Study Results

• What We Learned:

  Respiratory events occurring at the beginning of treatment were more frequent in this population of CF patients with advanced lung disease than in earlier studies of lumacaftor and ivacaftor. Overall 76% of participants completed treatment; initiating treatment at the half-dose was associated with a higher rate of treatment completion.

• Primary Findings:

  Effectiveness:

  Safety:

  This study was conducted between February 19th, 2015 and October 3rd, 2016. Of the 46 participants enrolled in the study, 35 (76%) completed the 24 weeks of treatment and 11 (24%) discontinued the study. Eight participants discontinued treatment due to adverse events with the most common reasons being abnormal breathing (n=3) and abnormal or difficult breathing (n=2). The primary objective was to assess the safety and tolerability of lumacaftor in combination with ivacaftor. Early in the study a serious respiratory event of difficulty breathing occurred, leading to a modification of the study to allow participants to initiate treatment with a half-dose for 1 to 2 weeks before moving to the full dose. A total of 28 participants (61%) started the study at the full dose and 18 participants (39%) started the study at half dose. Overall, 65% of participants experienced a respiratory adverse event with most events occurring on the first day. Participants who received the half dose at the start of the study had a lower occurrence and shorter duration of respiratory events (56% vs 71% - occurrence and 4 vs 9 days - duration). Additional evaluations showed the treatment with lumacaftor in combination with ivacaftor resulted in a significant reduction in sweat chloride concentration (average change from baseline to 24 weeks -20.2 mmol/L, p<0.0001).

• Citation:

  J Cyst Fibros ;DOI 10.1016/j.jcf.2017.09.012 [Epub ahead of print]

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