Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

January 31, 2010 updated by: Pharmaxis

A Cross-Over Comparative Study of Inhaled Mannitol, Alone and in Combination With Daily rhDNase, in Children With Cystic Fibrosis

The purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

20

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

  • United Kingdom
      • London, United Kingdom
        • Royal Brompton Hospital
      • London, United Kingdom
        • Great Ormond Hospital for Children

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

8 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Known diagnosis of cystic fibrosis (sweat test or genotype)
  • Of either gender
  • Aged between 8 and 18 years
  • Have a baseline FEV1 of <70% of the predicted normal value
  • Currently taking rhDNase for at least 4 weeks

Exclusion Criteria:

  • Currently active asthma, uncontrolled hypertension, colonised with Burkholderia cepacia or MRSA
  • Listed for transplantation
  • Known intolerance to mannitol, rhDNase or bronchodilators

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
Experimental: 1
Drug: mannitol
400mg BD for 12 weeks
Active Comparator: 2
DNase daily for 12 weeks
Drug: Dornase alpha
2.5mg daily for 2 weeks
Other Names:
  • rhDNase, pulmozyme
Other: 3
combination
Drug: mannitol + pulmozyme
combination

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase
Time Frame: 12 weeks
12 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
to compare mannitol to rhDNase on FVC
Time Frame: 12 weeks
12 weeks
to assess whether the effects of mannitol are additive to rhDNase
Time Frame: 12 weeks
12 weeks
to demonstrate that mannitol does not cause deterioration in airway inflammation
Time Frame: 12 weeks
12 weeks
to assess whether mannitol reduces the bacterial load in the lung
Time Frame: 12 weeks
12 weeks
to assess whether the effects of mannitol are beneficial to quality of life
Time Frame: 12 weeks
12 weeks
to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone
Time Frame: 12 weeks
12 weeks

Collaborators and Investigators

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

Sponsor

Pharmaxis

Investigators

  • Principal Investigator: Andrew Bush, FRCPCH, Royal Brompton and Harefiled NHS Trust
  • Principal Investigator: Colin Wallis, FRCPCH, Great Ormond Street Hospital For Children NHS Foundation Trust

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

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

November 1, 2005

Primary Completion (Actual)

February 1, 2008

Study Completion (Actual)

February 1, 2008

Study Registration Dates

First Submitted

June 30, 2005

First Submitted That Met QC Criteria

July 1, 2005

First Posted (Estimate)

July 4, 2005

Study Record Updates

Last Update Posted (Estimate)

February 2, 2010

Last Update Submitted That Met QC Criteria

January 31, 2010

Last Verified

January 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DPM-CF-203

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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