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Restore CFTR Function Completed with Results

Study of VX-445 Triple Combination in Teens and Adults With Cystic Fibrosis (CF) Who Have One Copy of F508del and One Copy of a Gating or Residual Function Mutation (Vertex VX18-445-104)

This study evaluated the safety and effectiveness of VX-445 (elexacaftor), tezacaftor, and ivacaftor in combination called TRIKAFTA®. This study was for people with CF 12 years and older with one copy of the F508del mutation and one copy of a gating or residual function mutation.

 All participants had a 4-week run-in period of either ivacaftor (IVA) or tezacaftor/ivacaftor (TEZ/IVA). Following the run-in, participants were randomly assigned to receive elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) or keep taking their IVA or TEZ/IVA for 8 weeks.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    One Copy F508del

  • FEV1% Predicted:

    40 to 90%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Study participants must have one copy of the F508del mutation and one copy of a gating or residual function mutation. Click here to see a list of eligible mutations. 

Study Results

  • What We Learned:

    This study found a significant improvement in lung function of 3.7 percentage points in FEV1 percent of predicted (ppFEV1) (p<0.0001) in participants treated with ELX/TEZ/IVA compared to their baseline after a 4-week run-in of treatment on IVA or TEZ/IVA. Similarly, the study saw improvements in sweat chloride (mean absolute within-group change of -22.3 mmol/L (p<0.0001)) from baseline through 8 weeks in participants treated with ELX/TEZ/IVA. Overall, safety data were similar to those observed in previous Phase 3 studies of ELX/TEZ/IVA, and the triple combination was generally well tolerated. In the study, two (2) participants taking IVA or TEZ/IVA and one (1) participant taking ELX/TEZ/IVA discontinued treatment due to adverse events.

  • Primary Findings:

    Effectiveness:

    A total of 132 participants received ELX/TEZ/IVA and 126 participants were in the control group that received either IVA or TEZ/IVA.

     

    The primary objective of this study was improvement in lung function from baseline through 8 weeks of treatment, as measured by the mean absolute within-group improvement from baseline in ppFEV1. This study met its primary objective of a significant improvement in lung function of 3.7 percentage points in ppFEV1 (p<0.0001) in participants treated with ELX/TEZ/IVA compared to their baseline after a 4-week run-in of treatment on IVA or TEZ/IVA.

     

    Statistically significant improvements were also seen in sweat chloride (mean absolute within-group change of -22.3 mmol/L (p<0.0001)) from baseline through 8 weeks in participants treated with ELX/TEZ/IVA.

    Safety:

    Overall, safety data were similar to those observed in previous Phase 3 studies of ELX/TEZ/IVA, and the triple combination was generally well tolerated. The majority of adverse events were mild or moderate. The most common adverse event that occurred in 15% or more patients, regardless of treatment arm, was headache. Serious adverse events were observed in 3.8% (n=5) of the participants who received ELX/TEZ/IVA and in 8.7% (n=11) of the participants who received IVA or TEZ/IVA. In the study, two (2) participants taking IVA or TEZ/IVA and one (1) participant taking ELX/TEZ/IVA discontinued treatment due to adverse events.

     

    These results have been provided by a Vertex Press Release and are not peer reviewed.

  • Citation:

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

Study Design

  • Study Type: ?more info

    Interventional

  • Randomized Study: ?more info

    Yes

  • Placebo Controlled: ?more info

    No

  • Length of Participation:

    20 weeks

  • Number of Study Visits:

    8

Additional Information

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    One Copy F508del

  • FEV1% Predicted:

    40 to 90%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

Study participants must have one copy of the F508del mutation and one copy of a gating or residual function mutation. Click here to see a list of eligible mutations. 

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