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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Mannitol
placebo
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 Mannitol, Cystic Fibrosis, Mucolytic, Exacerbation, FEV1, Quality of Life

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Main 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

Main 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

Sites / Locations

  • Childrens Hospital at Westmead
  • Sydney Childrens Hospital
  • Royal Brisbane Children's Hospital
  • The Prince Charles Hospital
  • Royal Adelaide Hospital
  • Royal Childrens Hospital
  • Beaumont Hospital
  • National Children's Hospital
  • Our Lady's Hospital for Sick Children
  • St Vincent's University Hospital
  • Alder Hey Children's Hospital
  • Belfast City Hospital
  • Children's Hospital for Wales
  • Llandough Hospital
  • Birmingham Children's Hospital
  • Birmingham Heartlands Hospital
  • Bristol Royal Hospital for Children
  • Bristol Royal Infirmary
  • Addenbrooke's Hospital
  • Papworth Hospital
  • Seacroft Hospital
  • Cardiothoracic Centre
  • The London Chest Hospital
  • Freeman Hospital
  • Norfolk and Norwich University Hospital
  • Nottingham City Hospital
  • Northern General Hospital
  • Sheffield Children's Hospital
  • Southampton General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

1

2

Arm Description

Outcomes

Primary Outcome Measures

To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control

Secondary Outcome Measures

To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF on existing RhDNase treatment compared to control. (key objective)
Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)
Improves quality of life (key objective)
Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
Reduces days in hospital due to pulmonary exacerbations
Improves other measures of lung function
Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)
Reduces hospital and community care costs

Full Information

First Posted
March 12, 2007
Last Updated
June 23, 2010
Sponsor
Pharmaxis
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1. Study Identification

Unique Protocol Identification Number
NCT00446680
Brief Title
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
Official Title
Long Term Administration of Inhaled Dry Powder Mannitol In Cystic Fibrosis - A Safety and Efficacy Study
Study Type
Interventional

2. Study Status

Record Verification Date
June 2010
Overall Recruitment Status
Completed
Study Start Date
March 2007 (undefined)
Primary Completion Date
May 2010 (Actual)
Study Completion Date
May 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Pharmaxis

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
Mannitol, Cystic Fibrosis, Mucolytic, Exacerbation, FEV1, Quality of Life

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
340 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Experimental
Arm Title
2
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Mannitol
Intervention Description
400mg BD for 6 months followed by a 6 month open label period
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
placebo BD for 6 months
Primary Outcome Measure Information:
Title
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF compared to control
Time Frame
6 months
Secondary Outcome Measure Information:
Title
To determine the effects of 400 mg twice-daily administration of IDPM on FEV1 in patients with CF on existing RhDNase treatment compared to control. (key objective)
Time Frame
6 months
Title
Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective)
Time Frame
6 months / 12 months
Title
Improves quality of life (key objective)
Time Frame
6 months
Title
Reduces days on IV antibiotics, rescue oral or inhaled antibiotics
Time Frame
6 months / 12 months
Title
Reduces days in hospital due to pulmonary exacerbations
Time Frame
6 months / 12 months
Title
Improves other measures of lung function
Time Frame
6 months
Title
Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination)
Time Frame
6 months / 12 months
Title
Reduces hospital and community care costs
Time Frame
6 months / 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main 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 Main 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Brett Charlton, MBBS
Organizational Affiliation
Pharmaxis Ltd Australia
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Dr Diana Bilton
Organizational Affiliation
Papworth Hospital Cambridge, UK
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Dr Philip Robinson
Organizational Affiliation
Royal Children's Hospital, Melbourne, Australia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Childrens Hospital at Westmead
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Sydney Childrens Hospital
City
Sydney
State/Province
New South Wales
Country
Australia
Facility Name
Royal Brisbane Children's Hospital
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
The Prince Charles Hospital
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4032
Country
Australia
Facility Name
Royal Adelaide Hospital
City
Adelaide
State/Province
South Australia
Country
Australia
Facility Name
Royal Childrens Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Beaumont Hospital
City
Dublin
Country
Ireland
Facility Name
National Children's Hospital
City
Dublin
Country
Ireland
Facility Name
Our Lady's Hospital for Sick Children
City
Dublin
Country
Ireland
Facility Name
St Vincent's University Hospital
City
Dublin
Country
Ireland
Facility Name
Alder Hey Children's Hospital
City
West Derby
State/Province
Liverpool
Country
United Kingdom
Facility Name
Belfast City Hospital
City
Belfast
State/Province
Northern Ireland
ZIP/Postal Code
BT9 7AB
Country
United Kingdom
Facility Name
Children's Hospital for Wales
City
Cardiff
State/Province
Wales
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Facility Name
Llandough Hospital
City
Cardiff
State/Province
Wales
ZIP/Postal Code
CF64 2XX
Country
United Kingdom
Facility Name
Birmingham Children's Hospital
City
Birmingham
Country
United Kingdom
Facility Name
Birmingham Heartlands Hospital
City
Birmingham
Country
United Kingdom
Facility Name
Bristol Royal Hospital for Children
City
Bristol
Country
United Kingdom
Facility Name
Bristol Royal Infirmary
City
Bristol
Country
United Kingdom
Facility Name
Addenbrooke's Hospital
City
Cambridge
Country
United Kingdom
Facility Name
Papworth Hospital
City
Cambridge
Country
United Kingdom
Facility Name
Seacroft Hospital
City
Leeds
Country
United Kingdom
Facility Name
Cardiothoracic Centre
City
Liverpool
ZIP/Postal Code
L14 3PE
Country
United Kingdom
Facility Name
The London Chest Hospital
City
London
ZIP/Postal Code
E2 9JX
Country
United Kingdom
Facility Name
Freeman Hospital
City
Newcastle
ZIP/Postal Code
NE7 7DN
Country
United Kingdom
Facility Name
Norfolk and Norwich University Hospital
City
Norwich
ZIP/Postal Code
NR4 7UY
Country
United Kingdom
Facility Name
Nottingham City Hospital
City
Nottingham
Country
United Kingdom
Facility Name
Northern General Hospital
City
Sheffield
Country
United Kingdom
Facility Name
Sheffield Children's Hospital
City
Sheffield
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom

12. IPD Sharing Statement

Citations:
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
32358807
Citation
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.
Results Reference
derived
PubMed Identifier
21478216
Citation
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.
Results Reference
derived

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

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