Study Results

• What We Learned:

  This study found that participants receiving azithromycin in addition to TIS therapy experienced a significant reduction in risk of exacerbation, as measured by time to first exacerbation requiring antibiotics and increased weight when compared to placebo. Azithromycin was well-tolerated.

• Primary Findings:

  Effectiveness:

  This study was conducted between June 2014 and March 2017. A total of 221 participants were randomized and enrolled (azithromycin: N=110 and placebo: N=111). Following recommendation by the Data Safety Monitoring Board (DSMB) overseeing the trial, enrollment was stopped early by the National Heart, Lung, and Blood Institute (NHLBI) because the trial data showed early efficacy based on primary endpoint parameters determined prior to the study starting. The trial was amended to continue as an open-label study for the remainder of the planned study follow-up.

   

  The primary efficacy endpoint was time to pulmonary exacerbation requiring antibiotics. The study met its primary endpoint. Risk of pulmonary exacerbation was reduced by 44% in participants receiving oral azithromycin on top of their TIS therapy when compared with placebo (p=0.046).

   

  The study also found that weight increased by 1.27 kg in the azithromycin group compared to the placebo group (p=0.046). No significant difference was observed in time to Pa reoccurrence between the two groups.

  Safety:

  Azithromycin was well-tolerated and no significant differences in serious adverse events were observed between the two groups.

• Citation:

  Am J Respir Crit Care Med ;DOI 10.1164/rccm.201802-0215OC [Epub ahead of print]

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