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

June 23, 2010 updated by: Pharmaxis
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

340

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2145
        • Childrens Hospital at Westmead
      • Sydney, New South Wales, Australia
        • Sydney Childrens Hospital
    • Queensland
      • Brisbane, Queensland, Australia, 4032
        • The Prince Charles Hospital
      • Brisbane, Queensland, Australia, 4029
        • Royal Brisbane Children's Hospital
    • South Australia
      • Adelaide, South Australia, Australia
        • Royal Adelaide Hospital
    • Victoria
      • Melbourne, Victoria, Australia, 3052
        • Royal Childrens Hospital
  • Ireland
      • Dublin, Ireland
        • Beaumont Hospital
      • Dublin, Ireland
        • St Vincent's University Hospital
      • Dublin, Ireland
        • Our Lady's Hospital For Sick Children
      • Dublin, Ireland
        • National Children's Hospital
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Heartlands Hospital
      • Birmingham, United Kingdom
        • Birmingham Children's Hospital
      • Bristol, United Kingdom
        • Bristol Royal Hospital for Children
      • Bristol, United Kingdom
        • Bristol Royal Infirmary
      • Cambridge, United Kingdom
        • Papworth Hospital
      • Cambridge, United Kingdom
        • Addenbrooke's Hospital
      • Leeds, United Kingdom
        • Seacroft Hospital
      • Liverpool, United Kingdom, L14 3PE
        • Cardiothoracic Centre
      • London, United Kingdom, E2 9JX
        • The London Chest Hospital
      • Newcastle, United Kingdom, NE7 7DN
        • Freeman Hospital
      • Norwich, United Kingdom, NR4 7UY
        • Norfolk and Norwich University Hospital
      • Nottingham, United Kingdom
        • Nottingham City Hospital
      • Sheffield, United Kingdom
        • Northern General Hospital
      • Sheffield, United Kingdom
        • Sheffield Children's Hospital
      • Southampton, United Kingdom
        • Southampton General Hospital
    • Liverpool
      • West Derby, Liverpool, United Kingdom
        • Alder Hey Children's Hospital
    • Northern Ireland
      • Belfast, Northern Ireland, United Kingdom, BT9 7AB
        • Belfast City Hospital
    • Wales
      • Cardiff, Wales, United Kingdom, CF64 2XX
        • LLandough Hospital
      • Cardiff, Wales, United Kingdom, CF14 4XW
        • Children's Hospital for Wales

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
6 months

Secondary Outcome Measures

Outcome Measure
Time Frame
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
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
6 months / 12 months
Improves quality of life (key objective)
Time Frame: 6 months
6 months
Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
Time Frame: 6 months / 12 months
6 months / 12 months
Reduces days in hospital due to pulmonary exacerbations
Time Frame: 6 months / 12 months
6 months / 12 months
Improves other measures of lung function
Time Frame: 6 months
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
6 months / 12 months
Reduces hospital and community care costs
Time Frame: 6 months / 12 months
6 months / 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2007

Primary Completion (Actual)

May 1, 2010

Study Completion (Actual)

May 1, 2010

Study Registration Dates

First Submitted

March 12, 2007

First Submitted That Met QC Criteria

March 12, 2007

First Posted (Estimate)

March 13, 2007

Study Record Updates

Last Update Posted (Estimate)

June 25, 2010

Last Update Submitted That Met QC Criteria

June 23, 2010

Last Verified

June 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DPM-CF-301

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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