- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00455130
A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis
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.
Study Overview
Study Type
Phase
- Phase 2
Contacts and Locations
Study Locations
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New South Wales
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Sydney, New South Wales, Australia, 2050
- Royal Prince Alfred Hospital
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Sydney, New South Wales, Australia, 2145
- Childrens Hospital at Westmead
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Queensland
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Brisbane, Queensland, Australia
- Prince Charles Hospital
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Victoria
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Melbourne, Victoria, Australia, 3181
- The Alfred Hospital
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Melbourne, Victoria, Australia, 3052
- Royal Children's Hospital
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Western Australia
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Perth, Western Australia, Australia, 6840
- Princess Margaret Hospital for Children
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Perth, Western Australia, Australia
- Sir Charles Gairdner
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North Island
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Auckland, North Island, New Zealand
- Greenlane Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Double
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
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FEV1
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Secondary Outcome Measures
Outcome Measure |
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Quality of life
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Safety
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Other measures of lung function
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Sputum microbiology
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Sputum rheology
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Brett Charlton, Pharmaxis
Publications and helpful links
General Publications
- 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.
- 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.
Study record dates
Study Major Dates
Study Start
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- DPM-CF-201
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Cystic Fibrosis
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Hospital de Clinicas de Porto AlegreUnknownCystic Fibrosis | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in Children | Cystic Fibrosis With ExacerbationBrazil
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University of Colorado, DenverCystic Fibrosis FoundationTerminatedCystic Fibrosis-related Diabetes | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in ChildrenUnited States
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Royal College of Surgeons, IrelandThe Hospital for Sick Children; Imperial College London; Erasmus Medical Center; University College Dublin and other collaboratorsActive, not recruitingCystic Fibrosis | Adherence, Medication | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in Children | Cystic Fibrosis Liver DiseaseUnited Kingdom, Ireland
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Herlev and Gentofte HospitalCopenhagen University Hospital, DenmarkActive, not recruitingMyocardial Infarction | Heart Diseases | Heart Failure | Stroke | Cystic Fibrosis | Heart Failure, Diastolic | Heart Failure, Systolic | Left Ventricular Dysfunction | Cystic Fibrosis-related Diabetes | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of Pancreas | Cystic Fibrosis, Pulmonary | Cystic...Denmark
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The Hospital for Sick ChildrenCanadian Cystic Fibrosis FoundationActive, not recruitingCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in ChildrenCanada
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Arrowhead PharmaceuticalsTerminatedCystic Fibrosis, PulmonaryAustralia, New Zealand
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AzurRx SASCompletedCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of PancreasTurkey, Hungary
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Dartmouth-Hitchcock Medical CenterTrustees of Dartmouth CollegeWithdrawnCystic Fibrosis-related Diabetes | Cystic Fibrosis Liver Disease | CF - Cystic FibrosisUnited States
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University Hospital, BordeauxCompleted
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University of PortsmouthUniversity Hospital Southampton NHS Foundation Trust; Loughborough University; Queen Alexandra HospitalTerminated
Clinical Trials on Inhaled mannitol
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PharmaxisCompletedCystic FibrosisUnited Kingdom
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PharmaxisCompletedBronchiectasisUnited States, United Kingdom, Australia, New Zealand, Argentina, Belgium, Chile, Germany, Netherlands
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PharmaxisCompletedHealthy | BronchiectasisAustralia
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Pharmaxisethica Clinical Research Inc.; Europe: KasaConsult bvba, Hoegaarden, Belgium; Argentina: Resolution Latin America; Buenos Aires, ArgentinaCompletedCystic FibrosisUnited States, France, Belgium, Canada, Argentina, Germany, Netherlands
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PharmaxisCompletedCystic FibrosisUnited States, Spain, Israel, Belgium, Czechia, South Africa, Italy, Mexico, Argentina, Australia, Canada, Hungary, New Zealand, Poland, Romania, Russian Federation, Slovakia, Ukraine
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Tim CorcoranCompleted
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Seoul National University HospitalUnknown
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PharmaxisCompletedCystic FibrosisUnited Kingdom
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Shandong UniversityUnknown
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Synspira, Inc.TerminatedLung Diseases | Cystic Fibrosis | Pulmonary Disease | Antibiotic Resistant Infection | Respiratory Tract Disease | Cystic Fibrosis Pulmonary Exacerbation | Lung Inflammation | Burkholderia Infections | Lung Infection | Multi-antibiotic Resistance | Pulmonary Inflammation | Lung Infection Pseudomonal | Cystic Fibrosis... and other conditionsUnited Kingdom