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Restore CFTR Protein Closed to Enrollment

Study of Trikafta® in people with CF ages 12 years and older who have partial function CFTR mutations (Sorscher Partial Function 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 evidence of partial CFTR function. Partial function is defined as a sweat chloride level < 80 mmol/L and/or pancreatic sufficiency (no pancreatic enzyme supplement therapy).

This study will enroll 22  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.
 

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

Potential participants in the study must have a CFTR genotype that is not eligible for Trikafta®.  Additionally participants must have evidence of partial CFTR function; either sweat chloride <80mmol/L or must not be prescribed pancreatic enzyme replacement treatment (PERT)

Study Design

  • Study Type: ?more info

    Interventional

  • Randomized Study: ?more info

    No

  • Placebo Controlled: ?more info

    No

  • Length of Participation:

    2 months

  • Number of Study Visits:

    5

Additional Information

Study Sites

  • Closed to Enrollment

    Alabama

    University of Alabama at Birmingham, Birmingham, AL 35233

  • Closed to Enrollment

    Georgia

    Emory University, Atlanta, GA 30324

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

Potential participants in the study must have a CFTR genotype that is not eligible for Trikafta®.  Additionally participants must have evidence of partial CFTR function; either sweat chloride <80mmol/L or must not be prescribed pancreatic enzyme replacement treatment (PERT)

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