Study Results

• What We Learned:

  This study showed that the participants taking lumacaftor and ivacaftor had significant improvements in lung function (measured by lung clearance index (LCI)) and in sweat chloride compared to the participants taking placebo. The safety profile for lumacaftor and ivacaftor was similar to what was seen in previous phase 3 studies.

• Primary Findings:

  Effectiveness:

  This study was conducted between July 23rd, 2015 and September 20th, 2016. A total of 206 participants were enrolled and randomly assigned to receive either lumacaftor and ivacaftor (n=104) or placebo (n=102). Of the 206 participants randomized, two participants withdrew from the study prior to dosing (one from each group). The reasons for withdrawal were unrelated to treatment. The primary endpoint of the study was the average change in lung function over the 24-week treatment period as measured by mean absolute change in lung clearance index (LCI). By day 15 of active treatment, participants who received lumacaftor and ivacaftor saw significant improvement in lung function compared to those taking placebo (-1.09 units LCI, p<0.0001). This improvement was sustained throughout the 24-week period.

   

  The study also evaluated sweat chloride concentration for both participant groups. Participants taking lumacaftor and ivacaftor saw a significant reduction in their sweat chloride concentration compared to those on placebo (-20.8 mmol/L, p<0.0001). Additionally, significant increases in body mass index (BMI) from baseline were observed in both groups at week 24.

  Safety:

  The overall safety profile of lumacaftor and ivacaftor in this study was consistent with previous phase 3 studies of this combination therapy. Serious adverse events were reported in 13 of the 103 participants (13%) who received lumacaftor and ivacaftor and 11 of the 101 participants (11%) who received placebo. Three participants in the lumacaftor and ivacaftor group (3%) and two in the placebo group (2%) discontinued treatment due to an adverse event. Common adverse events included respiratory symptoms and elevated liver enzymes, both of which are known effects of the combination therapy.

• Citation:

  Lancet ;DOI 10.1016/S2213-2600(17)30215-1

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