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Restore CFTR Function 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 (VX-661) 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)/VX-661 (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)/VX-661 (100mg daily) or placebo in combination with ivacaftor/VX-661. The results provided below are limited to the 100mg and 200mg doses in people who have one F508del mutation and one minimal function mutation and from the 200mg dose in people who have two copies of the F508del mutation.

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:

    This study found that there was a significant improvement in lung function and in sweat chloride in participants who have one copy of F508del and one copy of a minimal function CFTR mutation and were receiving 200mg of VX-152 in combination with ivacaftor/VX-661. There was no significant improvement in lung function in participants have two copies of the F508del CFTR mutation and were receiving 200mg of VX-152 in combination with ivacaftor/VX-661. However, a significant reduction in sweat chloride was seen. VX-152 in combination with ivacaftor/VX-661 was generally well-tolerated. One participant on the triple combination therapy discontinued due to an adverse event.

  • Primary Findings:

    Effectiveness:

    The primary objective of the study was safety and tolerability. VX-152 in combination with ivacaftor/VX-661 was generally well-tolerated. One participant on the triple combination therapy discontinued due to an adverse event (pneumonia in the VX-152 200mg group).

     

    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.

     

    Safety:

  • 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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