Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00117208
• Other study ID #: DPM-CF-203
• Status: Completed
• Phase: Phase 2
• First received: June 30, 2005
• Last updated: January 31, 2010
• Start date: November 2005
• Est. completion date: February 2008

Study information

• Verified date: January 2010
• Source: Pharmaxis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: February 2008
• Est. primary completion date: February 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 8 Years to 18 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• mannitol
400mg BD for 12 weeks
• mannitol + pulmozyme
combination
• Dornase alpha
2.5mg daily for 2 weeks

Locations

Country	Name	City	State
United Kingdom	Great Ormond Hospital for Children	London
United Kingdom	Royal Brompton Hospital	London

Sponsors (1)

Lead Sponsor	Collaborator
Pharmaxis

Country where clinical trial is conducted

United Kingdom, 

References & Publications (8)

Daviskas E, Anderson SD, Brannan JD, Chan HK, Eberl S, Bautovich G. Inhalation of dry-powder mannitol increases mucociliary clearance. Eur Respir J. 1997 Nov;10(11):2449-54. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

Minasian C, Wallis C, Metcalfe C, Bush A. Bronchial provocation testing with dry powder mannitol in children with cystic fibrosis. Pediatr Pulmonol. 2008 Nov;43(11):1078-84. doi: 10.1002/ppul.20903. — View Citation

Minasian C, Wallis C, Metcalfe C, Bush A. Comparison of inhaled mannitol, daily rhDNase and a combination of both in children with cystic fibrosis: a randomised trial. Thorax. 2010 Jan;65(1):51-6. doi: 10.1136/thx.2009.116970. Epub 2009 Dec 8. — View Citation

Robinson M, Daviskas E, Eberl S, Baker J, Chan HK, Anderson SD, Bye PT. The effect of inhaled mannitol on bronchial mucus clearance in cystic fibrosis patients: a pilot study. Eur Respir J. 1999 Sep;14(3):678-85. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase		12 weeks
Secondary	to compare mannitol to rhDNase on FVC		12 weeks
Secondary	to assess whether the effects of mannitol are additive to rhDNase		12 weeks
Secondary	to demonstrate that mannitol does not cause deterioration in airway inflammation		12 weeks
Secondary	to assess whether mannitol reduces the bacterial load in the lung		12 weeks
Secondary	to assess whether the effects of mannitol are beneficial to quality of life		12 weeks
Secondary	to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone		12 weeks

External Links

•   Pharmaxis homepage