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Restore CFTR Protein Completed with Results
Study of effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in people with CF who have one copy of the F508del CFTR mutation (Vertex VX17-445-102)
This study evaluated the effectiveness and safety of the CFTR modulator drug VX-445 (elexacaftor) in combination with tezacaftor and ivacaftor (ELX/TEZ/IVA). This triple combination drug is known as Trikafta™. This study was for people with cystic fibrosis ages 12 and older with one copy of the F508del CFTR mutation and one copy of a minimal function mutation.
Participants were randomly assigned to receive elexacaftor (200mg once daily) plus tezacaftor (100mg once daily) plus ivacaftor (150mg every 12 hours) or to receive placebo for 24 weeks.
Eligibility
See other primary eligibility criteria for more information.
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Age:
12 Years and Older -
Mutation(s):
One Copy F508del -
FEV1% Predicted:
40 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Participants in this study must have one F508del mutation and one minimal function mutation. Please click on the link in the description above to see the list of eligible mutations.
Study Results
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What We Learned:
This study found that treatment with ELX/TEZ/IVA when compared with placebo resulted in improvements in lung function and patient reported outcomes, as well as decreases in sweat chloride concentration and a lower annual rate of pulmonary exacerbations. ELX/TEZ/IVA was generally well-tolerated. Two participants (1%) in the ELX/TEZ/IVA group withdrew from the study; one due to rash, and one due to an increase in blood pressure in the portal vein system involving the liver.
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Primary Findings:
Effectiveness:
This study was conducted between June 2018 and April 2019. In the study, 405 participants were randomly assigned and 403 participants received treatment (n=200 in the ELX/TEZ/IVA group; n=203 in the placebo group).
The primary outcome of the study was change in lung function from the beginning of the study to week 4 (measured by absolute change in ppFEV1). This study met its primary outcome. Treatment with ELX/TEZ/IVA resulted in an improvement of 13.6% ppFEV1 compared to a -0.2% decrease in ppFEV1 in the placebo group. Therefore, ELX/TEZ/IVA resulted in a 13.8% ppFEV1 lung function improvement when compared to placebo.
Improvements were also seen in sweat chloride concentrations. ELX/TEZ/IVA resulted in a -42.4 mmol/L decrease compared with placebo (-0.4 mmol/L). Treatment with ELZ/TEZ/IVA led to a 63 decrease in the annual rate of pulmonary exacerbations than placebo. The CFQR- RD score (a patient reported outcome questionnaire to measure respiratory symptoms) improved in the ELZ/TEZ/IVA group by a difference of 20.2 points compared with placebo.
Safety:
ELX/TEZ/IVA was generally well-tolerated. Two participants (1%) in the ELX/TEZ/IVA group withdrew from the study; one due to rash, and one due to an increase in blood pressure in the portal vein system involving the liver.
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Citation:
N Eng J Med ;DOI 10.1056/NEJMoa1908639;381(19):1809-1819
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
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Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
32 weeks -
Number of Study Visits:
10
Additional Information
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Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
12 Years and Older -
Mutation(s):
One Copy F508del -
FEV1% Predicted:
40 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Participants in this study must have one F508del mutation and one minimal function mutation. Please click on the link in the description above to see the list of eligible mutations.

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