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Restore CFTR Protein Completed with Results

VX 809 and ivacaftor in people with CF who are aged 12 years and older and have two copies of the F508del-CFTR mutation (105) (Vertex VX-809-105)

This was an open-label follow-on study that evaluated the safety and efficacy of lumacaftor in combination with ivacaftor (Orkambi®) in people with CF, 12 years and older and who have two copies of the F508del genetic mutation. This study (PROGRESS) was a 96-week extension study in participants who had previously completed one of two 24-week placebo-controlled trials (TRANSPORT and TRAFFIC). Participants in these studies who had previously received lumacaftor/ivacaftor remained on the same dose in the PROGRESS study while participants who had received placebo were randomly assigned to receive lumacaftor (400mg every 12 hours) in combination with ivacaftor (250mg every 12 hours) or lumacaftor (600 mg once daily) in combination with ivacaftor (250mg every 12 hours).

 

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies 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

Subjects who have participated in Study VX-809-103 or VX-809-104 will be offered the opportunity to enroll in either a treatment or observational cohort in this study. Subjects must be Delta F508 homozygous to participate in this study.

Study Results

  • What We Learned:

    Results show that there were no new safety concerns associated with the commercially available lumacaftor (400mg)/ivacaftor (250mg) product in this long-term safety study. Additionally, lumacaftor/ivacaftor was associated with sustained improvements in body weight and participant reported outcomes relating to lung function.

  • Primary Findings:

    Effectiveness:

    Safety:

    This study occurred between October 24, 2013 and April 7, 2016. Out of the 1030 participants enrolled, 513 participants received lumacaftor (600 mg once daily) in combination with ivacaftor (250mg every 12 hours) and 517 participants received lumacaftor (400mg every 12 hours) in combination with ivacaftor (250mg every 12 hours). Of the 517 participants in the 400 mg group, 215 (42%) completed all 96 weeks of treatment. The 400mg lumacaftor/250mg ivacaftor dose is the commercially available product therefore this manuscript only reported the results of this treatment group. The primary endpoint was to assess the long-term safety of lumacaftor/ivacaftor as determined by study related adverse events as well as laboratory and clinical outcomes. No new safety concerns were observed in this longer-term safety follow-up study. Additional evaluations showed that the positive outcomes of lumacaftor/ivacaftor on lung function, nutritional status, and pulmonary exacerbation rates continued to be observed with up to 120 weeks of treatment.

  • Citation:

    Lancet ;DOI 10.1016/S2213-2600(16)30427-1

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:

    96 weeks

  • Number of Study Visits:

    7

Additional Information

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    Two Copies 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

Subjects who have participated in Study VX-809-103 or VX-809-104 will be offered the opportunity to enroll in either a treatment or observational cohort in this study. Subjects must be Delta F508 homozygous to participate in this study.

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