Cystic FibrosisPhase 2NCT00455130
A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis
A Phase 2 study testing Inhaled mannitol in people with Cystic Fibrosis, run by Syntara at 8 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Inhaled mannitol
- How many people
- Not given
- Where
- 8 sitesin 2 countries
- Study dates
- Mar 2004to Aug 2005
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of cystic fibrosis (sweat test/genotype)
- Aged 8 years or older
- 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.
- 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
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- Able to administer the dry powder mannitol
- Are capable of and have given informed consent
- Clinically stable at study entry
Who cannot join
- Investigators, site personnel directly affiliated with this study, and their immediate families.
- Subjects under the age of 8 years.
- Subjects with currently active asthma
- Subjects using hypertonic saline treatment in the last 2 weeks
- Considered "terminally ill" or listed for transplantation
- Requiring home oxygen or assisted ventilation
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- Colonisation with Burkholderia cepacia
- Significant episode of hemoptysis (>60 mls) in the previous 12 months
- Myocardial Infarction in the six months prior to enrolment.
- Cerebral Vascular Accident in the six months prior to enrolment.
- Ocular surgery in the three months prior to enrolment.
- Abdominal surgery in the three months prior to enrolment.
- Subjects who are breast feeding or pregnant.
- Female subjects of reproductive capability, not using a reliable form of contraception
- Inability to obtain informed consent from the subject or subject's authorised representative.
- Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
- Known intolerance to mannitol or beta2 agonists.
- Uncontrolled hypertension - systolic BP > 160 and or diastoli
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
Australia7 sites
Prince Charles Hospital
Brisbane, Queensland
Royal Children's Hospital
Melbourne, Victoria, 3052
The Alfred Hospital
Melbourne, Victoria, 3181
Princess Margaret Hospital for Children
Perth, Western Australia, 6840
Sir Charles Gairdner
Perth, Western Australia
Royal Prince Alfred Hospital
Sydney, New South Wales, 2050
Childrens Hospital at Westmead
Sydney, New South Wales, 2145
About this study
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.
- Conditions
- Cystic Fibrosis
- Being tested
- Inhaled mannitol
- Keywords
- mannitol, cystic fibrosis, mucolytic, mucoactive
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inhaled mannitolDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
FEV1
Secondary outcomes (5)
Other measures of lung function
Quality of life
Sputum microbiology
Sputum rheology
Safety
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2007 (estimated)
- Study startMar 2004
- Study completionAug 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Sponsor and investigators
- Lead sponsor
- Syntara
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Brett Charlton, Study director, Syntara
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00455130
- Sponsor's protocol ID
- DPM-CF-201
- Status last verified
- Jan 2010
- First submitted
- 2 Apr 2007
- Last updated
- 2 Feb 2010
- Results posted
- No
Publications
Jaques A, Daviskas E, Turton JA, McKay K, Cooper P, Stirling RG, Robertson CF, Bye PTP, LeSouef PN, Shadbolt B, Anderson SD, Charlton B. Inhaled mannitol improves lung function in cystic fibrosis. Chest. 2008 Jun;133(6):1388-1396. doi: 10.1378/chest.07-2294. Epub 2008 Mar 13.
PubMed 18339790Daviskas E, Anderson SD, Jaques A, Charlton B. Inhaled mannitol improves the hydration and surface properties of sputum in patients with cystic fibrosis. Chest. 2010 Apr;137(4):861-8. doi: 10.1378/chest.09-2017. Epub 2009 Oct 31.
PubMed 19880909
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00455130
- Acronym / sponsor ID
- DPM-CF-201
- Conditions
- Cystic Fibrosis
- Keywords
- mannitol, cystic fibrosis, mucolytic, mucoactive
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- SyntaraIndustry (pharma, biotech, devices)
- Study start
- Mar 2004
- Study completion
- Aug 2005 (actual)
- First posted
- 3 Apr 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Confirmed diagnosis of cystic fibrosis (sweat test/genotype)
- Aged 8 years or older
- 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.
- As determined by the investigator, are capable and willing to
- Are capable of and have given informed consent
Key exclusion criteria (18 listed)
- Investigators, site personnel directly affiliated with this study, and their immediate families.
- Subjects under the age of 8 years.
- Subjects with currently active asthma
- Subjects using hypertonic saline treatment in the last 2 weeks
- Considered "terminally ill" or listed for transplantation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Inhaled mannitol | Drug | – |
Primary outcome
FEV1
5 secondary outcomes
Other measures of lung function
Quality of life
Sputum microbiology
Sputum rheology
Safety
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2010.
- Results publication: PubMed 18339790Jaques A, Daviskas E, Turton JA, McKay K, Cooper P, Stirling RG, Robertson CF, Bye PTP, LeSouef PN, Shadbolt B, Anderson SD, Charlton B. Inhaled mannitol improves lung function in cystic fibrosis. Chest. 2008 Jun;133(6):1388-1396. doi: 10.1378/chest.07-2294. Epub 2008 Mar 13.
- Results publication: PubMed 19880909Daviskas E, Anderson SD, Jaques A, Charlton B. Inhaled mannitol improves the hydration and surface properties of sputum in patients with cystic fibrosis. Chest. 2010 Apr;137(4):861-8. doi: 10.1378/chest.09-2017. Epub 2009 Oct 31.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Brett Charlton study director
Syntara
8 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Inhaled mannitol in people with Cystic Fibrosis, run by Syntara at 8 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Feb 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Australia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
FEV1. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00455130, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Feb 2010; AzTrials last refreshed its copy on 31 Oct 2026.