Study Results

• What We Learned:

• Primary Findings:

  Effectiveness:

  This Phase 3 multi-center, multi-national study was conducted between September 2008 and April 2010. A total of 305 people participated and received either 400 mg inhaled mannitol (184) or control (121). The majority of participants completed the first 6 months of the study (83% of those who received 40 mg inhaled mannitol and 88% of those receiving control. The primary endpoint of the study was the absolute change in FEV1 from baseline; there was a trend toward improvement in lung function in the inhaled mannitol group (106.5 mL) compared to the control group (52.4 mL) but this did not reach statistical significance (p=0.059).

   

  For other lung function 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 participants who were originally treated with the 400 mg mannitol, and improved in the original control group.

  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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