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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/NCT00455130




Registration number
NCT00455130
Ethics application status
Date submitted
2/04/2007
Date registered
3/04/2007
Date last updated
2/02/2010

Titles & IDs
Public title
A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis
Scientific title
Secondary ID [1] 0 0
DPM-CF-201
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 - Inhaled mannitol

Treatment: Drugs: Inhaled mannitol
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
FEV1
Timepoint [1] 0 0
Secondary outcome [1] 0 0
Other measures of lung function
Timepoint [1] 0 0
Secondary outcome [2] 0 0
Quality of life
Timepoint [2] 0 0
Secondary outcome [3] 0 0
Sputum microbiology
Timepoint [3] 0 0
Secondary outcome [4] 0 0
Sputum rheology
Timepoint [4] 0 0
Secondary outcome [5] 0 0
Safety
Timepoint [5] 0 0

Eligibility
Key inclusion criteria
1. Confirmed diagnosis of cystic fibrosis (sweat test/genotype)

2. Aged 8 years or older

3. Have FEV1 between 40% and 80% of predicted for height, age and gender OR a decrease in
FEV1 of 20% or more than that recorded 6-12 months previously.

4. As determined by the investigator, are capable and willing to

- Use the study diary as required for this protocol

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

- Able to administer the dry powder mannitol

5. Are capable of and have given informed consent

6. Clinically stable at study entry
Minimum age
8 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Investigators, site personnel directly affiliated with this study, and their immediate
families.

2. Subjects under the age of 8 years.

3. Subjects with currently active asthma

4. Subjects using hypertonic saline treatment in the last 2 weeks

5. Considered "terminally ill" or listed for transplantation

6. Requiring home oxygen or assisted ventilation

7. Colonisation with Burkholderia cepacia

8. Significant episode of hemoptysis (>60 mls) in the previous 12 months

9. Myocardial Infarction in the six months prior to enrolment.

10. Cerebral Vascular Accident in the six months prior to enrolment.

11. Ocular surgery in the three months prior to enrolment.

12. Abdominal surgery in the three months prior to enrolment.

13. Subjects who are breast feeding or pregnant.

14. Female subjects of reproductive capability, not using a reliable form of contraception

15. Inability to obtain informed consent from the subject or subject's authorised
representative.

16. Subjects who have participated in another investigative drug study parallel to, or
within 4 weeks of study entry.

17. Known intolerance to mannitol or beta2 agonists.

18. Uncontrolled hypertension - systolic BP > 160 and or diastoli

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?



Intervention assignment
Crossover
Other design features
Phase
Phase 2
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
1/03/2004
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2005
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Sydney
Recruitment hospital [2] 0 0
Childrens Hospital at Westmead - Sydney
Recruitment hospital [3] 0 0
Prince Charles Hospital - Brisbane
Recruitment hospital [4] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [5] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [6] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment hospital [7] 0 0
Sir Charles Gairdner - Perth
Recruitment postcode(s) [1] 0 0
2050 - Sydney
Recruitment postcode(s) [2] 0 0
2145 - Sydney
Recruitment postcode(s) [3] 0 0
- Brisbane
Recruitment postcode(s) [4] 0 0
3052 - Melbourne
Recruitment postcode(s) [5] 0 0
3181 - Melbourne
Recruitment postcode(s) [6] 0 0
6840 - Perth
Recruitment postcode(s) [7] 0 0
- Perth
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
North Island

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

Ethics approval
Ethics application status

Summary
Brief summary
Cystic fibrosis is the most frequent lethal genetic disease of childhood. Causes disruption
of glandular function of the pancreas, intestine, liver, lungs (causing chronic lung
infection with emphysema), sweat glands and reproductive organs. We know that many CF
patients die of lung failure, brought about in part by repeated lung infections caused by
thick, sticky mucus that cannot be readily cleared from the lung.

Inhaled mannitol is an osmotic agent that has been investigated in a number of small studies
that have examined mucociliary clearance, quality of life and lung function in CF and
bronchiectasis. The promising results of these studies warrant futher investigation. The aim
of this study is to assess the safety and efficacy of inhaled mannitol when administered
twice a day over two weeks in CF.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00455130
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Brett Charlton
Address 0 0
Syntara
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/NCT00455130