Cystic FibrosisPhase 3NCT00446680
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
A Phase 3 study testing Mannitol in people with Cystic Fibrosis, run by Syntara at 29 sites in 3 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Mannitol
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 340 participants
- Where
- 29 sitesin 3 countries
- Study dates
- Mar 2007to May 2010
- Phase
- Phase 3
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Main Inclusion Criteria:
- 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 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
Trial locations and contacts
29 sites in 3 countries.
29 sites in 3 countries
United Kingdom19 sites
Belfast City Hospital
Belfast, Northern Ireland, BT9 7AB
Birmingham Children's Hospital
Birmingham
Birmingham Heartlands Hospital
Birmingham
Bristol Royal Hospital for Children
Bristol
Bristol Royal Infirmary
Bristol
Addenbrooke's Hospital
Cambridge
Papworth Hospital
Cambridge
Children's Hospital for Wales
Cardiff, Wales, CF14 4XW
Llandough Hospital
Cardiff, Wales, CF64 2XX
Seacroft Hospital
Leeds
Cardiothoracic Centre
Liverpool, L14 3PE
The London Chest Hospital
London, E2 9JX
Freeman Hospital
Newcastle, NE7 7DN
Norfolk and Norwich University Hospital
Norwich, NR4 7UY
Nottingham City Hospital
Nottingham
Northern General Hospital
Sheffield
Sheffield Children's Hospital
Sheffield
Southampton General Hospital
Southampton
Alder Hey Children's Hospital
West Derby, Liverpool
Australia6 sites
Royal Adelaide Hospital
Adelaide, South Australia
Royal Brisbane Children's Hospital
Brisbane, Queensland, 4029
The Prince Charles Hospital
Brisbane, Queensland, 4032
Royal Childrens Hospital
Melbourne, Victoria, 3052
Childrens Hospital at Westmead
Sydney, New South Wales, 2145
Sydney Childrens Hospital
Sydney, New South Wales
Ireland4 sites
Beaumont Hospital
Dublin
National Children's Hospital
Dublin
Our Lady's Hospital for Sick Children
Dublin
St Vincent's University Hospital
Dublin
About this study
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.
- Conditions
- Cystic Fibrosis
- Being tested
- Mannitol, placebo
- Keywords
- Mannitol, Cystic Fibrosis, Mucolytic, Exacerbation, FEV1, Quality of Life
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MannitolDrug
400mg BD for 6 months followed by a 6 month open label period
placeboDrug
placebo BD for 6 months
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: Mannitol |
| 2 | Placebo comparator | Not described Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
Measured: 6 months
Secondary outcomes (8)
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)
Measured: 6 months
Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)
Measured: 6 months / 12 months
Improves quality of life (key objective)
Measured: 6 months
Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
Measured: 6 months / 12 months
Reduces days in hospital due to pulmonary exacerbations
Measured: 6 months / 12 months
Improves other measures of lung function
Measured: 6 months
Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)
Measured: 6 months / 12 months
Reduces hospital and community care costs
Measured: 6 months / 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2007 (estimated)
- Study startMar 2007
- Primary completionMay 2010
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 340 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Syntara
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Brett Charlton, MBBS, Study director, Pharmaxis Ltd AustraliaDr Diana Bilton, Principal investigator, Papworth Hospital NHS Foundation TrustDr Philip Robinson, Principal investigator, Royal Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00446680
- Sponsor's protocol ID
- DPM-CF-301
- Status last verified
- Jun 2010
- First submitted
- 12 Mar 2007
- Last updated
- 25 Jun 2010
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.
PubMed 33735508Nevitt SJ, Thornton J, Murray CS, Dwyer T. Inhaled mannitol for cystic fibrosis. Cochrane Database Syst Rev. 2020 May 1;5(5):CD008649. doi: 10.1002/14651858.CD008649.pub4.
PubMed 32358807Bilton D, Robinson P, Cooper P, Gallagher CG, Kolbe J, Fox H, Jaques A, Charlton B; CF301 Study Investigators. Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study. Eur Respir J. 2011 Nov;38(5):1071-80. doi: 10.1183/09031936.00187510. Epub 2011 Apr 8.
PubMed 21478216
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
- ClinicalTrials.gov ID
- NCT00446680
- Acronym / sponsor ID
- DPM-CF-301
- Conditions
- Cystic Fibrosis
- Keywords
- Mannitol, Cystic Fibrosis, Mucolytic, Exacerbation, FEV1, Quality of Life
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 340 participants (estimated)
- Lead sponsor
- SyntaraIndustry (pharma, biotech, devices)
- Study start
- Mar 2007
- Primary completion
- May 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 13 Mar 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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Mannitol |
| 2 | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| Mannitol400mg BD for 6 months followed by a 6 month open label period | Drug | 1 |
| placeboplacebo BD for 6 months | Drug | 2 |
Primary outcome
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
Time frame: 6 months
8 secondary outcomes
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)
Time frame: 6 months
Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)
Time frame: 6 months / 12 months
Improves quality of life (key objective)
Time frame: 6 months
Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
Time frame: 6 months / 12 months
Reduces days in hospital due to pulmonary exacerbations
Time frame: 6 months / 12 months
Improves other measures of lung function
Time frame: 6 months
Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)
Time frame: 6 months / 12 months
Reduces hospital and community care costs
Time frame: 6 months / 12 months
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 25 Jun 2010.
- Derived from this study: PubMed 33735508Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.
- Derived from this study: PubMed 32358807Nevitt SJ, Thornton J, Murray CS, Dwyer T. Inhaled mannitol for cystic fibrosis. Cochrane Database Syst Rev. 2020 May 1;5(5):CD008649. doi: 10.1002/14651858.CD008649.pub4.
- Derived from this study: PubMed 21478216Bilton D, Robinson P, Cooper P, Gallagher CG, Kolbe J, Fox H, Jaques A, Charlton B; CF301 Study Investigators. Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study. Eur Respir J. 2011 Nov;38(5):1071-80. doi: 10.1183/09031936.00187510. Epub 2011 Apr 8.
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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, MBBS study director
Pharmaxis Ltd Australia
Dr Diana Bilton principal investigator
Papworth Hospital NHS Foundation Trust
Dr Philip Robinson principal investigator
Royal Children's Hospital
29 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mannitol in people with Cystic Fibrosis, run by Syntara at 29 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 25 Jun 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United Kingdom, Australia, Ireland. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control (time frame: 6 months). 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 NCT00446680, which also lists 3 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 25 Jun 2010.