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Restore CFTR Protein Completed with Results

Phase 2 study of VX-152 combination drug in people with cystic fibrosis (Vertex VX-152-102)

This study evaluated the safety and tolerability of the drug VX-152 in combination with ivacaftor and tezacaftor in people who either have two copies of the F508del CFTR mutation or have one copy of F508del and one copy of a minimal function CFTR mutation.

 In Part 1 (F508del and minimal function group), participants were randomized to receive one of three different doses (100mg, 200mg or 300mg every 12 hours) of VX-152 in combination with ivacaftor (150mg every 12 hours)/tezacaftor (100mg daily) or triple combination placebo. In Part 2 (F508del/F508del group), participants were randomized to receive one of two different doses (200mg or 300mg every 12 hours) of VX-152 in combination with ivacaftor (150mg every 12 hours)/tezacaftor (100mg daily) or placebo in combination with ivacaftor/tezacaftor.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years and Older

  • Mutation(s):

    Two Copies F508del or 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

There are over 195 minimal function mutations that are eligible for this study. Some of the most common eligible mutations include: G542X, N1303K, W1282X, R553X, 621+1G->T, 1717-1G->A, 3120+1G->A, I507del, R1162X, 3659delC, 1898+1G->A, G85E, R560T, R347P, 2184insA, 2184delA, Q493X This is not a complete list of eligible mutations. Please contact a research coordinator to find out if your particular mutation is eligible for this study.

Study Results

  • What We Learned:

    In a study to test multiple doses of VX-152 in combination with people also treated with tezacaftor and ivacaftor, VX-152 treatment over 2-4 weeks resulted in better ppFEV1 and more normal sweat chloride concentration when compared with placebo. No significant safety concerns were identified in this relatively short study.

  • Primary Findings:

    Effectiveness:

    This study was conducted between November 2018 and January 2020. The study enrolled 80 patients to different dose combinations of VX-152 in addition to tezacaftor and ivacaftor.  

     

    The primary outcome was safety and tolerability. The primary efficacy outcome was the change in percent predicted FEV1 over 2-4 weeks treatment periods. In nearly all doses, VX-152 was statistically significantly better than control (tezacaftor/ivacaftor plus placebo) when comparing the change in ppFEV1

     

    In Part 1 and 2, changes in lung function (measured by mean absolute change in FEV1 percent of predicted) and sweat chloride were also measured. In Part 1(F508del and minimal function group), 21 participants were enrolled (N=6 at 100mg, N=10 at 200mg, and N=5 in placebo). There was a significant improvement in lung function in the 200mg group (+9.7%; p=0.0017) and a significant reduction of sweat chloride in the 100mg group (-19.6; p=0.0004) when compared to placebo. This difference was measured at 2 weeks after beginning VX-152 or placebo.

     

    In Part 2 (F508del/F508del group), 14 participants were enrolled (N=10 at 200 mg and N=4 in placebo). Change in lung function was not significant, however there was a significant reduction in sweat chloride (-20.9; p=0.0010) when compared to placebo.

     

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

     

    A key secondary outcome was the change in sweat chloride concentration over the 2-4 week treatment periods within the comparative dose groups. The change in sweat chloride concentrations were significantly greater in the VX-152 groups compared with placebo.

    Safety:

    The number of all adverse events and treatment-related adverse events were not statistically significantly different between the groups compared with placebo.

  • 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:

    16 weeks

  • Number of Study Visits:

    10

Additional Information

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years and Older

  • Mutation(s):

    Two Copies F508del or 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

There are over 195 minimal function mutations that are eligible for this study. Some of the most common eligible mutations include: G542X, N1303K, W1282X, R553X, 621+1G->T, 1717-1G->A, 3120+1G->A, I507del, R1162X, 3659delC, 1898+1G->A, G85E, R560T, R347P, 2184insA, 2184delA, Q493X This is not a complete list of eligible mutations. Please contact a research coordinator to find out if your particular mutation is eligible for this study.

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