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Restore CFTR Protein Completed with Results

Study of VX-445 plus ivacaftor and tezacaftor in people with CF who have two copies of the F508del CFTR mutation (Vertex VX17-445-103)

This study evaluated the effectiveness and safety of the CFTR modulator drug VX-445 (elexacaftor) in combination with ivacaftor and tezacaftor. This triple combination is known as Trikafta™. This study was for people with CF ages 12 and older who have two copies of the F508del CFTR mutation and were already taking tezacaftor plus ivacaftor.

 All participants took tezacaftor (100mg daily) plus ivacaftor (150mg every 12 hours) for 4 weeks as a run-in period so that all participants in the study started on the two drugs before being randomly assigned to a treatment group. Following the run-in period, participants were randomly assigned to stay on tezacaftor and ivacaftor or to take elexacaftor (200mg daily) plus tezacaftor (100mg daily) and ivacaftor (150mg every 12 hours) for 4 weeks.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies 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

Participants must be homozygous for F508del.

Study Results

  • What We Learned:

    This study found that elexacaftor plus tezacaftor plus ivacaftor (ELX/TEZ/IVA) resulted in improvements in lung function above the baseline established after 4 weeks of treatment with tezacaftor plus ivacaftor (TEZ/IVA). Significant improvements in both sweat chloride concentration and a patient reported outcome questionnaire (CFQR-R RD) were noticed. ELX/TEZ/IVA was generally well-tolerated. Rash was seen in two (4%) participants in the ELX/TEZ/IVA group and also in two (4%) participants in the TEZ/IVA group. All four participants with rash were female.

  • Primary Findings:

    Effectiveness:

    This study was conducted between August 3rd and December 28th, 2018. The study enrolled 113 participants and 107 participants completed the 4-week run-in with TEZ/IVA and were randomly selected to receive either ELX/TEZ/IVA (n= 55) or to remain on TEZ/IVA alone (n=52).

     

    The primary outcome of the study was the change in lung function from the 4-week run-in period with TEZ/IVA to 4 weeks of treatment. The addition of ELX to TEZ/IVA resulted in a 10.4% pp FEV1 change after 4 weeks of treatment; participants in the TEZ/IVA alone group experienced a small change of 0.4% ppFEV1 after 4 weeks of treatment. The difference in lung function improvement between the two groups was 10 % ppFEV1 (95% CI 7.4- 12; p<0.0001).

     

    In addition to improvement in lung function, the ELX/TEZ/IVA group also saw improvements in sweat chloride concentration compared to the TEZ/IVA group (TEZ/IVA= 1.7 mmol/L; ELX/TEZ/IVA= -43.4 mmol/L). Improvements in the CFQ-R RD score (a patient reported outcome questionnaire), were also seen the ELX/TEZ/IVA group when compared to the TEZ/IVA group (TEZ/IVA= -1.4; ELX/TEZ/IVA= 16).

    Safety:

    ELX/TEZ/IVA was generally well-tolerated. Rash was seen in two (4%) participants in the ELZ/TEZ/IVA group and in two (4%) participants in the TEZ/IVA group. All four participants with rash were female.

  • Citation:

    Lancet ;DOI 10.1016/S0140-6736(19)32597-8(19):32597-32598

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:

    10 weeks

  • Number of Study Visits:

    7

Additional Information

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies 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

Participants must be homozygous for F508del.

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