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Mannitol Dose Response Study 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: NCT00251056
Recruitment Status : Completed
First Posted : November 9, 2005
Last Update Posted : August 29, 2008
Information provided by:

Brief Summary:
Many cystic fibrosis patients die of lung failure caused by repeated lung infections from thick, sticky mucus. Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology and replenishing the airway surface liquid layer in these patients, thereby enhancing the shift of stagnant mucus from the lungs. The study aim is to determine the optimal dose of mannitol to generate clinical improvement in patients with cystic fibrosis.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 48 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase IIa Randomised, Open Label, Dose Response Study to Determine the Optimum Dose of Dry Powder Mannitol Required to Generate Clinical Improvement In Patients With Cystic Fibrosis
Study Start Date : October 2005
Actual Primary Completion Date : August 2008
Actual Study Completion Date : August 2008

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Active Comparator: 1 Drug: mannitol
40 mg BD

Active Comparator: 2 Drug: mannitol
120mg BD

Active Comparator: 3 Drug: mannitol
240mg BD

Active Comparator: 4 Drug: mannitol
400mg BD

Primary Outcome Measures :
  1. FEV1 [ Time Frame: 2 weeks ]
  2. FVC [ Time Frame: 2 weeks ]

Secondary Outcome Measures :
  1. other measures of lung function [ Time Frame: various ]
  2. QOL [ Time Frame: 2 weeks ]
  3. sputum microbiology [ Time Frame: 2 weeks ]
  4. safety [ Time Frame: 2 weeks ]
  5. sputum clearance and cough [ Time Frame: 2 weeks ]
  6. respiratory symptoms [ Time Frame: 2 weeks ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of cystic fibrosis (sweat test/genotype)
  • 7 years or older
  • FEV1 between 40% and 90% of predicted for height, age and gender.
  • Able to perform acceptable-quality spirometry
  • Clinically stable in the week up to study entry
  • No additional antibiotics or additional oral steroids for a period of 14 days before study entry (routine antibiotics permitted)

Exclusion Criteria

  • Currently active asthma
  • Subjects colonized with Burkholderia cepacia or MRSA
  • Considered "terminally ill" or listed for transplantation
  • Requiring home oxygen or assisted ventilation
  • Concurrent illness that in the investigators opinion may contribute to an increased and unacceptable risk if the subject was enrolled in the study (e.g. significant varicies, portal hypertension, cor pulmonale)
  • Significant episode of haemoptysis (>60 mLs) in the previous 12 months
  • Heart attack or stroke in last 3 months
  • Known aortic or cerebral aneurysm
  • Subjects who are breast feeding or pregnant.
  • At risk females unwilling to use appropriate contraception to prevent pregnancy during the course of the study
  • Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
  • Known intolerance to mannitol or unable to take any form of bronchodilator medications.
  • Uncontrolled hypertension, systolic BP > 200 or diastolic BP> than 100
  • Concurrent use of beta blocker medication
  • Concurrent use of hypertonic saline


  • Concurrent use of other pharmacological mucolytic agents other than Pulmozyme


  • Concurrent use of other pharmacological mucolytic agents including Pulmozyme

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): NCT00251056

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Hospital de Niños Superiora Sor María Ludovica
La Plata, Buenos Aires, Argentina, B1904CSI
Hospital Pediatrico
Resistencia, Chaco, Argentina
Hospital Interzonal Especializado Materno Infantil (HIEMI)
Buenos Aires, Argentina
Hospital General de Niños
Caba, Argentina
Hospital Pediatrico Dr Humberto J Notti
Mendoza, Argentina
Canada, British Columbia
BC Children's Hospital
Vancouver, British Columbia, Canada, V6H 3V4
St Pauls Hospital
Vancouver, British Columbia, Canada, V6Z1Y6
Canada, Newfoundland and Labrador
Janeway Children's Health and Rehabilitation Center
St Johns, Newfoundland and Labrador, Canada, A1B 3V6
Canada, Nova Scotia
Queen Elizabeth II Health Sciences Centre
Halifax, Nova Scotia, Canada, B3H 3A7
Canada, Ontario
Hamilton Health Sciences Corporation
Hamilton, Ontario, Canada, L8N 3Z5
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
St Michaels Hospital
Toronto, Ontario, Canada
Sponsors and Collaborators
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Principal Investigator: Elizabeth Tullis, MD St Michaels Hospital, Toronto, Ontario, Canada
Study Director: Brett Charlton, MBBS PhD Pharmaxis Ltd, Sydney, NSW, Australia
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Responsible Party: Brett Charlton, Pharmaxis Ltd Identifier: NCT00251056    
Other Study ID Numbers: DPM-CF-202
First Posted: November 9, 2005    Key Record Dates
Last Update Posted: August 29, 2008
Last Verified: August 2008
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