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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

March 12, 2007
June 23, 2010
March 2007
May 2010   (final data collection date for primary outcome measure)
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 ]
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
Complete list of historical versions of study NCT00446680 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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)
  • To assess whether IDPM treatment:
  • Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)
  • Improves quality of life (key objective)
  • Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
  • Reduces days in hospital due to pulmonary exacerbations
  • Improves other measures of lung function
  • Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)
  • Reduces hospital and community care costs
Not Provided
Not Provided
 
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study

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.

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Cystic Fibrosis
  • Drug: Mannitol
    400mg BD for 6 months followed by a 6 month open label period
  • Drug: placebo
    placebo BD for 6 months
  • Experimental: 1
    Intervention: Drug: Mannitol
  • Placebo Comparator: 2
    Intervention: Drug: placebo
Bilton 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
340
May 2010
May 2010   (final data collection date for primary outcome measure)

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
Both
6 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Ireland,   United Kingdom
 
NCT00446680
DPM-CF-301
Yes
Brett Charlton, Pharmaxis Ltd
Pharmaxis
Not Provided
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
Pharmaxis
June 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP