Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

Syntara

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: mannitol + pulmozyme; Drug: mannitol; Drug: Dornase alpha

Study type: Interventional

Funder types: Industry

Identifiers

NCT00117208

DPM-CF-203

Details and patient eligibility

About

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.

Enrollment: 20 patients

Sex: All

Ages: 8 to 18 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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20 participants in 3 patient groups

1

Experimental group

Treatment:

Drug: mannitol

2

Active Comparator group

Description:

DNase daily for 12 weeks

Treatment:

Drug: Dornase alpha

3

Other group

Description:

combination

Treatment:

Drug: mannitol + pulmozyme