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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00455130
Recruitment Status : Completed
First Posted : April 3, 2007
Last Update Posted : February 2, 2010
Information provided by:

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Inhaled mannitol Phase 2

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Study Type : Interventional  (Clinical Trial)
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double
Primary Purpose: Treatment
Study Start Date : March 2004
Actual Study Completion Date : August 2005

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Mannitol

Primary Outcome Measures :
  1. FEV1

Secondary Outcome Measures :
  1. Other measures of lung function
  2. Quality of life
  3. Sputum microbiology
  4. Sputum rheology
  5. Safety

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Confirmed diagnosis of cystic fibrosis (sweat test/genotype)
  2. Aged 8 years or older
  3. 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.
  4. 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
  5. Are capable of and have given informed consent
  6. Clinically stable at study entry

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00455130

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Australia, New South Wales
Royal Prince Alfred Hospital
Sydney, New South Wales, Australia, 2050
Childrens Hospital at Westmead
Sydney, New South Wales, Australia, 2145
Australia, Queensland
Prince Charles Hospital
Brisbane, Queensland, Australia
Australia, Victoria
Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
The Alfred Hospital
Melbourne, Victoria, Australia, 3181
Australia, Western Australia
Princess Margaret Hospital for Children
Perth, Western Australia, Australia, 6840
Sir Charles Gairdner
Perth, Western Australia, Australia
New Zealand
Greenlane Hospital
Auckland, North Island, New Zealand
Sponsors and Collaborators
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Study Director: Brett Charlton Pharmaxis
Publications of Results:
Layout table for additonal information Identifier: NCT00455130    
Other Study ID Numbers: DPM-CF-201
First Posted: April 3, 2007    Key Record Dates
Last Update Posted: February 2, 2010
Last Verified: January 2010
Keywords provided by Pharmaxis:
cystic fibrosis
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Diuretics, Osmotic
Natriuretic Agents
Physiological Effects of Drugs