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Restore CFTR ProteinClosed to Enrollment

Study of Trikafta® in people with CF ages 12 years and older who have partial function CFTR mutations , protocol number Sorscher Partial Function Sub-Study

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Restore CFTR ProteinClosed to Enrollment

Study of Trikafta® in people with CF ages 12 years and older who have partial function CFTR mutations , protocol number 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).

  • Age:

    12 Years and Older

  • Mutation(s):

    No Copies F508del

  • FEV1% Predicted:

    No FEV1 Limit

  • Number of Visits:

    5

  • Length of Participation:

    2 months

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