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Restore CFTR Function Completed with Results

Phase 3 study of VX-659 triple combination drug in people with CF ages 12 years and older who have two copies of the F508del mutation (VX17-659-103)

This study evaluated the safety and effectiveness of the drug VX-659 in combination with tezacaftor and ivacaftor in people with cystic fibrosis ages 12 and older with two copies of the F508del CFTR mutation.

 All participants in the study received tezacaftor/ivacaftor alone for a 4 week run-in period before being randomly assigned to continue taking tezacaftor (100mg)/ivacaftor (150mg) alone or receiving VX-659 (240mg)/tezacaftor (100mg)/ivacaftor (150mg) 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:

    Treatment with VX-659/tezacaftor/ivacaftor resulted in robust improvements in lung function and reduced sweat chloride compared with participants who received tezacaftor/ivacaftor alone.  It was also generally safe and well-tolerated with more patients experiencing increases in liver enzymes compared with participants who received placebo.  The sponsor (Vertex) ultimately decided to not move forward with development of this triple combination, electing to move forward with the VX-445 (elexacaftor) triple combination instead.

  • Primary Findings:

    Effectiveness:

    This study enrolled 116 participants between May and July 2018. Of those, 111 participants completed the run-in portion and were randomly assigned to continue on tezacaftor/ivacaftor (N=57) or to receive VX-659/tezacaftor/ivacaftor (N= 54).   Changes in sweat chloride and FEV1 were measured from the end of the run-in period to after 4 weeks of treatment; thus, participants in the tezacaftor/ivacaftor group were not expected to see further improvements.  Treatment with VX-659/tezacaftor/ivacaftor resulted in a further improvement in FEV1 of 10.2 percentage points compared with the tezacaftor/ivacaftor alone group and an additional decrease in sweat chloride of -47.2 mmol/L.

     

    Safety:

    VX-659/tezacaftor/ivacaftor was generally well tolerated; however, increases in liver enzymes were noted more frequently in VX-659/tezacaftor/ivacaftor treated participants.

  • 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

    Yes

  • 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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