Study Results

• What We Learned:

  This study found that in the TEZ/IVA group, 7 (14%) participants experienced a respiratory adverse event, compared to 10 (21.3%) participants in the placebo group. A small improvement of 2.2% ppFEV1 in lung function was seen in the TEZ/IVA group compared to placebo (-0.6% ppFEV1). Overall, participants in this study had few respiratory side effects and they did not occur more often in those treated with TEZ/IVA compared with placebo.

• Primary Findings:

  Effectiveness:

  Safety:

  This study was conducted between June 2017 and August 2018. In the study, 48 participants in the tezacaftor/ivacaftor (TEZ/IVA) group and 45 in the placebo group completed the study.

   

  The primary objective of the study was to evaluate the frequency of specific respiratory adverse events: chest discomfort, shortness of breath, chest tightness, asthma, bronchospasm, bronchial hyperreactivity, and wheezing. In the TEZ/IVA group, 7 (14%) participants experienced one of these respiratory adverse events. In the placebo group, 10 (21.3%) participants experienced one of these respiratory events. The most common of these respiratory events was shortness of breath (TEZ/IVA: 5 [10.0%]; placebo: 5 [10.6%]).  The only respiratory adverse event that occurred more frequently in the TEZ/IVA group compared with the placebo group was respiratory chest tightness; TEZ/IVA: 3 [6.0%]; placebo: 1 [2.1%]), but this event was uncommon and the difference was not significant.

   

  A small improvement of 2.2% ppFEV1 in lung function was seen in the TEZ/IVA group compared with a -0.6% ppFEV1 decline in the placebo group. Overall, participants in this study had few respiratory side effects and they did not occur more often in those treated with TEZ/IVA compared with placebo.

   

• Citation:

  J Cyst Fibros 2020;DOI 10.1016/j.jcf.2020.06.001

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