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Restore CFTR Protein Completed with Results
Open-label study of ivacaftor in people with CF who have a non-G551D CFTR gating mutation (Vertex VX-770-112)
This study evaluated the long-term safety of ivacaftor (Kalydeco®) in participants who have a non-G551D CFTR gating mutation. This study was for people who had participated in one of the VX11-770-110, VX12-770-111, or VX12-770-113 studies.
Participants were given the option to enroll in either an ivacaftor group or an observational group. Participants enrolled in the ivacaftor group received ivacaftor (150mg every 12 hours) for up to 104 weeks. Participants enrolled in the observational group did not receive ivacaftor and were observed for up to two years.
Eligibility
See other primary eligibility criteria for more information.
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Age:
6 Years and Older -
Mutation(s):
One Copy F508del or 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
Participation in either VX-770-110 or VX-770-111. To be eligble for the treatment arm eligible subjects must have completed treatment in either study. To be eligible for the observation arm, subjects must have completed at least 4 weeks of treatment in either VX-770-110 or VX-770-111.
Study Results
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What We Learned:
This study found that ivacaftor appeared to be generally well-tolerated at a dose of 150mg. It resulted in an increase in lung function, similar to what was seen in previous studies (VX11-770-110, VX12-770-111, and VX12-770-113).
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Primary Findings:
Effectiveness:
Safety:
This study was conducted between February 2013 and May 2016. In the ivacaftor group, 121 participants were enrolled and 35 completed the study (29%). In the observational group, 4 participants were enrolled and 3 completed the study (75%). The majority (n=60, 70%) of participants in the ivacaftor group that did not complete the study, withdrew because ivacaftor in commercial form (Kalydeco®) became available during the study and participants chose to switch.
The primary endpoint was safety and tolerability from the beginning of the study to week 108. Researchers assessed the number of participants who experienced an adverse event that developed during treatment (TEAEs) or a serious adverse (SAEs) event.
Ivacaftor was generally well-tolerated at a dose of 150mg, similar to what was seen in previous studies (VX11-770-110, VX12-770-111, and VX12-770-113). Additionally, ivacaftor improved lung function from the beginning of the study to week 104, as measured by absolute change in ppFEV1 (participants enrolled from the 110 study= 4.6%, 111 study= 4.9%, and 113 study: 6.3%).
These results have been provided from Clinicaltrials.gov.
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Citation:
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
No -
Placebo Controlled: ?more info
No -
Length of Participation:
2 years -
Number of Study Visits:
7
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:
6 Years and Older -
Mutation(s):
One Copy F508del or 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
Participation in either VX-770-110 or VX-770-111. To be eligble for the treatment arm eligible subjects must have completed treatment in either study. To be eligible for the observation arm, subjects must have completed at least 4 weeks of treatment in either VX-770-110 or VX-770-111.
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