Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis

NCT ID: NCT00117208

Last Updated: 2010-02-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-11-30

Study Completion Date

2008-02-29

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.

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Group Type EXPERIMENTAL

mannitol

Intervention Type DRUG

400mg BD for 12 weeks

2

DNase daily for 12 weeks

Group Type ACTIVE_COMPARATOR

Dornase alpha

Intervention Type DRUG

2.5mg daily for 2 weeks

3

combination

Group Type OTHER

mannitol + pulmozyme

Intervention Type DRUG

combination

Interventions

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mannitol

400mg BD for 12 weeks

Intervention Type DRUG

mannitol + pulmozyme

combination

Intervention Type DRUG

Dornase alpha

2.5mg daily for 2 weeks

Intervention Type DRUG

Other Intervention Names

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rhDNase, pulmozyme

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
Minimum Eligible Age

8 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Syntara

INDUSTRY

Sponsor Role lead

Principal Investigators

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Andrew Bush, FRCPCH

Role: PRINCIPAL_INVESTIGATOR

Royal Brompton and Harefiled NHS Trust

Colin Wallis, FRCPCH

Role: PRINCIPAL_INVESTIGATOR

Great Ormond Street Hospital for Children NHS Foundation Trust

Locations

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Great Ormond Hospital for Children

London, , United Kingdom

Site Status

Royal Brompton Hospital

London, , United Kingdom

Site Status

Countries

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United Kingdom

References

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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.

Reference Type BACKGROUND
PMID: 11684212 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 10543292 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 9426077 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 10351929 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 11171717 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15691238 (View on PubMed)

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.

Reference Type RESULT
PMID: 19996349 (View on PubMed)

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.

Reference Type RESULT
PMID: 18972410 (View on PubMed)

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.

Reference Type DERIVED
PMID: 33735508 (View on PubMed)

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.

Reference Type DERIVED
PMID: 17337677 (View on PubMed)

Other Identifiers

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DPM-CF-203

Identifier Type: -

Identifier Source: org_study_id

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