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Study Name: Inhaled Dry Powder Mannitol in Cystic Fibrosis
Study Type: Interventional
Intervention Category: Restore Airway Surface Liquid
Study Sponsor: Pharmaxis
Study Phase: 3
Recruitment Status: Trial Completed
Study Drug(s): Bronchitol
Number of Participants Being Recruited: 300
Single / Multi-Center: Multi-Center
STUDY BACKGROUND INFORMATION:
This study looks at how safe and how well treatment with inhaled dry powder mannitol works in people with cystic fibrosis. Previous studies showed better lung function and fewer respiratory symptoms after a 2 week treatment with mannitol. The study seeks to show that increasing the clearance of mucus out of the lungs will improve lung function and decrease lung infections. The initial 6 month blinded phase will be followed with an additional 6 months of open label treatment.
ELIGIBILITY
Age: >= 6 Years
FEV1: 30 - 89 Percent Predicted
P. aeruginosa status: Not applicable
B. cepacia status: Not applicable
Other Primary Eligibility Requirements:

Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.
FOR MORE INFORMATION:
Sponsor Contact Information: Figueroa, Lorena
Lorena.Figueroa@pharmaxis.com.au
Trial Specific Link on ClinicalTrials.gov: http://www.clinicaltrials.gov/ct2/show/NCT00630812?term=mannitol+and+cystic+fibrosis&rank=1
Clinical Research Terms Glossary: Click here
TRIAL RESULTS:
Primary Efficacy:

Three hundred five subjects with CF (age >/= 6 years) were enrolled in Phase 3 studies in North America, South America, and Europe to evaluate safety and efficacy of inhaled dry powder mannitol. Subjects received either 400 mg mannitol or control (low dose mannitol) twice daily for 26 weeks, followed by a 26-week open-label extension.
The primary endpoint, absolute change in FEV1 from baseline in treated versus control groups, averaged over the study period was not met. The mean improvement in FEV1 was greater in the mannitol group than the control group (106.5 vs. 52.4 ml) but did not meet statistical significance (p=0.059).

Secondary Efficacy:

For other spirometric measures (percent predicted FEV1 and FVC), the 400-mg inhaled mannitol dose resulted in a statistically and clinically significant average improvement in lung function over the treatment period compared with control.
No significant differences were seen in quality of life measures, pulmonary exacerbation rates, or hospitalization rates.
In the 26-week open-label extension study, FEV1 was maintained in the original treated group, and improved in the original control group to the same degree.

Safety:

Mannitol was well tolerated. Adverse events and sputum microbiology were similar in both the treatment and control groups.

Citation:

Am J Respir Crit Care Med 2012;185(6):645-652

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