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Restore CFTR Function Completed with Results

Phase 3 study of lumacaftor/ivacaftor in children with CF (Vertex VX15-809-115)

This study evaluated the safety and effectiveness of lumacaftor in combination with ivacaftor (Orkambi®) in people who have two copies of the F508del mutation.

 All participants in this open-label study received lumacaftor/ivacaftor. In Part A of the study, depending on their weight, participants received either lumacaftor (100mg every 12 hours) in combination with ivacaftor (125mg every 12 hours) or lumacaftor (150mg every 12 hours) in combination with ivacaftor (188mg every 12 hours) for 15 days. In Part B, depending on their weight, participants received either lumacaftor (100mg every 12 hours) in combination with ivacaftor (125mg every 12 hours) or lumacaftor (150mg every 12 hours) in combination with ivacaftor (188mg every 12 hours) for 24 weeks.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    Two Copies 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

Subjects who are homozygous for the F508del CFTR mutation are included

Study Results

  • What We Learned:

    The study found that lumacaftor/ivacaftor was generally well-tolerated and that there were no new safety findings compared to prior studies of lumacaftor/ivacaftor in older populations.

  • Primary Findings:

    Effectiveness:

    Safety:

    This study was conducted between May 2016 and March 2017. The study enrolled 68 participants (Part A N=11, Part B N=57). 

     

    The primary objective of the study was safety. The study found that lumacaftor/ivacaftor was generally well-tolerated and that there were no new safety findings compared to prior studies of lumacaftor/ivacaftor in older populations.

     

    Participants had decreases in sweat chloride (Part A= -33.5 mmol/L, Part B= -30.7 mmol/L) and improvements in nutritional status as measured by change in weight (weight-for-age z score) and body mass index (BMI-for-age z score).

     

    These results have been provided from Clincialtrials.gov and a Vertex Press Release and have not been peer reviewed.

  • 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

    No

  • Placebo Controlled: ?more info

    No

  • Length of Participation:

    32 weeks

  • Number of Study Visits:

    11

Additional Information

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    2 Years to 5 Years

  • Mutation(s):

    Two Copies 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

Subjects who are homozygous for the F508del CFTR mutation are included

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