Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
This study has been completed.
Sponsor:
Pharmaxis
Information provided by:
Pharmaxis
ClinicalTrials.gov Identifier:
NCT00446680
First received: March 12, 2007
Last updated: June 23, 2010
Last verified: June 2010
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Cystic Fibrosis |
Drug: Mannitol Drug: placebo |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study |
Resource links provided by NLM:
Genetics Home Reference related topics:
cystic fibrosis
Drug Information available for:
Mannitol
U.S. FDA Resources
Further study details as provided by Pharmaxis:
Primary Outcome Measures:
- To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control [ Time Frame: 6 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- 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) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective) [ Time Frame: 6 months / 12 months ] [ Designated as safety issue: No ]
- Improves quality of life (key objective) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Reduces days on IV antibiotics, rescue oral or inhaled antibiotics [ Time Frame: 6 months / 12 months ] [ Designated as safety issue: No ]
- Reduces days in hospital due to pulmonary exacerbations [ Time Frame: 6 months / 12 months ] [ Designated as safety issue: No ]
- Improves other measures of lung function [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination) [ Time Frame: 6 months / 12 months ] [ Designated as safety issue: Yes ]
- Reduces hospital and community care costs [ Time Frame: 6 months / 12 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 340 |
| Study Start Date: | March 2007 |
| Study Completion Date: | May 2010 |
| Primary Completion Date: | May 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 1 |
Drug: Mannitol
400mg BD for 6 months followed by a 6 month open label period
|
| Placebo Comparator: 2 |
Drug: placebo
placebo BD for 6 months
|
Eligibility| Ages Eligible for Study: | 6 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00446680
Locations
| Australia, New South Wales | |
| Childrens Hospital at Westmead | |
| Sydney, New South Wales, Australia, 2145 | |
| Sydney Childrens Hospital | |
| Sydney, New South Wales, Australia | |
| Australia, Queensland | |
| Royal Brisbane Children's Hospital | |
| Brisbane, Queensland, Australia, 4029 | |
| The Prince Charles Hospital | |
| Brisbane, Queensland, Australia, 4032 | |
| Australia, South Australia | |
| Royal Adelaide Hospital | |
| Adelaide, South Australia, Australia | |
| Australia, Victoria | |
| Royal Childrens Hospital | |
| Melbourne, Victoria, Australia, 3052 | |
| Ireland | |
| 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 | |
| United Kingdom | |
| Alder Hey Children's Hospital | |
| West Derby, Liverpool, United Kingdom | |
| Belfast City Hospital | |
| Belfast, Northern Ireland, United Kingdom, BT9 7AB | |
| Llandough Hospital | |
| Cardiff, Wales, United Kingdom, CF64 2XX | |
| Children's Hospital for Wales | |
| Cardiff, Wales, United Kingdom, CF14 4XW | |
| Birmingham Children's Hospital | |
| Birmingham, United Kingdom | |
| Birmingham Heartlands Hospital | |
| Birmingham, United Kingdom | |
| Bristol Royal Infirmary | |
| Bristol, United Kingdom | |
| Bristol Royal Hospital for Children | |
| Bristol, United Kingdom | |
| Addenbrooke's Hospital | |
| Cambridge, United Kingdom | |
| Papworth Hospital | |
| Cambridge, United Kingdom | |
| Seacroft Hospital | |
| Leeds, United Kingdom | |
| Cardiothoracic Centre | |
| Liverpool, United Kingdom, L14 3PE | |
| The London Chest Hospital | |
| London, United Kingdom, E2 9JX | |
| Freeman Hospital | |
| Newcastle, United Kingdom, NE7 7DN | |
| Norfolk and Norwich University Hospital | |
| Norwich, United Kingdom, NR4 7UY | |
| 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 | |
Sponsors and Collaborators
Pharmaxis
Investigators
| Study Director: | Brett Charlton, MBBS | Pharmaxis Ltd Australia |
| Principal Investigator: | Dr Diana Bilton | Papworth Hospital Cambridge UK |
| Principal Investigator: | Dr Philip Robinson | Royal Children's Hospital Melbourne Australia |
More Information
No publications provided by Pharmaxis
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Brett Charlton, Pharmaxis Ltd |
| ClinicalTrials.gov Identifier: | NCT00446680 History of Changes |
| Other Study ID Numbers: | DPM-CF-301 |
| Study First Received: | March 12, 2007 |
| Last Updated: | June 23, 2010 |
| Health Authority: | Australia: Department of Health and Ageing Therapeutic Goods Administration United Kingdom: Medicines and Healthcare Products Regulatory Agency Ireland: Ministry of Health New Zealand: Medsafe |
Keywords provided by Pharmaxis:
|
Mannitol Cystic Fibrosis Mucolytic |
Exacerbation FEV1 Quality of Life |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Pathologic Processes |
Mannitol Diuretics, Osmotic Diuretics Natriuretic Agents Physiological Effects of Drugs Pharmacologic Actions Cardiovascular Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on May 16, 2013