NCT00117208

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Nov 2005

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 30, 2005

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 4, 2005

Completed
4 months until next milestone

Study Start

First participant enrolled

November 1, 2005

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2008

Completed
Last Updated

February 2, 2010

Status Verified

January 1, 2010

Enrollment Period

2.3 years

First QC Date

June 30, 2005

Last Update Submit

January 31, 2010

Conditions

Cystic Fibrosis

Keywords

cystic fibrosis

Outcome Measures

Primary Outcomes (1)

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

    12 weeks

Secondary Outcomes (6)

  • 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

  • +1 more secondary outcomes

Study Arms (3)

1

EXPERIMENTAL
Drug: mannitol

2

ACTIVE COMPARATOR

DNase daily for 12 weeks

Drug: Dornase alpha

3

OTHER

combination

Drug: mannitol + pulmozyme

Interventions

mannitolDRUG

400mg BD for 12 weeks

1
mannitol + pulmozymeDRUG

combination

3
Dornase alphaDRUG

2.5mg daily for 2 weeks

Also known as: rhDNase, pulmozyme
2

Eligibility Criteria

Age8 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • Currently active asthma, uncontrolled hypertension, colonised with Burkholderia cepacia or MRSA
  • Listed for transplantation
  • Known intolerance to mannitol, rhDNase or bronchodilators

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Great Ormond Hospital for Children

London, United Kingdom

Location

Royal Brompton Hospital

London, United Kingdom

Location

Related Publications (10)

  • 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.

    PMID: 11684212BACKGROUND

  • 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.

    PMID: 10543292BACKGROUND

  • 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.

    PMID: 9426077BACKGROUND

  • 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.

    PMID: 10351929BACKGROUND

  • 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.

    PMID: 11171717BACKGROUND

  • 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.

    PMID: 15691238BACKGROUND

  • 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.

    PMID: 19996349RESULT

  • 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.

    PMID: 18972410RESULT

  • 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.

    PMID: 33735508DERIVED

  • 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.

    PMID: 17337677DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Mannitoldornase alfa

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Sugar AlcoholsAlcoholsOrganic ChemicalsCarbohydrates

Study Officials

  • Andrew Bush, FRCPCH

    Royal Brompton and Harefiled NHS Trust

    PRINCIPAL INVESTIGATOR

  • Colin Wallis, FRCPCH

    Great Ormond Street Hospital for Children NHS Foundation Trust

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

June 30, 2005

First Posted

July 4, 2005

Study Start

November 1, 2005

Primary Completion

February 1, 2008

Study Completion

February 1, 2008

Last Updated

February 2, 2010

Record last verified: 2010-01

Locations

GB(2)