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. 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.

Official Title

Conditions

Cystic Fibrosis

Study Type

Interventional

Study Design

Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Further Details

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]

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]
• Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective) [Time Frame: 6 months / 12 months]
• Improves quality of life (key objective) [Time Frame: 6 months]
• Reduces days on IV antibiotics, rescue oral or inhaled antibiotics [Time Frame: 6 months / 12 months]
• Reduces days in hospital due to pulmonary exacerbations [Time Frame: 6 months / 12 months]
• Improves other measures of lung function [Time Frame: 6 months]
• Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination) [Time Frame: 6 months / 12 months]
• Reduces hospital and community care costs [Time Frame: 6 months / 12 months]

Study Start

March 2007; Expected completion: December 2008

Eligibility & Criteria

• Ages Eligible for Study: 6 Years and above
• Genders Eligible for Study: Both

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

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

Total Enrolment

250

Contact Details

Childrens Hospital at Westmead, Sydney, New South Wales, 2145, Australia; Recruiting

• Karen McKay: KarenM8@chw.edu.au

Sydney Childrens Hospital, Sydney, New South Wales, Australia; Recruiting

• Barbara Donovan: Barbara.O’donovan@sesiahs.health.gov.au

Royal Brisbane Children’s Hospital, Brisbane, Queensland, 4029, Australia; Recruiting

• Helen Petsky, Dr: Helen_Petsky@health.qld.gov.au

The Prince Charles Hospital, Brisbane, Queensland, 4032, Australia; Recruiting

• Lisel Robbins: 07 3139 4193; Lisel_Robbins@health.qld.gov.au

Royal Adelaide Hospital, Adelaide, South Australia, Australia; Recruiting

• Kirsty Herewane: kherewan@mail.rah.sa.gov.au

Royal Childrens Hospital, Melbourne, Victoria, 3052, Australia; Recruiting

• Julie Smith: julie.smith@rch.org.au