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Restore CFTR Protein Completed with Results
Phase 3 study of Vertex 661 and ivacaftor in people with CF who have one copy of the F508del-CFTR mutation and a second CFTR mutation predicted to have residual function (Vertex VX-661-108)
This study evaluated the safety and effectiveness of the drug VX-661 in combination with ivacaftor (Kalydeco®) versus ivacaftor alone versus placebo. This study was for people with CF who have one copy of the F508del CFTR mutation and a CFTR mutation associated with residual CFTR function. The study included two sequential treatment periods. Participants were randomized to receive two out of three possible treatment regimens : 1) VX-661 plus ivacaftor combination (100mg of VX-661 once daily and 150 mg of ivacaftor every 12 hours), 2) ivacaftor alone (150 mg of ivacaftor every 12 hours), or 3) placebo.
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
Only male and female subjects aged 12 years or older with CF, heterozygous for the F508del-CFTR mutation, and a second allele with a CFTR mutation predicted to have residual function are included. Eligible mutations for this study include: 2789+5G->A, D110E, R352Q, 3849+10kbC->T, D110H, A455E, 3272-26A->G, R117C, D579G, R1070W, 711+3A->G, E193K, S945L, F1074L, E56K, L206W, S977F, D1152H, P67L, F1052V, D1270N, R74W, R347H, K1060T, E831X
Study Results
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What We Learned:
This study found that participants who received VX-661 plus ivacaftor had significantly increased lung function (FEV1) and reported higher quality of life (CFQ-R) when compared with ivacaftor alone or compared with placebo. Overall, the safety profile was similar between all three treatment groups.
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Primary Findings:
Effectiveness:
This study was conducted between March 17th, 2015 and February 16th, 2017. Of the 248 participants enrolled in the study, 234 (95%) completed both treatment periods. The primary objective of the study was to evaluate the effectiveness of VX-661 plus ivacaftor and ivacaftor alone as compared to placebo. Both treatment with VX-661 plus ivacaftor and ivacaftor alone resulted in significant improvements in lung function (absolute change in ppFEV1) as compared with placebo (6.8% for VX-661 plus ivacaftor; 4.7% for ivacaftor alone, p<0.001). Additionally, when comparing VX-661 plus ivacaftor to ivacaftor alone, VX-661 plus ivacaftor resulted in a significantly greater improvement in lung function than ivacaftor alone (p<0.001).
Researchers also looked at a participant self-reported quality of life questionnaire (CFQ-R). Participants taking VX-661 plus ivacaftor reported higher scores associated with quality of life than those taking ivacaftor alone (11.1 points versus 9.7 points, p<0.001).
Safety:
Overall, the safety profile was similar across all treatment groups and the placebo group. Most adverse events were mild or moderate. No participants discontinued due to VX-661 plus ivacaftor.
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Citation:
N Eng J Med ;DOI 10.1056/NEJMoa1709847 [Epub ahead of print]
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:
33 weeks -
Number of Study Visits:
12
Additional Information
-
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
Only male and female subjects aged 12 years or older with CF, heterozygous for the F508del-CFTR mutation, and a second allele with a CFTR mutation predicted to have residual function are included. Eligible mutations for this study include: 2789+5G->A, D110E, R352Q, 3849+10kbC->T, D110H, A455E, 3272-26A->G, R117C, D579G, R1070W, 711+3A->G, E193K, S945L, F1074L, E56K, L206W, S977F, D1152H, P67L, F1052V, D1270N, R74W, R347H, K1060T, E831X
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