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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00446680




Registration number
NCT00446680
Ethics application status
Date submitted
12/03/2007
Date registered
13/03/2007
Date last updated
25/06/2010

Titles & IDs
Public title
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
Scientific title
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
Secondary ID [1] 0 0
DPM-CF-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Mannitol
Treatment: Drugs - placebo

Experimental: 1 -

Placebo Comparator: 2 -


Treatment: Drugs: Mannitol
400mg BD for 6 months followed by a 6 month open label period

Treatment: Drugs: placebo
placebo BD for 6 months
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
Timepoint [1] 0 0
6 months
Secondary outcome [1] 0 0
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF on existing RhDNase treatment compared to control. (key objective)
Timepoint [1] 0 0
6 months
Secondary outcome [2] 0 0
Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)
Timepoint [2] 0 0
6 months / 12 months
Secondary outcome [3] 0 0
Improves quality of life (key objective)
Timepoint [3] 0 0
6 months
Secondary outcome [4] 0 0
Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
Timepoint [4] 0 0
6 months / 12 months
Secondary outcome [5] 0 0
Reduces days in hospital due to pulmonary exacerbations
Timepoint [5] 0 0
6 months / 12 months
Secondary outcome [6] 0 0
Improves other measures of lung function
Timepoint [6] 0 0
6 months
Secondary outcome [7] 0 0
Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)
Timepoint [7] 0 0
6 months / 12 months
Secondary outcome [8] 0 0
Reduces hospital and community care costs
Timepoint [8] 0 0
6 months / 12 months

Eligibility
Key inclusion criteria
Main

- Written informed consent

- Confirmed diagnosis of cystic fibrosis

- Aged > 6 years

- FEV1 >30 % and < 90% predicted

- Able to perform all the techniques necessary to measure lung function

Main
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- "Terminally ill" or listed for lung transplantation

- Had a lung transplant

- Using nebulised hypertonic saline

- Significant episode of haemoptysis (>60 mL) in the three months prior to enrolment

- Recent myocardial infarction or cerebral vascular accident

- Breast feeding or pregnant, or plan to become pregnant while in the study
participating in another investigative drug study, parallel to, or within 4 weeks of
study entry

- Allergy or intolerance to mannitol

- Using beta blockers

- Have a condition or be in a situation which in the Investigator's opinion may put the
subject at significant risk, may confound results or may interfere significantly with
the patient's participation in the study

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/03/2007
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/05/2010
Sample size
Target
340
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Childrens Hospital at Westmead - Sydney
Recruitment hospital [2] 0 0
Sydney Childrens Hospital - Sydney
Recruitment hospital [3] 0 0
Royal Brisbane Children's Hospital - Brisbane
Recruitment hospital [4] 0 0
The Prince Charles Hospital - Brisbane
Recruitment hospital [5] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [6] 0 0
Royal Childrens Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
- Sydney
Recruitment postcode(s) [3] 0 0
4029 - Brisbane
Recruitment postcode(s) [4] 0 0
4032 - Brisbane
Recruitment postcode(s) [5] 0 0
- Adelaide
Recruitment postcode(s) [6] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
Ireland
State/province [1] 0 0
Dublin
Country [2] 0 0
United Kingdom
State/province [2] 0 0
Liverpool
Country [3] 0 0
United Kingdom
State/province [3] 0 0
Northern Ireland
Country [4] 0 0
United Kingdom
State/province [4] 0 0
Wales
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Birmingham
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Bristol
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Cambridge
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Leeds
Country [9] 0 0
United Kingdom
State/province [9] 0 0
London
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Newcastle
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Norwich
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Nottingham
Country [13] 0 0
United Kingdom
State/province [13] 0 0
Sheffield
Country [14] 0 0
United Kingdom
State/province [14] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Syntara
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to determine the efficacy and safety of chronic treatment with
inhaled dry powder mannitol in subjects with cystic fibrosis. Previous studies have
demonstrated an improvement in lung function related to small airways obstruction and a
significant improvement in respiratory symptoms and quality of life after a 2 week treatment
with mannitol. This current study seeks to support these early findings and to extend the
evidence to support its use as a mucoactive therapy in cystic fibrosis. In particular, the
hypothesis that enhanced mucus clearance will improve the lung function and clinical
presentation in this population, will be investigated. We also hypothesize that enhanced
mucociliary clearance will result in a sustained reduction in mucus load, thus providing less
opportunity for bacteria to proliferate, affording a reduction in antibiotic use and
hospitalizations. The initial 6 month blinded phase will be followed with an additional 6
months of open label treatment.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00446680
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Brett Charlton, MBBS
Address 0 0
Pharmaxis Ltd Australia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00446680