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Restore CFTR Protein Completed with Results

Phase 3 study of VX-659 triple combination drug in people with CF 12 years and older who have one copy of the F508del mutation and one copy of a minimal function mutation (VX17-659-102)

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 one copy of the F508del CFTR mutation and one copy of a minimal function mutation.

 Participants in this study were randomly assigned to receive VX-659 (240mg)/tezacaftor (100mg)/ivacaftor (150mg) or placebo twice daily for 24 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

Participants must have one F508del mutation and one minimal function mutation.  Please click on the link in the description above to see the list of eligible mutations.

Study Results

  • What We Learned:

    Treatment with VX-659/tezacaftor/ivacaftor resulted in robust improvements in lung function, reduced sweat chloride and reduced frequency of pulmonary exacerbations compared with placebo.  It was also generally safe and well-tolerated with more patients experiencing increases in liver enzymes compared with participants receiving 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 382 participants between March and August, 2018.  Participants were randomly assigned to receive either VX-659/tezacaftor/ivacaftor (N=192) or placebo. After 24 weeks of treatment, the VX-659/tezacaftor/ivacaftor group had an improvement in lung function, as measured by FEV1 of 13.4 percentage points compared with a decline of 0.8 percentage points for the placebo group.

     

    The effect of VX-659/tezacaftor/ivacaftor on pulmonary exacerbations was similarly robust; 17 of 192 participants treated with VX-659/tezacaftor/ivacaftor experienced a pulmonary exacerbation compared with 116 of 190 placebo treated participants.  Additionally, treatment with VX-659/tezacaftor/ivacaftor resulted in a decrease in sweat chloride of 44.6 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:

    32 weeks

  • Number of Study Visits:

    10

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

Participants must have one F508del mutation and one minimal function mutation.  Please click on the link in the description above to see the list of eligible mutations.

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