001
The Children's Hospital at Westmead
Westmead
NSW 2145
Country 18145 0
Australia
Phone 18145 0
+61298453395
Fax 18145 0
+61298453396
Email 18145 0
hiran.selvadurai@health.nsw.gov.au
Contact person for scientific queries
Name 9073 0
Dr Hiran Selvadurai
Address 9073 0
Department of Respiratory Medicine
Corner of Hawkesbury Road and Hainsworth Street
Locked Bag 4001
The Children's Hospital at Westmead
Westmead
NSW 2145
Country 9073 0
Australia
Phone 9073 0
+61298453395
Fax 9073 0
+61298453396
Email 9073 0
hiran.selvadurai@health.nsw.gov.au

No information has been provided regarding IPD availability
Summary results
No Results