Mannitol Dose Response Study in Cystic Fibrosis

This study has been completed.
Sponsor:
Information provided by:
Pharmaxis
ClinicalTrials.gov Identifier:
NCT00251056
First received: June 30, 2005
Last updated: August 27, 2008
Last verified: August 2008
  Purpose

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 Intervention Phase
Cystic Fibrosis
Drug: mannitol
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Pharmaxis:

Primary Outcome Measures:
  • FEV1 [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]
  • FVC [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • other measures of lung function [ Time Frame: various ] [ Designated as safety issue: No ]
  • QOL [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]
  • sputum microbiology [ Time Frame: 2 weeks ] [ Designated as safety issue: Yes ]
  • safety [ Time Frame: 2 weeks ] [ Designated as safety issue: Yes ]
  • sputum clearance and cough [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]
  • respiratory symptoms [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]

Enrollment: 48
Study Start Date: October 2005
Study Completion Date: August 2008
Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   7 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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

Canada:

  • Concurrent use of other pharmacological mucolytic agents other than Pulmozyme

Argentina:

  • Concurrent use of other pharmacological mucolytic agents including Pulmozyme
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00251056

Locations
Argentina
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
St Pauls Hospital
Vancouver, British Columbia, Canada, V6Z1Y6
BC Children's Hospital
Vancouver, British Columbia, Canada, V6H 3V4
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
St Michaels Hospital
Toronto, Ontario, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
Pharmaxis
Investigators
Principal Investigator: Elizabeth Tullis, MD St Michaels Hospital, Toronto, Ontario, Canada
Study Director: Brett Charlton, MBBS PhD Pharmaxis Ltd, Sydney, NSW, Australia
  More Information

No publications provided

Responsible Party: Brett Charlton, Pharmaxis Ltd
ClinicalTrials.gov Identifier: NCT00251056     History of Changes
Other Study ID Numbers: DPM-CF-202
Study First Received: June 30, 2005
Last Updated: August 27, 2008
Health Authority: Canada: Health Canada
Argentina: Human Research Bioethics Committee

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

ClinicalTrials.gov processed this record on September 18, 2014