Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study

NCT ID: NCT00446680

Last Updated: 2010-06-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 340 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-03-31
• Study Completion Date: 2010-05-31

Brief Summary

The purpose of this study is to determine the efficacy and safety of chronic treatment with inhaled dry powder mannitol in subjects with cystic fibrosis. Previous studies have demonstrated an improvement in lung function related to small airways obstruction and a significant improvement in respiratory symptoms and quality of life after a 2 week treatment with mannitol. This current study seeks to support these early findings and to extend the evidence to support its use as a mucoactive therapy in cystic fibrosis. In particular, the hypothesis that enhanced mucus clearance will improve the lung function and clinical presentation in this population, will be investigated. We also hypothesize that enhanced mucociliary clearance will result in a sustained reduction in mucus load, thus providing less opportunity for bacteria to proliferate, affording a reduction in antibiotic use and hospitalizations. The initial 6 month blinded phase will be followed with an additional 6 months of open label treatment.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

1

Group Type: EXPERIMENTAL

Mannitol

Intervention Type: DRUG

400mg BD for 6 months followed by a 6 month open label period

2

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

placebo BD for 6 months

Interventions

Mannitol

400mg BD for 6 months followed by a 6 month open label period

Intervention Type: DRUG

placebo

placebo BD for 6 months

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Written informed consent
• Confirmed diagnosis of cystic fibrosis
• Aged > 6 years
• FEV1 >30 % and < 90% predicted
• Able to perform all the techniques necessary to measure lung function

Exclusion Criteria

• "Terminally ill" or listed for lung transplantation
• Had a lung transplant
• Using nebulised hypertonic saline
• Significant episode of haemoptysis (>60 mL) in the three months prior to enrolment
• Recent myocardial infarction or cerebral vascular accident
• Breast feeding or pregnant, or plan to become pregnant while in the study participating in another investigative drug study, parallel to, or within 4 weeks of study entry
• Allergy or intolerance to mannitol
• Using beta blockers
• Have a condition or be in a situation which in the Investigator's opinion may put the subject at significant risk, may confound results or may interfere significantly with the patient's participation in the study

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Syntara

INDUSTRY

Sponsor Role: lead

Responsible Party

Pharmaxis Ltd

Principal Investigators

Brett Charlton, MBBS

Role: STUDY_DIRECTOR

Pharmaxis Ltd Australia

Dr Diana Bilton

Role: PRINCIPAL_INVESTIGATOR

Papworth Hospital NHS Foundation Trust

Dr Philip Robinson

Role: PRINCIPAL_INVESTIGATOR

Royal Children's Hospital

Locations

Childrens Hospital at Westmead

Sydney, New South Wales, Australia

Sydney Childrens Hospital

Sydney, New South Wales, Australia

Royal Brisbane Children's Hospital

Brisbane, Queensland, Australia

The Prince Charles Hospital

Brisbane, Queensland, Australia

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Royal Childrens Hospital

Melbourne, Victoria, Australia

Beaumont Hospital

Dublin, , Ireland

National Children's Hospital

Dublin, , Ireland

Our Lady's Hospital for Sick Children

Dublin, , Ireland

St Vincent's University Hospital

Dublin, , Ireland

Alder Hey Children's Hospital

West Derby, Liverpool, United Kingdom

Belfast City Hospital

Belfast, Northern Ireland, United Kingdom

Children's Hospital for Wales

Cardiff, Wales, United Kingdom

Llandough Hospital

Cardiff, Wales, United Kingdom

Birmingham Children's Hospital

Birmingham, , United Kingdom

Birmingham Heartlands Hospital

Birmingham, , United Kingdom

Bristol Royal Hospital for Children

Bristol, , United Kingdom

Bristol Royal Infirmary

Bristol, , United Kingdom

Addenbrooke's Hospital

Cambridge, , United Kingdom

Papworth Hospital

Cambridge, , United Kingdom

Seacroft Hospital

Leeds, , United Kingdom

Cardiothoracic Centre

Liverpool, , United Kingdom

The London Chest Hospital

London, , United Kingdom

Freeman Hospital

Newcastle, , United Kingdom

Norfolk and Norwich University Hospital

Norwich, , United Kingdom

Nottingham City Hospital

Nottingham, , United Kingdom

Northern General Hospital

Sheffield, , United Kingdom

Sheffield Children's Hospital

Sheffield, , United Kingdom

Southampton General Hospital

Southampton, , United Kingdom

Countries

Australia; Ireland; United Kingdom

References

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)

Nevitt SJ, Thornton J, Murray CS, Dwyer T. Inhaled mannitol for cystic fibrosis. Cochrane Database Syst Rev. 2020 May 1;5(5):CD008649. doi: 10.1002/14651858.CD008649.pub4.

Reference Type: DERIVED

PMID: 32358807 (View on PubMed)

Bilton D, Robinson P, Cooper P, Gallagher CG, Kolbe J, Fox H, Jaques A, Charlton B; CF301 Study Investigators. Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study. Eur Respir J. 2011 Nov;38(5):1071-80. doi: 10.1183/09031936.00187510. Epub 2011 Apr 8.

Reference Type: DERIVED

PMID: 21478216 (View on PubMed)

Other Identifiers

DPM-CF-301

Identifier Type: -

Identifier Source: org_study_id