Study Results

• What We Learned:

  This study found that participants, who received mannitol, over a 26-week treatment period, had greater improvements in lung function than those who received placebo. These improvements, while statistically significant, were less than what was seen in previous phase 3 studies with mannitol. Mannitol well-tolerated and has an adverse event profile similar to that of the placebo group.

• Primary Findings:

  Effectiveness:

  This study was conducted between October 2014 and February 2017. The study enrolled 423 participants in 21 countries with more than a quarter of the participants in the USA. The primary goal of the study was to evaluate the effect of mannitol on change in lung function (measured by FEV1) from baseline over the 26-week treatment period. The study showed that participants who received mannitol had greater improvement in lung function, at the end of the 26 week period, than the participants that received placebo (2.2% relative change in FEV1, p=0.025). While the improvement was statistically significant, it was less than what was seen in the previous phase 3 study conducted of mannitol.

   

  No statistically significant differences were seen between mannitol and placebo when researchers looked at additional measurements such as time to first pulmonary exacerbation or the number of days in the hospital due to a pulmonary exacerbation.

  Safety:

  Mannitol was generally well tolerated with similar rates of adverse events as seen in the placebo group. These data are preliminary and have not been peer-reviewed. These data were provided in a Pharmaxis Press Release.

• Citation:

For current information about the overall development status of this drug, please check the Drug Development Pipeline.