search
Back to results

A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Inhaled mannitol
Sponsored by
Pharmaxis
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring mannitol, cystic fibrosis, mucolytic, mucoactive

Eligibility Criteria

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

Inclusion Criteria:

  1. Confirmed diagnosis of cystic fibrosis (sweat test/genotype)
  2. Aged 8 years or older
  3. Have FEV1 between 40% and 80% of predicted for height, age and gender OR a decrease in FEV1 of 20% or more than that recorded 6-12 months previously.

  4. As determined by the investigator, are capable and willing to

    • Use the study diary as required for this protocol
    • Able to perform all of the techniques necessary to measure lung function
    • Able to administer the dry powder mannitol
  5. Are capable of and have given informed consent
  6. Clinically stable at study entry

Exclusion Criteria:

  1. Investigators, site personnel directly affiliated with this study, and their immediate families.
  2. Subjects under the age of 8 years.
  3. Subjects with currently active asthma
  4. Subjects using hypertonic saline treatment in the last 2 weeks
  5. Considered "terminally ill" or listed for transplantation
  6. Requiring home oxygen or assisted ventilation
  7. Colonisation with Burkholderia cepacia
  8. Significant episode of hemoptysis (>60 mls) in the previous 12 months
  9. Myocardial Infarction in the six months prior to enrolment.
  10. Cerebral Vascular Accident in the six months prior to enrolment.
  11. Ocular surgery in the three months prior to enrolment.
  12. Abdominal surgery in the three months prior to enrolment.
  13. Subjects who are breast feeding or pregnant.
  14. Female subjects of reproductive capability, not using a reliable form of contraception
  15. Inability to obtain informed consent from the subject or subject's authorised representative.
  16. Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry.
  17. Known intolerance to mannitol or beta2 agonists.
  18. Uncontrolled hypertension - systolic BP > 160 and or diastoli

Sites / Locations

  • Royal Prince Alfred Hospital
  • Childrens Hospital at Westmead
  • Prince Charles Hospital
  • Royal Children's Hospital
  • The Alfred Hospital
  • Princess Margaret Hospital for Children
  • Sir Charles Gairdner
  • Greenlane Hospital

Outcomes

Primary Outcome Measures

FEV1

Secondary Outcome Measures

Other measures of lung function
Quality of life
Sputum microbiology
Sputum rheology
Safety

Full Information

First Posted
April 2, 2007
Last Updated
January 31, 2010
Sponsor
Pharmaxis
search

1. Study Identification

Unique Protocol Identification Number
NCT00455130
Brief Title
A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis
Study Type
Interventional

2. Study Status

Record Verification Date
January 2010
Overall Recruitment Status
Completed
Study Start Date
March 2004 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
August 2005 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Pharmaxis

4. Oversight

5. Study Description

Brief Summary
Cystic fibrosis is the most frequent lethal genetic disease of childhood. Causes disruption of glandular function of the pancreas, intestine, liver, lungs (causing chronic lung infection with emphysema), sweat glands and reproductive organs. We know that many CF patients die of lung failure, brought about in part by repeated lung infections caused by thick, sticky mucus that cannot be readily cleared from the lung. Inhaled mannitol is an osmotic agent that has been investigated in a number of small studies that have examined mucociliary clearance, quality of life and lung function in CF and bronchiectasis. The promising results of these studies warrant futher investigation. The aim of this study is to assess the safety and efficacy of inhaled mannitol when administered twice a day over two weeks in CF.

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, mucoactive

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
Double
Allocation
Randomized

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Inhaled mannitol
Primary Outcome Measure Information:
Title
FEV1
Secondary Outcome Measure Information:
Title
Other measures of lung function
Title
Quality of life
Title
Sputum microbiology
Title
Sputum rheology
Title
Safety

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Confirmed diagnosis of cystic fibrosis (sweat test/genotype) Aged 8 years or older Have FEV1 between 40% and 80% of predicted for height, age and gender OR a decrease in FEV1 of 20% or more than that recorded 6-12 months previously. As determined by the investigator, are capable and willing to Use the study diary as required for this protocol Able to perform all of the techniques necessary to measure lung function Able to administer the dry powder mannitol Are capable of and have given informed consent Clinically stable at study entry Exclusion Criteria: Investigators, site personnel directly affiliated with this study, and their immediate families. Subjects under the age of 8 years. Subjects with currently active asthma Subjects using hypertonic saline treatment in the last 2 weeks Considered "terminally ill" or listed for transplantation Requiring home oxygen or assisted ventilation Colonisation with Burkholderia cepacia Significant episode of hemoptysis (>60 mls) in the previous 12 months Myocardial Infarction in the six months prior to enrolment. Cerebral Vascular Accident in the six months prior to enrolment. Ocular surgery in the three months prior to enrolment. Abdominal surgery in the three months prior to enrolment. Subjects who are breast feeding or pregnant. Female subjects of reproductive capability, not using a reliable form of contraception Inability to obtain informed consent from the subject or subject's authorised representative. Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry. Known intolerance to mannitol or beta2 agonists. Uncontrolled hypertension - systolic BP > 160 and or diastoli
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Brett Charlton
Organizational Affiliation
Pharmaxis
Official's Role
Study Director
Facility Information:
Facility Name
Royal Prince Alfred Hospital
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Childrens Hospital at Westmead
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Prince Charles Hospital
City
Brisbane
State/Province
Queensland
Country
Australia
Facility Name
Royal Children's Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
The Alfred Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3181
Country
Australia
Facility Name
Princess Margaret Hospital for Children
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6840
Country
Australia
Facility Name
Sir Charles Gairdner
City
Perth
State/Province
Western Australia
Country
Australia
Facility Name
Greenlane Hospital
City
Auckland
State/Province
North Island
Country
New Zealand

12. IPD Sharing Statement

Citations:
PubMed Identifier
18339790
Citation
Jaques A, Daviskas E, Turton JA, McKay K, Cooper P, Stirling RG, Robertson CF, Bye PTP, LeSouef PN, Shadbolt B, Anderson SD, Charlton B. Inhaled mannitol improves lung function in cystic fibrosis. Chest. 2008 Jun;133(6):1388-1396. doi: 10.1378/chest.07-2294. Epub 2008 Mar 13.
Results Reference
result
PubMed Identifier
19880909
Citation
Daviskas E, Anderson SD, Jaques A, Charlton B. Inhaled mannitol improves the hydration and surface properties of sputum in patients with cystic fibrosis. Chest. 2010 Apr;137(4):861-8. doi: 10.1378/chest.09-2017. Epub 2009 Oct 31.
Results Reference
result

Learn more about this trial

A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis

We'll reach out to this number within 24 hrs