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Restore CFTR Protein Completed with Results
Ivacaftor in People with CF who have a Non G551D Gating Mutation (KONNECTION) (Vertex VX-770-111)
The purpose of this trial was to look at the safety and effectiveness of ivacaftor in people with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D). Participants were randomized to 1 of 2 treatment groups: 1) ivacaftor 150mg for 8 weeks followed by eight weeks of placebo or 2) placebo for eight weeks followed by eight weeks of ivacaftor 150 mg. The study also included a 16 week open-label extension where all participants received ivacaftor.
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:
40 to 105%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Eligible subjects must have at least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349. Eligible subjects cannot have the G551 mutation in either allele.
Study Results
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What We Learned:
Study results show that ivacaftor was associated with a significant improvement in lung function compared to placebo. Additionally, the frequency of adverse events was similar between treatment groups and across both parts of the study.
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Primary Findings:
Effectiveness:
This international multicenter study was conducted between July 2012 and October 2013. A total of 39 participants were enrolled and completed 24 weeks of treatment. The primary efficacy outcome was absolute change in percent predicted FEV1 through eight and 24 weeks of ivacaftor treatment. During the placebo controlled part of the study, participants receiving ivacaftor demonstrated a significant improvement in lung function (measured as absolute percent predicted FEV1) (+7.5%) compared to the placebo group (-3.2%).
Improvements were also noted in body mass index, sweat chloride values and CFQ-R respiratory domain quality of life scores while participants received ivacaftor. The improvements observed during the placebo controlled portion of the study were maintained during the open-label period.
Safety:
Ivacaftor was well-tolerated; adverse events reported by 83.8% of participants while receiving placebo vs. 73.7% of participants while receiving ivacaftor.
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Citation:
J Cyst Fibros 2014;DOI 10.1016/j.jcf.2014.09.005;13(6):674-680
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:
40 weeks -
Number of Study Visits:
11
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:
40 to 105%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Eligible subjects must have at least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349. Eligible subjects cannot have the G551 mutation in either allele.
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