Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis
Information source: Pharmaxis
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Cystic Fibrosis
Intervention: mannitol (Drug); mannitol + pulmozyme (Drug); Dornase alpha (Drug)
Phase: Phase 2
Sponsored by: Pharmaxis
Official(s) and/or principal investigator(s):
Andrew Bush, FRCPCH, Principal Investigator, Affiliation: Royal Brompton and Harefiled NHS Trust
Colin Wallis, FRCPCH, Principal Investigator, Affiliation: Great Ormond Street Hospital for Children
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: A Cross-Over Comparative Study of Inhaled Mannitol, Alone and in Combination With Daily rhDNase, in Children With Cystic Fibrosis
Study design: Treatment, Randomized, Open Label, Uncontrolled, Crossover Assignment, Safety/Efficacy Study
Primary outcome: FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase
to compare mannitol to rhDNase on FVC
to assess whether the effects of mannitol are additive to rhDNase
to demonstrate that mannitol does not cause deterioration in airway inflammation
to assess whether mannitol reduces the bacterial load in the lung
to assess whether the effects of mannitol are beneficial to quality of life
to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone
Minimum age: 8 Years.
Maximum age: 18 Years.
- 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
- Currently active asthma, uncontrolled hypertension, colonised with Burkholderia
cepacia or MRSA
- Listed for transplantation
- Known intolerance to mannitol, rhDNase or bronchodilators
Locations and Contacts
Royal Brompton Hospital, London, United Kingdom
Great Ormond Hospital for Children, London, United Kingdom
Suri R, Metcalfe C, Lees B, Grieve R, Flather M, Normand C, Thompson S, Bush A, Wallis C. Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial. Lancet. 2001 Oct 20;358(9290):1316-21.
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Daviskas E, Anderson SD, Eberl S, Chan HK, Bautovich G. Inhalation of dry powder mannitol improves clearance of mucus in patients with bronchiectasis. Am J Respir Crit Care Med. 1999 Jun;159(6):1843-8.
Daviskas E, Anderson SD, Eberl S, Chan HK, Young IH. The 24-h effect of mannitol on the clearance of mucus in patients with bronchiectasis. Chest. 2001 Feb;119(2):414-21.
Daviskas E, Anderson SD, Gomes K, Briffa P, Cochrane B, Chan HK, Young IH, Rubin BK. Inhaled mannitol for the treatment of mucociliary dysfunction in patients with bronchiectasis: effect on lung function, health status and sputum. Respirology. 2005 Jan;10(1):46-56.
Starting date: November 2005
Ending date: February 2008
Last updated: February 27, 2008