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

Cystic Fibrosis Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00455130
• Other study ID #: DPM-CF-201
• Status: Completed
• Phase: Phase 2
• First received: April 2, 2007
• Last updated: January 31, 2010
• Start date: March 2004
• Est. completion date: August 2005

Study information

• Verified date: January 2010
• Source: Pharmaxis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Australia: Department of Health and Ageing Therapeutic Goods AdministrationNew Zealand: Medsafe
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: August 2005
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 8 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Inhaled mannitol

Locations

Country	Name	City	State
Australia	Prince Charles Hospital	Brisbane	Queensland
Australia	Royal Children's Hospital	Melbourne	Victoria
Australia	The Alfred Hospital	Melbourne	Victoria
Australia	Princess Margaret Hospital for Children	Perth	Western Australia
Australia	Sir Charles Gairdner	Perth	Western Australia
Australia	Childrens Hospital at Westmead	Sydney	New South Wales
Australia	Royal Prince Alfred Hospital	Sydney	New South Wales
New Zealand	Greenlane Hospital	Auckland	North Island

Sponsors (1)

Lead Sponsor	Collaborator
Pharmaxis

Countries where clinical trial is conducted

Australia,  New Zealand, 

References & Publications (2)

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. — View Citation

Jaques A, Daviskas E, Turton JA, McKay K, Cooper P, Stirling RG, Robertson CF, Bye PT, Lesouëf PN, Shadbolt B, Anderson SD, Charlton B. Inhaled mannitol improves lung function in cystic fibrosis. Chest. 2008 Jun;133(6):1388-96. doi: 10.1378/chest.07-2294. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	FEV1
Secondary	Other measures of lung function
Secondary	Quality of life
Secondary	Sputum microbiology
Secondary	Sputum rheology
Secondary	Safety