Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
- Registration Number
- NCT00446680
- Lead Sponsor
- Syntara
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 340
- 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
- "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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description 2 placebo - 1 Mannitol -
- Primary Outcome Measures
Name Time Method To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control 6 months
- Secondary Outcome Measures
Name Time Method 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 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 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
Trial Locations
- Locations (29)
National Children's Hospital
🇮🇪Dublin, Ireland
Our Lady's Hospital for Sick Children
🇮🇪Dublin, Ireland
Royal Childrens Hospital
🇦🇺Melbourne, Victoria, Australia
Belfast City Hospital
🇬🇧Belfast, Northern Ireland, United Kingdom
Children's Hospital for Wales
🇬🇧Cardiff, Wales, United Kingdom
Llandough Hospital
🇬🇧Cardiff, Wales, United Kingdom
Beaumont Hospital
🇮🇪Dublin, Ireland
Birmingham Heartlands Hospital
🇬🇧Birmingham, United Kingdom
Bristol Royal Infirmary
🇬🇧Bristol, United Kingdom
Addenbrooke's Hospital
🇬🇧Cambridge, United Kingdom
Royal Brisbane Children's Hospital
🇦🇺Brisbane, Queensland, Australia
Freeman Hospital
🇬🇧Newcastle, United Kingdom
Childrens Hospital at Westmead
🇦🇺Sydney, New South Wales, Australia
Sydney Childrens Hospital
🇦🇺Sydney, New South Wales, Australia
Royal Adelaide Hospital
🇦🇺Adelaide, South Australia, Australia
Alder Hey Children's Hospital
🇬🇧West Derby, Liverpool, United Kingdom
St Vincent's University Hospital
🇮🇪Dublin, Ireland
Bristol Royal Hospital for Children
🇬🇧Bristol, United Kingdom
The Prince Charles Hospital
🇦🇺Brisbane, Queensland, Australia
Seacroft Hospital
🇬🇧Leeds, United Kingdom
Cardiothoracic Centre
🇬🇧Liverpool, United Kingdom
Norfolk and Norwich University Hospital
🇬🇧Norwich, United Kingdom
Birmingham Children's Hospital
🇬🇧Birmingham, United Kingdom
Northern General Hospital
🇬🇧Sheffield, United Kingdom
The London Chest Hospital
🇬🇧London, United Kingdom
Nottingham City Hospital
🇬🇧Nottingham, United Kingdom
Papworth Hospital
🇬🇧Cambridge, United Kingdom
Sheffield Children's Hospital
🇬🇧Sheffield, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom