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Restore CFTR Protein Closed to Enrollment
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.
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 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
-
Phase: ?more info
Phase Two -
Study Sponsor: ?more info
Linnemann, Rachel -
Study Drugs:
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
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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