Cystic FibrosisPhase 2NCT00251056
Mannitol Dose Response Study in Cystic Fibrosis
A Phase 2 study testing mannitol in people with Cystic Fibrosis, run by Syntara at 12 sites in 2 countries.
Trial summary
Many cystic fibrosis patients die of lung failure caused by repeated lung infections from thick, sticky mucus. Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology and replenishing the airway surface liquid layer in these patients, thereby enhancing the… Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- mannitol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 12 sitesin 2 countries
- Study dates
- Oct 2005to Aug 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 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of cystic fibrosis (sweat test/genotype)
- 7 years or older
- FEV1 between 40% and 90% of predicted for height, age and gender.
- Able to perform acceptable-quality spirometry
- Clinically stable in the week up to study entry
- No additional antibiotics or additional oral steroids for a period of 14 days before study entry (routine antibiotics permitted)
Who cannot join
- Currently active asthma
- Subjects colonized with Burkholderia cepacia or MRSA
- Considered "terminally ill" or listed for transplantation
- Requiring home oxygen or assisted ventilation
- Concurrent illness that in the investigators opinion may contribute to an increased and unacceptable risk if the subject was enrolled in the study (e.g. significant varicies, portal hypertension, cor pulmonale)
- Significant episode of haemoptysis (>60 mLs) in the previous 12 months
Show all 19Show fewer
- Heart attack or stroke in last 3 months
- Known aortic or cerebral aneurysm
- Subjects who are breast feeding or pregnant.
- At risk females unwilling to use appropriate contraception to prevent pregnancy during the course of the study
- Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
- Known intolerance to mannitol or unable to take any form of bronchodilator medications.
- Uncontrolled hypertension, systolic BP > 200 or diastolic BP> than 100
- Concurrent use of beta blocker medication
- Concurrent use of hypertonic saline
- Canada:
- Concurrent use of other pharmacological mucolytic agents other than Pulmozyme
- Argentina:
- Concurrent use of other pharmacological mucolytic agents including Pulmozyme
Trial locations and contacts
12 sites in 2 countries.
12 sites in 2 countries
Canada7 sites
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, B3H 3A7
Hamilton Health Sciences Corporation
Hamilton, Ontario, L8N 3Z5
Janeway Children's Health and Rehabilitation Center
St. John's, Newfoundland and Labrador, A1B 3V6
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
St Michaels Hospital
Toronto, Ontario
BC Children's Hospital
Vancouver, British Columbia, V6H 3V4
St Pauls Hospital
Vancouver, British Columbia, V6Z1Y6
Argentina5 sites
Hospital Interzonal Especializado Materno Infantil (HIEMI)
Buenos Aires
Hospital General de Niños
CABA
Hospital de Niños Superiora Sor María Ludovica
La Plata, Buenos Aires, B1904CSI
Hospital Pediatrico Dr Humberto J Notti
Mendoza
Hospital Pediatrico
Resistencia, Chaco Province
About this study
Official title: A Phase IIa Randomised, Open Label, Dose Response Study to Determine the Optimum Dose of Dry Powder Mannitol Required to Generate Clinical Improvement In Patients With Cystic Fibrosis
Many cystic fibrosis patients die of lung failure caused by repeated lung infections from thick, sticky mucus. Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology and replenishing the airway surface liquid layer in these patients, thereby enhancing the shift of stagnant mucus from the lungs. The study aim is to determine the optimal dose of mannitol to generate clinical improvement in patients with cystic fibrosis.
- Conditions
- Cystic Fibrosis
- Being tested
- mannitol
- 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
120mg BD
mannitolDrug
40 mg BD
mannitolDrug
240mg BD
mannitolDrug
400mg BD
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | Not described Receives: Drug: mannitol |
| 2 | Active comparator | Not described Receives: Drug: mannitol |
| 3 | Active comparator | Not described Receives: Drug: mannitol |
| 4 | Active comparator | Not described Receives: Drug: mannitol |
What is being measured
The primary outcome is the main question the study is designed to answer.
FEV1
Measured: 2 weeks
FVC
Measured: 2 weeks
Secondary outcomes (6)
other measures of lung function
Measured: various
QOL
Measured: 2 weeks
sputum microbiology
Measured: 2 weeks
safety
Measured: 2 weeks
sputum clearance and cough
Measured: 2 weeks
respiratory symptoms
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2005 (estimated)
- Study startOct 2005
- Primary completionAug 2008
- Study completionAug 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Syntara
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Elizabeth Tullis, MD, Principal investigator, St Michaels Hospital, Toronto, Ontario, CanadaBrett Charlton, MBBS PhD, Study director, Pharmaxis Ltd, Sydney, NSW, Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00251056
- Sponsor's protocol ID
- DPM-CF-202
- Status last verified
- Aug 2008
- First submitted
- 30 Jun 2005
- Last updated
- 29 Aug 2008
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa Randomised, Open Label, Dose Response Study to Determine the Optimum Dose of Dry Powder Mannitol Required to Generate Clinical Improvement In Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00251056
- Acronym / sponsor ID
- DPM-CF-202
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- SyntaraIndustry (pharma, biotech, devices)
- Study start
- Oct 2005
- Primary completion
- Aug 2008 (actual)
- Study completion
- Aug 2008 (actual)
- First posted
- 9 Nov 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.
Many cystic fibrosis patients die of lung failure caused by repeated lung infections from thick, sticky mucus. Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology and replenishing the airway surface liquid layer in these patients, thereby enhancing the shift of stagnant mucus from the lungs. The study aim is to determine the optimal dose of mannitol to generate clinical improvement in patients with cystic fibrosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Drug: mannitol |
| 2 | Active comparator | Drug: mannitol |
| 3 | Active comparator | Drug: mannitol |
| 4 | Active comparator | Drug: mannitol |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of cystic fibrosis (sweat test/genotype)
- 7 years or older
- FEV1 between 40% and 90% of predicted for height, age and gender.
- Able to perform acceptable-quality spirometry
- Clinically stable in the week up to study entry
Key exclusion criteria (19 listed)
- Currently active asthma
- Subjects colonized with Burkholderia cepacia or MRSA
- Considered "terminally ill" or listed for transplantation
- Requiring home oxygen or assisted ventilation
- Concurrent illness that in the investigators opinion may contribute to an increased and unacceptable risk if the subject was enrolled in the study (e.g. significant varicies, portal hypertension, cor pulmonale)
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 |
|---|---|---|
| mannitol120mg BD | Drug | 2 |
| mannitol40 mg BD | Drug | 1 |
| mannitol240mg BD | Drug | 3 |
| mannitol400mg BD | Drug | 4 |
Primary outcome
FEV1
Time frame: 2 weeks
FVC
Time frame: 2 weeks
6 secondary outcomes
other measures of lung function
Time frame: various
QOL
Time frame: 2 weeks
sputum microbiology
Time frame: 2 weeks
safety
Time frame: 2 weeks
sputum clearance and cough
Time frame: 2 weeks
respiratory symptoms
Time frame: 2 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 29 Aug 2008.
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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.
Elizabeth Tullis, MD principal investigator
St Michaels Hospital, Toronto, Ontario, Canada
Brett Charlton, MBBS PhD study director
Pharmaxis Ltd, Sydney, NSW, Australia
12 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 mannitol in people with Cystic Fibrosis, run by Syntara at 12 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 6 inclusion and 19 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 29 Aug 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Canada, Argentina. 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 (time frame: 2 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Aug 2008; AzTrials last refreshed its copy on 31 Oct 2026.