NCT00455130

Brief Summary

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Timeline
Completed

Started Mar 2004

Shorter than P25 for phase_2

Geographic Reach
2 countries

8 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2004

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2005

Completed
1.7 years until next milestone

First Submitted

Initial submission to the registry

April 2, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 3, 2007

Completed
Last Updated

February 2, 2010

Status Verified

January 1, 2010

First QC Date

April 2, 2007

Last Update Submit

January 31, 2010

Conditions

Cystic Fibrosis

Keywords

mannitolcystic fibrosismucolyticmucoactive

Outcome Measures

Primary Outcomes (1)

  • FEV1

Secondary Outcomes (5)

  • Other measures of lung function

  • Quality of life

  • Sputum microbiology

  • Sputum rheology

  • Safety

Interventions

Inhaled mannitolDRUG

Eligibility Criteria

Age8 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Royal Prince Alfred Hospital

Sydney, New South Wales, 2050, Australia

Location

Childrens Hospital at Westmead

Sydney, New South Wales, 2145, Australia

Location

Prince Charles Hospital

Brisbane, Queensland, Australia

Location

Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3181, Australia

Location

Princess Margaret Hospital for Children

Perth, Western Australia, 6840, Australia

Location

Sir Charles Gairdner

Perth, Western Australia, Australia

Location

Greenlane Hospital

Auckland, North Island, New Zealand

Location

Related Publications (2)

  • Jaques A, Daviskas E, Turton JA, McKay K, Cooper P, Stirling RG, Robertson CF, Bye PTP, LeSouef PN, Shadbolt B, Anderson SD, Charlton B. Inhaled mannitol improves lung function in cystic fibrosis. Chest. 2008 Jun;133(6):1388-1396. doi: 10.1378/chest.07-2294. Epub 2008 Mar 13.

    PMID: 18339790RESULT

  • Daviskas E, Anderson SD, Jaques A, Charlton B. Inhaled mannitol improves the hydration and surface properties of sputum in patients with cystic fibrosis. Chest. 2010 Apr;137(4):861-8. doi: 10.1378/chest.09-2017. Epub 2009 Oct 31.

    PMID: 19880909RESULT

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Officials

  • Brett Charlton

    Syntara

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

April 2, 2007

First Posted

April 3, 2007

Study Start

March 1, 2004

Study Completion

August 1, 2005

Last Updated

February 2, 2010

Record last verified: 2010-01

Locations

AU(7)NZ(1)