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Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 2
Locations
United Kingdom
Study Type
Interventional
Intervention
mannitol
mannitol + pulmozyme
Dornase alpha
Sponsored by
Pharmaxis
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring cystic fibrosis

Eligibility Criteria

8 Years - 18 Years (Child, Adult)All SexesDoes not accept 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

Sites / Locations

  • Great Ormond Hospital for Children
  • Royal Brompton Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Active Comparator

Other

Arm Label

1

2

3

Arm Description

DNase daily for 12 weeks

combination

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
June 30, 2005
Last Updated
January 31, 2010
Sponsor
Pharmaxis
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1. Study Identification

Unique Protocol Identification Number
NCT00117208
Brief Title
Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis
Official Title
A Cross-Over Comparative Study of Inhaled Mannitol, Alone and in Combination With Daily rhDNase, in Children With Cystic Fibrosis
Study Type
Interventional

2. Study Status

Record Verification Date
January 2010
Overall Recruitment Status
Completed
Study Start Date
November 2005 (undefined)
Primary Completion Date
February 2008 (Actual)
Study Completion Date
February 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Pharmaxis

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
cystic fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Experimental
Arm Title
2
Arm Type
Active Comparator
Arm Description
DNase daily for 12 weeks
Arm Title
3
Arm Type
Other
Arm Description
combination
Intervention Type
Drug
Intervention Name(s)
mannitol
Intervention Description
400mg BD for 12 weeks
Intervention Type
Drug
Intervention Name(s)
mannitol + pulmozyme
Intervention Description
combination
Intervention Type
Drug
Intervention Name(s)
Dornase alpha
Other Intervention Name(s)
rhDNase, pulmozyme
Intervention Description
2.5mg daily for 2 weeks
Primary Outcome Measure Information:
Title
FEV1 after 12 weeks of each of the following treatment regimens: *mannitol only *rhDNase only *mannitol + rhDNase
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
to compare mannitol to rhDNase on FVC
Time Frame
12 weeks
Title
to assess whether the effects of mannitol are additive to rhDNase
Time Frame
12 weeks
Title
to demonstrate that mannitol does not cause deterioration in airway inflammation
Time Frame
12 weeks
Title
to assess whether mannitol reduces the bacterial load in the lung
Time Frame
12 weeks
Title
to assess whether the effects of mannitol are beneficial to quality of life
Time Frame
12 weeks
Title
to assess whether mannitol, or mannitol + rhDNase are cost-effective compared to rhDNase alone
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Andrew Bush, FRCPCH
Organizational Affiliation
Royal Brompton and Harefiled NHS Trust
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Colin Wallis, FRCPCH
Organizational Affiliation
Great Ormond Street Hospital for Children NHS Foundation Trust
Official's Role
Principal Investigator
Facility Information:
Facility Name
Great Ormond Hospital for Children
City
London
Country
United Kingdom
Facility Name
Royal Brompton Hospital
City
London
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
11684212
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. doi: 10.1016/S0140-6736(01)06412-1.
Results Reference
background
PubMed Identifier
10543292
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. doi: 10.1034/j.1399-3003.1999.14c30.x.
Results Reference
background
PubMed Identifier
9426077
Citation
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. doi: 10.1183/09031936.97.10112449.
Results Reference
background
PubMed Identifier
10351929
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. doi: 10.1164/ajrccm.159.6.9809074.
Results Reference
background
PubMed Identifier
11171717
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. doi: 10.1378/chest.119.2.414.
Results Reference
background
PubMed Identifier
15691238
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. doi: 10.1111/j.1440-1843.2005.00659.x.
Results Reference
background
PubMed Identifier
19996349
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.
Results Reference
result
PubMed Identifier
18972410
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):1078-1084. doi: 10.1002/ppul.20903.
Results Reference
result
PubMed Identifier
33735508
Citation
Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.
Results Reference
derived
PubMed Identifier
17337677
Citation
Enderby B, Doull I. Hypertonic saline inhalation in cystic fibrosis--salt in the wound, or sweet success? Arch Dis Child. 2007 Mar;92(3):195-6. doi: 10.1136/adc.2006.094979.
Results Reference
derived
Links:
URL
http://www.pharmaxis.com.au
Description
Pharmaxis homepage

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Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

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