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Restore CFTR Function Completed with Results

Vertex 809/770 in People with Cystic Fibrosis and F508del-CFTR mutation (Vertex VX-809-102)

This study looked at the safety and effectiveness of VX 809 (lumacaftor) when taken alone and when in combination with VX770 (ivacaftor) when given to people with CF who have either one or two copies of the F508del-CFTR 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% or greater

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

To be eligible for this study the participant must be either heterozygous or homozygous with the delta F508 mutation. NOTE: Sponsor added a 4th cohort that will open for recruitment in the Fall of 2013.

Study Results

  • What We Learned:

    Study results show that combination lumacaftor/ivacaftor treatment was associated with a significant decrease in sweat chloride concentration when compared to placebo.

  • Primary Findings:

    Effectiveness:

    This Phase 2 multi-national study was conducted between October 2010 and April 2014. Multiple different doses and dosage regimens were tested. The total number of participants was 188 most of whom had 2 copies of the F508del CFTR mutation (160 participants). The primary outcome was change in sweat chloride concentration during the combination treatment. Although results were variable between the several dosing regimens, overall a modest effect on sweat chloride was observed.

     

    Similarly, lung function (absolute change in FEV1) improved for some dosing regimens in the participants with 2 copies of F508del.

    Safety:

    Frequency and nature of adverse events were similar in the treatment and placebo groups during the combination treatment period; 12/97 participants experienced chest tightness or difficulty breathing when receiving lumacaftor (VX-809) alone.

  • Citation:

    Lancet ;DOI 10.1016/S2213-2600(14)70132-8

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:

    2 months

  • Number of Study Visits:

    6

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% or greater

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

To be eligible for this study the participant must be either heterozygous or homozygous with the delta F508 mutation. NOTE: Sponsor added a 4th cohort that will open for recruitment in the Fall of 2013.

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