Mannitol Dose Response Study in Cystic Fibrosis

August 27, 2008 updated by: Pharmaxis

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

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.

Study Overview

Study Type

Interventional

Enrollment (Actual)

48

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Buenos Aires, Argentina
        • Hospital Interzonal Especializado Materno Infantil (HIEMI)
      • Caba, Argentina
        • Hospital General de Niños
      • Mendoza, Argentina
        • Hospital Pediatrico Dr Humberto J Notti
    • Buenos Aires
      • La Plata, Buenos Aires, Argentina, B1904CSI
        • Hospital de Niños Superiora Sor María Ludovica
    • Chaco
      • Resistencia, Chaco, Argentina
        • Hospital Pediatrico
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • BC Children's Hospital
      • Vancouver, British Columbia, Canada, V6Z1Y6
        • St Pauls Hospital
    • Newfoundland and Labrador
      • St Johns, Newfoundland and Labrador, Canada, A1B 3V6
        • Janeway Children's Health and Rehabilitation Center
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 3A7
        • Queen Elizabeth II Health Sciences Centre
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Hamilton Health Sciences Corporation
      • Toronto, Ontario, Canada, M5G 1X8
        • The Hospital for Sick Children
      • Toronto, Ontario, Canada
        • St Michaels Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

7 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: 2
120mg BD
40 mg BD
240mg BD
400mg BD
Active Comparator: 1
120mg BD
40 mg BD
240mg BD
400mg BD
Active Comparator: 3
120mg BD
40 mg BD
240mg BD
400mg BD
Active Comparator: 4
120mg BD
40 mg BD
240mg BD
400mg BD

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
FEV1
Time Frame: 2 weeks
2 weeks
FVC
Time Frame: 2 weeks
2 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
other measures of lung function
Time Frame: various
various
QOL
Time Frame: 2 weeks
2 weeks
sputum microbiology
Time Frame: 2 weeks
2 weeks
safety
Time Frame: 2 weeks
2 weeks
sputum clearance and cough
Time Frame: 2 weeks
2 weeks
respiratory symptoms
Time Frame: 2 weeks
2 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Investigators

  • Principal Investigator: Elizabeth Tullis, MD, St Michaels Hospital, Toronto, Ontario, Canada
  • Study Director: Brett Charlton, MBBS PhD, Pharmaxis Ltd, Sydney, NSW, Australia

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2005

Primary Completion (Actual)

August 1, 2008

Study Completion (Actual)

August 1, 2008

Study Registration Dates

First Submitted

June 30, 2005

First Submitted That Met QC Criteria

November 7, 2005

First Posted (Estimate)

November 9, 2005

Study Record Updates

Last Update Posted (Estimate)

August 29, 2008

Last Update Submitted That Met QC Criteria

August 27, 2008

Last Verified

August 1, 2008

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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