Cystic FibrosisPhase 2NCT00117208
Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis
A Phase 2 study testing mannitol, mannitol + pulmozyme and Dornase alpha in people with Cystic Fibrosis, run by Syntara at 2 sites in United Kingdom.
Trial summary
The purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse… Read more about this study
- Who is it for?
- Ages 8 to 18 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- mannitol, mannitol + pulmozyme and Dornase alpha
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- Nov 2005to Feb 2008
- 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 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Known diagnosis of cystic fibrosis (sweat test or genotype)
- Of either gender
- Aged between 8 and 18 years
- Have a baseline FEV1 of <70% of the predicted normal value
- Currently taking rhDNase for at least 4 weeks
Who cannot join
- Currently active asthma, uncontrolled hypertension, colonised with Burkholderia cepacia or MRSA
- Listed for transplantation
- Known intolerance to mannitol, rhDNase or bronchodilators
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Great Ormond Hospital for Children
London
Royal Brompton Hospital
London
About this study
Official title: A Cross-Over Comparative Study of Inhaled Mannitol, Alone and in Combination With Daily rhDNase, in Children With Cystic Fibrosis
The purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.
- Conditions
- Cystic Fibrosis
- Being tested
- mannitol, mannitol + pulmozyme, Dornase alpha
- Keywords
- cystic fibrosis
- 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 12 weeks
mannitol + pulmozymeDrug
combination
Dornase alphaDrug
Also called rhDNase, pulmozyme
2.5mg daily for 2 weeks
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: mannitol |
| 2 | Active comparator | DNase daily for 12 weeks Receives: Drug: Dornase alpha |
| 3 | Other | combination Receives: Drug: mannitol + pulmozyme |
What is being measured
The primary outcome is the main question the study is designed to answer.
FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase
Measured: 12 weeks
Secondary outcomes (6)
to compare mannitol to rhDNase on FVC
Measured: 12 weeks
to assess whether the effects of mannitol are additive to rhDNase
Measured: 12 weeks
to demonstrate that mannitol does not cause deterioration in airway inflammation
Measured: 12 weeks
to assess whether mannitol reduces the bacterial load in the lung
Measured: 12 weeks
to assess whether the effects of mannitol are beneficial to quality of life
Measured: 12 weeks
to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2005 (estimated)
- Study startNov 2005
- Primary completionFeb 2008
- Study completionFeb 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)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
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Syntara
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Andrew Bush, FRCPCH, Principal investigator, Royal Brompton and Harefiled NHS TrustColin Wallis, FRCPCH, Principal investigator, Great Ormond Street Hospital for Children NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00117208
- Sponsor's protocol ID
- DPM-CF-203
- Status last verified
- Jan 2010
- First submitted
- 30 Jun 2005
- Last updated
- 2 Feb 2010
- Results posted
- No
Publications
Suri R, Metcalfe C, Lees B, Grieve R, Flather M, Normand C, Thompson S, Bush A, Wallis C. Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet. 2001 Oct 20;358(9290):1316-21. doi: 10.1016/S0140-6736(01)06412-1.
PubMed 11684212Robinson M, Daviskas E, Eberl S, Baker J, Chan HK, Anderson SD, Bye PT. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J. 1999 Sep;14(3):678-85. doi: 10.1034/j.1399-3003.1999.14c30.x.
PubMed 10543292Daviskas E, Anderson SD, Brannan JD, Chan HK, Eberl S, Bautovich G. Inhalation of dry-powder mannitol increases mucociliary clearance. Eur Respir J. 1997 Nov;10(11):2449-54. doi: 10.1183/09031936.97.10112449.
PubMed 9426077Daviskas E, Anderson SD, Eberl S, Chan HK, Bautovich G. Inhalation of dry powder mannitol improves clearance of mucus in patients with bronchiectasis. Am J Respir Crit Care Med. 1999 Jun;159(6):1843-8. doi: 10.1164/ajrccm.159.6.9809074.
PubMed 10351929Daviskas E, Anderson SD, Eberl S, Chan HK, Young IH. The 24-h effect of mannitol on the clearance of mucus in patients with bronchiectasis. Chest. 2001 Feb;119(2):414-21. doi: 10.1378/chest.119.2.414.
PubMed 11171717Daviskas E, Anderson SD, Gomes K, Briffa P, Cochrane B, Chan HK, Young IH, Rubin BK. Inhaled mannitol for the treatment of mucociliary dysfunction in patients with bronchiectasis: effect on lung function, health status and sputum. Respirology. 2005 Jan;10(1):46-56. doi: 10.1111/j.1440-1843.2005.00659.x.
PubMed 15691238Minasian C, Wallis C, Metcalfe C, Bush A. Comparison of inhaled mannitol, daily rhDNase and a combination of both in children with cystic fibrosis: a randomised trial. Thorax. 2010 Jan;65(1):51-6. doi: 10.1136/thx.2009.116970. Epub 2009 Dec 8.
PubMed 19996349Minasian C, Wallis C, Metcalfe C, Bush A. Bronchial provocation testing with dry powder mannitol in children with cystic fibrosis. Pediatr Pulmonol. 2008 Nov;43(11):1078-1084. doi: 10.1002/ppul.20903.
PubMed 18972410Yang 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 33735508Enderby B, Doull I. Hypertonic saline inhalation in cystic fibrosis--salt in the wound, or sweet success? Arch Dis Child. 2007 Mar;92(3):195-6. doi: 10.1136/adc.2006.094979.
