Study Results

• What We Learned:

  Study results show that the frequency of adverse events was similar in the treatment group compared to the placebo group, as well as across both parts of the study.

• Primary Findings:

  Effectiveness:

  This multi-center Phase 2 study was conducted between May 2007 and September 2008. A total of 39 participants were assigned to receive either ivacaftor at doses of 25, 75, or 150 mg or placebo twice a day for 14 days (during 2 14-day periods separated by a washout) [Part 1] or ivacaftor (at doses of 150 or 250 mg) or placebo twice a day for 28 consecutive days [Part 2].

   

  Safety:

  The frequency of adverse events was similar between the groups and between parts of the study. Two participants had severe adverse events (one participant had a diffuse macular rash and one participant with diabetes had elevated blood glucose levels on several occasions). All severe adverse events resolved without discontinuation of study drug.

• Citation:

  N Eng J Med 2010;363(21):1991-2003

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