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Mannitol Dose Response Study in Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

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

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

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

Inclusion Criteria: Diagnosis of cystic fibrosis (sweat test/genotype) 7 years or older FEV1 between 40% and 90% of predicted for height, age and gender. Able to perform acceptable-quality spirometry Clinically stable in the week up to study entry No additional antibiotics or additional oral steroids for a period of 14 days before study entry (routine antibiotics permitted) Exclusion Criteria Currently active asthma Subjects colonized with Burkholderia cepacia or MRSA Considered "terminally ill" or listed for transplantation Requiring home oxygen or assisted ventilation Concurrent illness that in the investigators opinion may contribute to an increased and unacceptable risk if the subject was enrolled in the study (e.g. significant varicies, portal hypertension, cor pulmonale) Significant episode of haemoptysis (>60 mLs) in the previous 12 months Heart attack or stroke in last 3 months Known aortic or cerebral aneurysm Subjects who are breast feeding or pregnant. At risk females unwilling to use appropriate contraception to prevent pregnancy during the course of the study Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry. Known intolerance to mannitol or unable to take any form of bronchodilator medications. Uncontrolled hypertension, systolic BP > 200 or diastolic BP> than 100 Concurrent use of beta blocker medication Concurrent use of hypertonic saline Canada: Concurrent use of other pharmacological mucolytic agents other than Pulmozyme Argentina: Concurrent use of other pharmacological mucolytic agents including Pulmozyme

Sites / Locations

  • Hospital de Niños Superiora Sor María Ludovica
  • Hospital Pediatrico
  • Hospital Interzonal Especializado Materno Infantil (HIEMI)
  • Hospital General de Niños
  • Hospital Pediatrico Dr Humberto J Notti
  • BC Children's Hospital
  • St Pauls Hospital
  • Janeway Children's Health and Rehabilitation Center
  • Queen Elizabeth II Health Sciences Centre
  • Hamilton Health Sciences Corporation
  • The Hospital for Sick Children
  • St Michaels Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Active Comparator

Active Comparator

Active Comparator

Active Comparator

Arm Label

1

2

3

4

Arm Description

Outcomes

Primary Outcome Measures

FEV1
FVC

Secondary Outcome Measures

other measures of lung function
QOL
sputum microbiology
safety
sputum clearance and cough
respiratory symptoms

Full Information

First Posted
June 30, 2005
Last Updated
August 27, 2008
Sponsor
Pharmaxis
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1. Study Identification

Unique Protocol Identification Number
NCT00251056
Brief Title
Mannitol Dose Response Study in Cystic Fibrosis
Official Title
A Phase IIa Randomised, Open Label, Dose Response Study to Determine the Optimum Dose of Dry Powder Mannitol Required to Generate Clinical Improvement In Patients With Cystic Fibrosis
Study Type
Interventional

2. Study Status

Record Verification Date
August 2008
Overall Recruitment Status
Completed
Study Start Date
October 2005 (undefined)
Primary Completion Date
August 2008 (Actual)
Study Completion Date
August 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Pharmaxis

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Many cystic fibrosis patients die of lung failure caused by repeated lung infections from thick, sticky mucus. Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology and replenishing the airway surface liquid layer in these patients, thereby enhancing the shift of stagnant mucus from the lungs. The study aim is to determine the optimal dose of mannitol to generate clinical improvement in patients with cystic fibrosis.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
48 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Active Comparator
Arm Title
2
Arm Type
Active Comparator
Arm Title
3
Arm Type
Active Comparator
Arm Title
4
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
mannitol
Intervention Description
120mg BD
Intervention Type
Drug
Intervention Name(s)
mannitol
Intervention Description
40 mg BD
Intervention Type
Drug
Intervention Name(s)
mannitol
Intervention Description
240mg BD
Intervention Type
Drug
Intervention Name(s)
mannitol
Intervention Description
400mg BD
Primary Outcome Measure Information:
Title
FEV1
Time Frame
2 weeks
Title
FVC
Time Frame
2 weeks
Secondary Outcome Measure Information:
Title
other measures of lung function
Time Frame
various
Title
QOL
Time Frame
2 weeks
Title
sputum microbiology
Time Frame
2 weeks
Title
safety
Time Frame
2 weeks
Title
sputum clearance and cough
Time Frame
2 weeks
Title
respiratory symptoms
Time Frame
2 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosis of cystic fibrosis (sweat test/genotype) 7 years or older FEV1 between 40% and 90% of predicted for height, age and gender. Able to perform acceptable-quality spirometry Clinically stable in the week up to study entry No additional antibiotics or additional oral steroids for a period of 14 days before study entry (routine antibiotics permitted) Exclusion Criteria Currently active asthma Subjects colonized with Burkholderia cepacia or MRSA Considered "terminally ill" or listed for transplantation Requiring home oxygen or assisted ventilation Concurrent illness that in the investigators opinion may contribute to an increased and unacceptable risk if the subject was enrolled in the study (e.g. significant varicies, portal hypertension, cor pulmonale) Significant episode of haemoptysis (>60 mLs) in the previous 12 months Heart attack or stroke in last 3 months Known aortic or cerebral aneurysm Subjects who are breast feeding or pregnant. At risk females unwilling to use appropriate contraception to prevent pregnancy during the course of the study Subjects who have participated in another investigative drug study parallel to, or within 4 weeks of study entry. Known intolerance to mannitol or unable to take any form of bronchodilator medications. Uncontrolled hypertension, systolic BP > 200 or diastolic BP> than 100 Concurrent use of beta blocker medication Concurrent use of hypertonic saline Canada: Concurrent use of other pharmacological mucolytic agents other than Pulmozyme Argentina: Concurrent use of other pharmacological mucolytic agents including Pulmozyme
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elizabeth Tullis, MD
Organizational Affiliation
St Michaels Hospital, Toronto, Ontario, Canada
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Brett Charlton, MBBS PhD
Organizational Affiliation
Pharmaxis Ltd, Sydney, NSW, Australia
Official's Role
Study Director
Facility Information:
Facility Name
Hospital de Niños Superiora Sor María Ludovica
City
La Plata
State/Province
Buenos Aires
ZIP/Postal Code
B1904CSI
Country
Argentina
Facility Name
Hospital Pediatrico
City
Resistencia
State/Province
Chaco
Country
Argentina
Facility Name
Hospital Interzonal Especializado Materno Infantil (HIEMI)
City
Buenos Aires
Country
Argentina
Facility Name
Hospital General de Niños
City
Caba
Country
Argentina
Facility Name
Hospital Pediatrico Dr Humberto J Notti
City
Mendoza
Country
Argentina
Facility Name
BC Children's Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
St Pauls Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6Z1Y6
Country
Canada
Facility Name
Janeway Children's Health and Rehabilitation Center
City
St Johns
State/Province
Newfoundland and Labrador
ZIP/Postal Code
A1B 3V6
Country
Canada
Facility Name
Queen Elizabeth II Health Sciences Centre
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3H 3A7
Country
Canada
Facility Name
Hamilton Health Sciences Corporation
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
St Michaels Hospital
City
Toronto
State/Province
Ontario
Country
Canada

12. IPD Sharing Statement

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Mannitol Dose Response Study in Cystic Fibrosis

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