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Restore CFTR Protein Completed with Results
A study to evaluate tezacaftor/ivacaftor in adults with cystic fibrosis and two copies of the F508del mutation (Vertex VX16-661-114)
This study evaluated the safety, effectiveness, and tolerability of the drug tezacaftor/ivacaftor (Symdeko®).This study was for people with CF ages 12 and older with two copies of the F508del CFTR mutation who had been taken off lumacaftor/ivacaftor (Orkambi®) due to respiratory side effects.
Participants were randomly assigned to receive tezacaftor/ivacaftor (100mg/150mg) in the morning and ivacaftor (150mg) in the evening (TEZ/IVA) or matched placebo for 56 days.
Eligibility
See other primary eligibility criteria for more information.
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Age:
12 Years and Older -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
25 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Must have two copies of the F508del CFTR mutation and have previously discontinued treatment with Orkambi
Study Results
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What We Learned:
This study found that in the TEZ/IVA group, 7 (14%) participants experienced a respiratory adverse event, compared to 10 (21.3%) participants in the placebo group. A small improvement of 2.2% ppFEV1 in lung function was seen in the TEZ/IVA group compared to placebo (-0.6% ppFEV1). Overall, participants in this study had few respiratory side effects and they did not occur more often in those treated with TEZ/IVA compared with placebo.
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Primary Findings:
Effectiveness:
Safety:
This study was conducted between June 2017 and August 2018. In the study, 48 participants in the tezacaftor/ivacaftor (TEZ/IVA) group and 45 in the placebo group completed the study.
The primary objective of the study was to evaluate the frequency of specific respiratory adverse events: chest discomfort, shortness of breath, chest tightness, asthma, bronchospasm, bronchial hyperreactivity, and wheezing. In the TEZ/IVA group, 7 (14%) participants experienced one of these respiratory adverse events. In the placebo group, 10 (21.3%) participants experienced one of these respiratory events. The most common of these respiratory events was shortness of breath (TEZ/IVA: 5 [10.0%]; placebo: 5 [10.6%]). The only respiratory adverse event that occurred more frequently in the TEZ/IVA group compared with the placebo group was respiratory chest tightness; TEZ/IVA: 3 [6.0%]; placebo: 1 [2.1%]), but this event was uncommon and the difference was not significant.
A small improvement of 2.2% ppFEV1 in lung function was seen in the TEZ/IVA group compared with a -0.6% ppFEV1 decline in the placebo group. Overall, participants in this study had few respiratory side effects and they did not occur more often in those treated with TEZ/IVA compared with placebo.
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Citation:
J Cyst Fibros 2020;DOI 10.1016/j.jcf.2020.06.001
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:
84 days -
Number of Study Visits:
5
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):
Two Copies F508del -
FEV1% Predicted:
25 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Must have two copies of the F508del CFTR mutation and have previously discontinued treatment with Orkambi
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