Study Results

• What We Learned:

  Lumacaftor in combination with ivacaftor was well tolerated at the doses tested. Treatment with lumacaftor in combination with ivacaftor resulted in significant improvements in sweat chloride, nutrition (BMI and weight), lung function (as measured by lung clearance index (LCI)) and quality of life (self-reported).

• Primary Findings:

  Effectiveness:

  Part B of the study was conducted between February 2015 and April 2015. Of the 58 participants enrolled in Part B, 54 completed all 24 weeks of treatment with lumacaftor/ivacaftor. Two participants discontinued treatment due to drug related adverse events. One participant was enrolled but did not meet eligibility criteria and one participant voluntarily withdrew from the study. While the primary objective of the study was to evaluate the safety of lumacaftor in combination with ivacaftor, researchers also looked at absolute change in sweat chloride, BMI, weight and height, lung function (measured by FEV1 and LCI), as well as a self-reported quality of life questionnaire (CFQ-R). At the end of the 24-week treatment period, there was a significant reduction in sweat chloride (p<0.0001), increase in BMI and weight (p<0.0001), improvement in lung clearance index (P=0.0018), and improvement in the average response to the self-reported quality of life questionnaire (p=0.0085). Lung function as measured by FEV1 was not significantly improved by week 24.

   

  Safety:

  Lumacaftor in combination with ivacaftor at doses tested was generally well tolerated in this younger population, similar to what was observed in older individuals with CF. Laboratory tests of liver enzymes were significantly elevated in 19.3% of participants at some point during the study and resulted in study drug being interrupted or stopped for 5.3% of participants.

• Citation:

  Am J Respir Crit Care Med ;[Epub ahead of print]

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