Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study

This study has been completed.
Sponsor:
Information provided by:
Pharmaxis
ClinicalTrials.gov Identifier:
NCT00446680
First received: March 12, 2007
Last updated: June 23, 2010
Last verified: June 2010
  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:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 July 26, 2014