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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00446680
• Other study ID #: DPM-CF-301
• Status: Completed
• Phase: Phase 3
• First received: March 12, 2007
• Last updated: June 23, 2010
• Start date: March 2007
• Est. completion date: May 2010

Study information

• Verified date: June 2010
• Source: Pharmaxis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Australia: Department of Health and Ageing Therapeutic Goods AdministrationUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyIreland: Ministry of HealthNew Zealand: Medsafe
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 340
• Est. completion date: May 2010
• Est. primary completion date: May 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Mannitol
400mg BD for 6 months followed by a 6 month open label period
• placebo
placebo BD for 6 months

Locations

Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide	South Australia
Australia	Royal Brisbane Children's Hospital	Brisbane	Queensland
Australia	The Prince Charles Hospital	Brisbane	Queensland
Australia	Royal Childrens Hospital	Melbourne	Victoria
Australia	Childrens Hospital at Westmead	Sydney	New South Wales
Australia	Sydney Childrens Hospital	Sydney	New South Wales
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
United Kingdom	Belfast City Hospital	Belfast	Northern Ireland
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Birmingham Heartlands Hospital	Birmingham
United Kingdom	Bristol Royal Hospital for Children	Bristol
United Kingdom	Bristol Royal Infirmary	Bristol
United Kingdom	Addenbrooke's Hospital	Cambridge
United Kingdom	Papworth Hospital	Cambridge
United Kingdom	Children's Hospital for Wales	Cardiff	Wales
United Kingdom	Llandough Hospital	Cardiff	Wales
United Kingdom	Seacroft Hospital	Leeds
United Kingdom	Cardiothoracic Centre	Liverpool
United Kingdom	The London Chest Hospital	London
United Kingdom	Freeman Hospital	Newcastle
United Kingdom	Norfolk and Norwich University Hospital	Norwich
United Kingdom	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	Alder Hey Children's Hospital	West Derby	Liverpool

Sponsors (1)

Lead Sponsor	Collaborator
Pharmaxis

Countries where clinical trial is conducted

Australia,  Ireland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control		6 months	No
Secondary	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)		6 months	No
Secondary	Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)		6 months / 12 months	No
Secondary	Improves quality of life (key objective)		6 months	No
Secondary	Reduces days on IV antibiotics, rescue oral or inhaled antibiotics		6 months / 12 months	No
Secondary	Reduces days in hospital due to pulmonary exacerbations		6 months / 12 months	No
Secondary	Improves other measures of lung function		6 months	No
Secondary	Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)		6 months / 12 months	Yes
Secondary	Reduces hospital and community care costs		6 months / 12 months	No