Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

Study Overview
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Brief Summary
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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.

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

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: mannitol
* Drug: mannitol + pulmozyme
* Drug: Dornase alpha


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Drug: mannitol<br>* 400mg BD for 12 weeks<br>|
| Active Comparator: 2<br>DNase daily for 12 weeks | Drug: Dornase alpha<br>* 2.5mg daily for 2 weeks<br>* Other names: rhDNase, pulmozyme;|
| Other: 3<br>combination | Drug: mannitol + pulmozyme<br>* combination<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cystic fibrosis