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

• ClinicalTrials.gov Identifier: NCT00455130
• Recruitment Status: Completed
• First Posted: April 3, 2007
• Last Update Posted: February 2, 2010
• Sponsor: Pharmaxis
• Information provided by: Pharmaxis

Tracking Information

• First Submitted Date: April 2, 2007
• First Posted Date: April 3, 2007
• Last Update Posted Date: February 2, 2010
• Study Start Date: March 2004
• Primary Completion Date: Not Provided
• Current Primary Outcome Measures (submitted: April 2, 2007): FEV1
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: April 2, 2007)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Phase 2 Study to Determine the Safety and Efficacy of Inhaled Dry Powder Mannitol in Cystic Fibrosis
• Official Title: Not Provided

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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: Double; Primary Purpose: Treatment
• Condition: Cystic Fibrosis
• Intervention: Drug: Inhaled mannitol
• Study Arms: Not Provided

Publications *

• Jaques A, Daviskas E, Turton JA, McKay K, Cooper P, Stirling RG, Robertson CF, Bye PTP, LeSouëf 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.

Recruitment Information

• Recruitment Status: Completed
• Enrollment: Not Provided
• Original Enrollment: Not Provided
• Actual Study Completion Date: August 2005
• Primary Completion Date: Not Provided

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 8 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, New Zealand

Administrative Information

• NCT Number: NCT00455130
• Other Study ID Numbers: DPM-CF-201
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Not Provided
• Study Sponsor: Pharmaxis
• Collaborators: Not Provided

Investigators

• Study Director: Brett Charlton; Pharmaxis

• PRS Account: Pharmaxis
• Verification Date: January 2010