A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis
Information source: Pharmaxis
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Cystic Fibrosis
Intervention: Inhaled mannitol (Drug)
Phase: Phase 2
Status: Completed
Sponsored by: Pharmaxis Official(s) and/or principal investigator(s): Brett Charlton, Study Director, Affiliation: Pharmaxis
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.
Clinical Details
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double-Blind, Primary Purpose: Treatment
Primary outcome: FEV1
Secondary outcome: Other measures of lung functionQuality of life Sputum microbiology Sputum rheology Safety
Eligibility
Minimum age: 8 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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
Locations and Contacts
Childrens Hospital at Westmead, Sydney, New South Wales 2145, Australia
Royal Prince Alfred Hospital, Sydney, New South Wales 2050, Australia
Greenlane Hospital, Auckland, North Island, New Zealand
Prince Charles Hospital, Brisbane, Queensland, Australia
Royal Children's Hospital, Melbourne, Victoria 3052, Australia
The Alfred Hospital, Melbourne, Victoria 3181, Australia
Princess Margaret Hospital for Children, Perth, Western Australia 6840, Australia
Sir Charles Gairdner, Perth, Western Australia, Australia
Additional Information
Starting date: March 2004
Last updated: January 31, 2010
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