PubMed 17337677
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 Cross-Over Comparative Study of Inhaled Mannitol, Alone and in Combination With Daily rhDNase, in Children With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00117208
- Acronym / sponsor ID
- DPM-CF-203
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- SyntaraIndustry (pharma, biotech, devices)
- Study start
- Nov 2005
- Primary completion
- Feb 2008 (actual)
- Study completion
- Feb 2008 (actual)
- First posted
- 4 Jul 2005 (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 medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: mannitol |
| 2 | Active comparator | Drug: Dornase alphaDNase daily for 12 weeks |
| 3 | Other | Drug: mannitol + pulmozymecombination |
Who may qualify
- Ages
- 8 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Known diagnosis of cystic fibrosis (sweat test or genotype)
- Of either gender
- Aged between 8 and 18 years
- Have a baseline FEV1 of <70% of the predicted normal value
- Currently taking rhDNase for at least 4 weeks
Key exclusion criteria (3 listed)
- Currently active asthma, uncontrolled hypertension, colonised with Burkholderia cepacia or MRSA
- Listed for transplantation
- Known intolerance to mannitol, rhDNase or bronchodilators
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 12 weeks | Drug | 1 |
| mannitol + pulmozymecombination | Drug | 3 |
| Dornase alphaAlso: rhDNase, pulmozyme2.5mg daily for 2 weeks | Drug | 2 |
Primary outcome
FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase
Time frame: 12 weeks
6 secondary outcomes
to compare mannitol to rhDNase on FVC
Time frame: 12 weeks
to assess whether the effects of mannitol are additive to rhDNase
Time frame: 12 weeks
to demonstrate that mannitol does not cause deterioration in airway inflammation
Time frame: 12 weeks
to assess whether mannitol reduces the bacterial load in the lung
Time frame: 12 weeks
to assess whether the effects of mannitol are beneficial to quality of life
Time frame: 12 weeks
to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone
Time frame: 12 weeks
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.
- Background: PubMed 11684212Suri R, Metcalfe C, Lees B, Grieve R, Flather M, Normand C, Thompson S, Bush A, Wallis C. Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet. 2001 Oct 20;358(9290):1316-21. doi: 10.1016/S0140-6736(01)06412-1.
- Background: PubMed 10543292Robinson M, Daviskas E, Eberl S, Baker J, Chan HK, Anderson SD, Bye PT. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J. 1999 Sep;14(3):678-85. doi: 10.1034/j.1399-3003.1999.14c30.x.
- Background: PubMed 9426077Daviskas E, Anderson SD, Brannan JD, Chan HK, Eberl S, Bautovich G. Inhalation of dry-powder mannitol increases mucociliary clearance. Eur Respir J. 1997 Nov;10(11):2449-54. doi: 10.1183/09031936.97.10112449.
- Background: PubMed 10351929Daviskas E, Anderson SD, Eberl S, Chan HK, Bautovich G. Inhalation of dry powder mannitol improves clearance of mucus in patients with bronchiectasis. Am J Respir Crit Care Med. 1999 Jun;159(6):1843-8. doi: 10.1164/ajrccm.159.6.9809074.
- Background: PubMed 11171717Daviskas E, Anderson SD, Eberl S, Chan HK, Young IH. The 24-h effect of mannitol on the clearance of mucus in patients with bronchiectasis. Chest. 2001 Feb;119(2):414-21. doi: 10.1378/chest.119.2.414.
- Background: PubMed 15691238Daviskas E, Anderson SD, Gomes K, Briffa P, Cochrane B, Chan HK, Young IH, Rubin BK. Inhaled mannitol for the treatment of mucociliary dysfunction in patients with bronchiectasis: effect on lung function, health status and sputum. Respirology. 2005 Jan;10(1):46-56. doi: 10.1111/j.1440-1843.2005.00659.x.
- Results publication: PubMed 19996349Minasian C, Wallis C, Metcalfe C, Bush A. Comparison of inhaled mannitol, daily rhDNase and a combination of both in children with cystic fibrosis: a randomised trial. Thorax. 2010 Jan;65(1):51-6. doi: 10.1136/thx.2009.116970. Epub 2009 Dec 8.
- Results publication: PubMed 18972410Minasian C, Wallis C, Metcalfe C, Bush A. Bronchial provocation testing with dry powder mannitol in children with cystic fibrosis. Pediatr Pulmonol. 2008 Nov;43(11):1078-1084. doi: 10.1002/ppul.20903.
- 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 17337677Enderby B, Doull I. Hypertonic saline inhalation in cystic fibrosis--salt in the wound, or sweet success? Arch Dis Child. 2007 Mar;92(3):195-6. doi: 10.1136/adc.2006.094979.
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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.
Andrew Bush, FRCPCH principal investigator
Royal Brompton and Harefiled NHS Trust
Colin Wallis, FRCPCH principal investigator
Great Ormond Street Hospital for Children NHS Foundation Trust
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing mannitol, mannitol + pulmozyme and Dornase alpha in people with Cystic Fibrosis, run by Syntara at 2 sites in United Kingdom.
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 5 inclusion and 3 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 2 sites in United Kingdom. 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 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase (time frame: 12 weeks). 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 NCT00117208, which also lists 10 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.