Cystic Fibrosis
Conditions
Keywords
Mannitol, Cystic Fibrosis, Mucolytic, Exacerbation, FEV1, Quality of Life
Brief 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 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.
Interventions
DRUGMannitol
400mg BD for 6 months followed by a 6 month open label period
DRUGplacebo
placebo BD for 6 months
Sponsors
Syntara
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control | 6 months |
Secondary
| Measure | Time frame |
|---|---|
| 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) | 6 months |
| Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective) | 6 months / 12 months |
| Improves quality of life (key objective) | 6 months |
| Reduces days on IV antibiotics, rescue oral or inhaled antibiotics | 6 months / 12 months |
| Reduces days in hospital due to pulmonary exacerbations | 6 months / 12 months |
| Improves other measures of lung function | 6 months |
| Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination) | 6 months / 12 months |
| Reduces hospital and community care costs | 6 months / 12 months |
Countries
Australia, Ireland, United Kingdom
Outcome results
None listed