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

This study has been completed.
Sponsor:
Information provided by:
Pharmaxis
ClinicalTrials.gov Identifier:
NCT00455130
First received: April 2, 2007
Last updated: January 31, 2010
Last verified: January 2010
  Purpose

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 Intervention Phase
Cystic Fibrosis
Drug: Inhaled mannitol
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Pharmaxis:

Primary Outcome Measures:
  • FEV1

Secondary Outcome Measures:
  • Other measures of lung function
  • Quality of life
  • Sputum microbiology
  • Sputum rheology
  • Safety

Study Start Date: March 2004
Study Completion Date: August 2005
  Eligibility

Ages Eligible for Study:   8 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00455130

Locations
Australia, New South Wales
Childrens Hospital at Westmead
Sydney, New South Wales, Australia, 2145
Royal Prince Alfred Hospital
Sydney, New South Wales, Australia, 2050
Australia, Queensland
Prince Charles Hospital
Brisbane, Queensland, Australia
Australia, Victoria
The Alfred Hospital
Melbourne, Victoria, Australia, 3181
Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
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
Pharmaxis
Investigators
Study Director: Brett Charlton Pharmaxis
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00455130     History of Changes
Other Study ID Numbers: DPM-CF-201
Study First Received: April 2, 2007
Last Updated: January 31, 2010
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
New Zealand: Medsafe

Keywords provided by Pharmaxis:
mannitol
cystic fibrosis
mucolytic
mucoactive

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes
Mannitol
Diuretics, Osmotic
Diuretics
Natriuretic Agents
Physiological Effects of Drugs
Pharmacologic Actions
Cardiovascular Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on April 23, 2014