Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

Phase 2

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: mannitol; Drug: mannitol + pulmozyme; Drug: Dornase alpha

• Registration Number: NCT00117208
• Lead Sponsor: Syntara

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2005-06-30
• Study First Submitted QC: 2005-07-01
• Study First Posted: 2005-07-04
• Study Start: 2005-11
• Primary Completion: 2008-02
• Study Completion: 2008-02
• Status Verified: 2010-01
• Last Update Submitted: 2010-01-31
• Last Update Posted: 2010-02-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
1	mannitol	-
3	mannitol + pulmozyme	combination
2	Dornase alpha	DNase daily for 12 weeks

Primary Outcome Measures

Name	Time	Method
FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase	12 weeks

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (2)

Great Ormond Hospital for Children; 🇬🇧 London, United Kingdom

Royal Brompton Hospital; 🇬🇧 London, United Kingdom