Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
1 other identifier
interventional
340
3 countries
29
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Mar 2007
Typical duration for phase_3
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 1, 2007
CompletedFirst Submitted
Initial submission to the registry
March 12, 2007
CompletedFirst Posted
Study publicly available on registry
March 13, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2010
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2010
CompletedJune 25, 2010
June 1, 2010
3.2 years
March 12, 2007
June 23, 2010
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
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)
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
- +3 more secondary outcomes
Study Arms (2)
1
EXPERIMENTAL2
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- "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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Syntaralead
Study Sites (29)
Childrens Hospital at Westmead
Sydney, New South Wales, 2145, Australia
Sydney Childrens Hospital
Sydney, New South Wales, Australia
Royal Brisbane Children's Hospital
Brisbane, Queensland, 4029, Australia
The Prince Charles Hospital
Brisbane, Queensland, 4032, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
Royal Childrens Hospital
Melbourne, Victoria, 3052, Australia
Beaumont Hospital
Dublin, Ireland
National Children's Hospital
Dublin, Ireland
Our Lady's Hospital for Sick Children
Dublin, Ireland
St Vincent's University Hospital
Dublin, Ireland
Alder Hey Children's Hospital
West Derby, Liverpool, United Kingdom
Belfast City Hospital
Belfast, Northern Ireland, BT9 7AB, United Kingdom
Children's Hospital for Wales
Cardiff, Wales, CF14 4XW, United Kingdom
Llandough Hospital
Cardiff, Wales, CF64 2XX, United Kingdom
Birmingham Children's Hospital
Birmingham, United Kingdom
Birmingham Heartlands Hospital
Birmingham, United Kingdom
Bristol Royal Hospital for Children
Bristol, United Kingdom
Bristol Royal Infirmary
Bristol, United Kingdom
Addenbrooke's Hospital
Cambridge, United Kingdom
Papworth Hospital
Cambridge, United Kingdom
Seacroft Hospital
Leeds, United Kingdom
Cardiothoracic Centre
Liverpool, L14 3PE, United Kingdom
The London Chest Hospital
London, E2 9JX, United Kingdom
Freeman Hospital
Newcastle, NE7 7DN, United Kingdom
Norfolk and Norwich University Hospital
Norwich, NR4 7UY, United Kingdom
Nottingham City Hospital
Nottingham, United Kingdom
Northern General Hospital
Sheffield, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom
Southampton General Hospital
Southampton, United Kingdom
Related Publications (3)
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.
PMID: 33735508DERIVEDNevitt 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.
PMID: 32358807DERIVEDBilton 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.
PMID: 21478216DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Brett Charlton, MBBS
Pharmaxis Ltd Australia
- PRINCIPAL INVESTIGATOR
Dr Diana Bilton
Papworth Hospital NHS Foundation Trust
- PRINCIPAL INVESTIGATOR
Dr Philip Robinson
Royal Children's Hospital
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
March 12, 2007
First Posted
March 13, 2007
Study Start
March 1, 2007
Primary Completion
May 1, 2010
Study Completion
May 1, 2010
Last Updated
June 25, 2010
Record last verified: 2010-06