A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis

January 31, 2010 updated by: Pharmaxis

Cystic fibrosis is the most frequent lethal genetic disease of childhood. Causes disruption of glandular function of the pancreas, intestine, liver, lungs (causing chronic lung infection with emphysema), sweat glands and reproductive organs. We know that many CF patients die of lung failure, brought about in part by repeated lung infections caused by thick, sticky mucus that cannot be readily cleared from the lung.

Inhaled mannitol is an osmotic agent that has been investigated in a number of small studies that have examined mucociliary clearance, quality of life and lung function in CF and bronchiectasis. The promising results of these studies warrant futher investigation. The aim of this study is to assess the safety and efficacy of inhaled mannitol when administered twice a day over two weeks in CF.

Study Overview

Status

Completed

Conditions

Cystic Fibrosis

Intervention / Treatment

Drug: Inhaled mannitol

Study Type

Interventional

Phase

Phase 2

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, 2050
    - Royal Prince Alfred Hospital
  - Sydney, New South Wales, Australia, 2145
    - Childrens Hospital at Westmead
- Queensland
  - Brisbane, Queensland, Australia
    - Prince Charles Hospital
- Victoria
  - Melbourne, Victoria, Australia, 3181
    - The Alfred Hospital
  - Melbourne, Victoria, Australia, 3052
    - Royal Children's Hospital
- Western Australia
  - Perth, Western Australia, Australia, 6840
    - Princess Margaret Hospital for Children
  - Perth, Western Australia, Australia
    - Sir Charles Gairdner
New Zealand
- North Island
  - Auckland, North Island, New Zealand
    - Greenlane Hospital

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

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

Accepts Healthy Volunteers

Genders Eligible for Study

All

Description

Inclusion Criteria:

Confirmed diagnosis of cystic fibrosis (sweat test/genotype)
Aged 8 years or older
Have FEV1 between 40% and 80% of predicted for height, age and gender OR a decrease in FEV1 of 20% or more than that recorded 6-12 months previously.
As determined by the investigator, are capable and willing to
- Use the study diary as required for this protocol
- Able to perform all of the techniques necessary to measure lung function
- Able to administer the dry powder mannitol
Are capable of and have given informed consent
Clinically stable at study entry

Exclusion Criteria:

Investigators, site personnel directly affiliated with this study, and their immediate families.
Subjects under the age of 8 years.
Subjects with currently active asthma
Subjects using hypertonic saline treatment in the last 2 weeks
Considered "terminally ill" or listed for transplantation
Requiring home oxygen or assisted ventilation
Colonisation with Burkholderia cepacia
Significant episode of hemoptysis (>60 mls) in the previous 12 months
Myocardial Infarction in the six months prior to enrolment.
Cerebral Vascular Accident in the six months prior to enrolment.
Ocular surgery in the three months prior to enrolment.
Abdominal surgery in the three months prior to enrolment.
Subjects who are breast feeding or pregnant.
Female subjects of reproductive capability, not using a reliable form of contraception
Inability to obtain informed consent from the subject or subject's authorised representative.
Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
Known intolerance to mannitol or beta2 agonists.
Uncontrolled hypertension - systolic BP > 160 and or diastoli

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: Crossover Assignment
Masking: Double

What is the study measuring?

Primary Outcome Measures

Outcome Measure
FEV1

Secondary Outcome Measures

Outcome Measure
Quality of life
Safety
Other measures of lung function
Sputum microbiology
Sputum rheology

Collaborators and Investigators

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

Sponsor

Pharmaxis

Investigators

Study Director: Brett Charlton, Pharmaxis

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, 2004

Study Completion (Actual)

August 1, 2005

Study Registration Dates

First Submitted

April 2, 2007

First Submitted That Met QC Criteria

April 2, 2007

First Posted (Estimate)

April 3, 2007

Study Record Updates

Last Update Posted (Estimate)

February 2, 2010

Last Update Submitted That Met QC Criteria

January 31, 2010

Last Verified

January 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

DPM-CF-201

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.