Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

Sponsor

Pharmaxis (Industry)

Overall Status

Completed

CT.gov ID

NCT00117208

Collaborator

(none)

Enrollment

Locations

Arms

Duration (Months)

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: mannitol Drug: mannitol + pulmozyme Drug: Dornase alpha	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

20 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Nov 1, 2005

Actual Primary Completion Date :

Feb 1, 2008

Actual Study Completion Date :

Feb 1, 2008

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase [12 weeks]

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

8 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Great Ormond Hospital for Children	London		United Kingdom
2	Royal Brompton Hospital	London		United Kingdom

Sponsors and Collaborators

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