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Restore CFTR Protein Enrolling

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

This study will look at the effectiveness of Trikafta, a drug intended to help CFTR function closer to normal. This study is 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 will enroll 20 participants who will receive Trikafta for approximately four weeks. Researchers will test the effectiveness of the drug by measuring changes in lung function and sweat chloride. They will also take skin biopsies and/or blood to test how the body’s cells respond to Trikafta and test to see if these cells can be used to predict clinical response for rare CF patient populations.

This study may require lung function tests, sweat chloride tests, skin biopsies, blood draws and/or other measures.

Learn More

Email a research coordinator to express interest in participating in this study.

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 a more comprehensive list of eligibility criteria and details on this study, 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 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

Study Sites

  • Enrolling

    Alabama

    University of Alabama at Birmingham, Birmingham, AL 35294

    Contact

    Heather Hathorne

    Phone: +1 (205) 638-9568

    Email:

  • Enrolling

    Georgia

    Emory University, Atlanta, GA 30327

    Contact

    Ashleigh Streby

    Phone: +1 (404) 727-0305

    Email:

Learn More

Email a research coordinator to express interest in participating in this study.

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 a more comprehensive list of eligibility criteria and details on this study, 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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