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Restore CFTR Protein Completed with Results

Study of Trikafta® in people with CF ages 12 years and older who have an N1303K CFTR mutation (N1303K Sub-Study )

This study looked at the effectiveness of Trikafta®, a drug intended to improve CFTR function. This study was for people ages 12 and older who have two CFTR mutations not approved for Trikafta® and a least one of these mutations must be N1303K.

This study enrolled 20 participants who received Trikafta® for four weeks. Participants received two orally administered pills of 100 mg elexacaftor, 50 mg tezacaftor, and 75 mg ivacaftor once daily in the morning, and 150 mg ivacaftor once daily in the evening. Researchers tested the effectiveness of the drug by measuring changes in lung function and sweat chloride. 

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

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

Other Primary Eligibility Criteria

Study participants must have two CFTR mutations not approved for Trikafta® and a least one of these mutations must be N1303K.

Study Results

  • What We Learned:

    Trikafta® treatment for four weeks was generally safe and well-tolerated. Participants did not show reduction in sweat chloride, however, participants showed significant lung function improvement compared with their baseline.

  • Primary Findings:

    Effectiveness:

    This study was conducted between June 7, 2022, and Oct 20, 2023. 

    The study enrolled a total of 20 participants and all participants completed the study. The primary outcome was change in sweat chloride compared with baseline. No significant change in sweat chloride was observed.

     

    A key secondary outcome was change in percent predicted FEV1 over the 4-week treatment period. Trikafta® treatment resulted in a 9.5% increase in participants’ baseline FEV1 lung function.

    Safety:

    Trikafta® doses appeared to be generally safe and well tolerated. There were no adverse events related to the study drug.

  • Citation:

    Lancet 2024;12(12):947-957

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:

    86 days

  • Number of Study Visits:

    5

Additional Information

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    12 Years and Older

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

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

Other Primary Eligibility Criteria

Study participants must have two CFTR mutations not approved for Trikafta® and a least one of these mutations must be N1303K.

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