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Completed

NCT Number: NCT00446680

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Childrens Hospital at Westmead, Sydney, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Mannitol

Drug

400mg BD for 6 months followed by a 6 month open label period

Placebo

Drug

placebo BD for 6 months

Primary outcomes

  1. 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

Secondary outcomes

  1. 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

  2. Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)

    Time frame: 6 months / 12 months

  3. Improves quality of life (key objective)

    Time frame: 6 months

  4. Reduces days on IV antibiotics, rescue oral or inhaled antibiotics

    Time frame: 6 months / 12 months

  5. Reduces days in hospital due to pulmonary exacerbations

    Time frame: 6 months / 12 months

  6. Improves other measures of lung function

    Time frame: 6 months

  7. Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)

    Time frame: 6 months / 12 months

  8. Reduces hospital and community care costs

    Time frame: 6 months / 12 months

Sponsors and collaborators

Lead sponsor

Syntara

Industry

Registry information

Important dates

Study start
2007
Primary completion
2010
Study completion
2010
First posted
Mar 13, 2007
Registry last updated
Jun 25, 2010

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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