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Restore CFTR Protein Completed with Results
Phase 3 study of Vertex 661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-107)
This study looked at the safety and effectiveness of the drug VX-661 in combination with ivacaftor (Kalydeco®) compared to placebo. This study was for people with CF who have one copy of the F508del CFTR mutation and a second CFTR mutation that is not likely to respond to this drug therapy. This study planned to enroll approximately 300 participants. Participants received either VX-661 plus ivacaftor in the morning and an additional dose of only ivacaftor in the evening or placebo both in the morning and in the evening for 12 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
Mutations eligible for this study include: Nonsense mutations: Q39X, W57X, E60X, R75X, E92X, Q98X, Y122X, L218X, Q220X, C276X, Q290X, G330X, W401X, Q414X, S434X, S466X, S489X, Q493X, W496X, Q525X, G542X, Q552X, R553X, E585X, G673X, R709X, K710X, L732X, R764X, R785X, R792X, E822X, W846X, R851X, Q890X, S912X, W1089X, Y1092X, E1104X, R1158X, R1162X, S1196X, W1204X, S1255X, W1282X, Q1313X Canonical splice mutations: 621+1G->T, 711+1G->T, 711+5G->A, 712-1G->T, 405+1G->A, 405+3A->C, 406-1G->A, 621+1G->T, 1248+1G->A, 1341+1G->A, 1717-1G->A, 1811+1.6kbA->G, 1811+1G->C, 1812-1G->A, 1898+1G->A, 2622+1G->A, 3120+1G->A, 3120G->A, 3850-3T->G, 3850-1G->A, 4005+1G->A, 4374+1G->T Frameshift mutations: 663delT, 2183AA->G, CFTRdel2,3, 3659delC, 394delTT, 2184insA, 3905insT, 2184delA, 1078delT, 1154insTC, 2183delAA->G, 2143delT, 1677delTA, 3876delA, 2307insA, 4382delA7, 4016insT, 2347delG, 3007delG, 574delA, 2711delT, 3791delC, CFTRdele22-23, 457TAT->G, 2043delG, 2869insG, 3600+2insT, 3737delA, 4040delA, 541delC Missense mutations: A46D6, T338I8, R347P, L927P, G85E, S341P6, L467P6, I507del, V520F, A559T6, R560T, R560S, A561E, Y569D6, L1065P, R1066C, R1066M, L1077P6, H1085R6, M1101K, N1303K
Study Results
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What We Learned:
The study was stopped by the CFF Data Safety Monitoring Board (DSMB) before completion based on the apparent lack of benefit in this participant population.
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Primary Findings:
Effectiveness:
This study was conducted between August 2015 and April 2016. The study enrolled 168 participants (N=85 placebo, N=81 VX-661 plus ivacaftor).
After 150 participants were enrolled and completed at least 8 weeks of dosing, the CFF DSMB conducted a data analysis. This analysis looked at how effective and safe the drug was in the first half of study participants to determine whether to stop the study or to continue enrolling participants into the study.
The analysis showed that the combination of VX-661 and ivacaftor in this participant population did not result in an adequate improvement in lung function to support continuing the study (-0.1% FEV1 in placebo vs. 1% FEV1 in VX-661 plus ivacaftor). The CFF DSMB recommended that Vertex stop the study.
Safety:
No safety concerns were noted in the CFF DSMB's review of the data.
These data are preliminary and have not been peer-reviewed. These data were provided by ClinicalTrials.gov and a Vertex Press Release.
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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
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
20 weeks -
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:
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
Mutations eligible for this study include: Nonsense mutations: Q39X, W57X, E60X, R75X, E92X, Q98X, Y122X, L218X, Q220X, C276X, Q290X, G330X, W401X, Q414X, S434X, S466X, S489X, Q493X, W496X, Q525X, G542X, Q552X, R553X, E585X, G673X, R709X, K710X, L732X, R764X, R785X, R792X, E822X, W846X, R851X, Q890X, S912X, W1089X, Y1092X, E1104X, R1158X, R1162X, S1196X, W1204X, S1255X, W1282X, Q1313X Canonical splice mutations: 621+1G->T, 711+1G->T, 711+5G->A, 712-1G->T, 405+1G->A, 405+3A->C, 406-1G->A, 621+1G->T, 1248+1G->A, 1341+1G->A, 1717-1G->A, 1811+1.6kbA->G, 1811+1G->C, 1812-1G->A, 1898+1G->A, 2622+1G->A, 3120+1G->A, 3120G->A, 3850-3T->G, 3850-1G->A, 4005+1G->A, 4374+1G->T Frameshift mutations: 663delT, 2183AA->G, CFTRdel2,3, 3659delC, 394delTT, 2184insA, 3905insT, 2184delA, 1078delT, 1154insTC, 2183delAA->G, 2143delT, 1677delTA, 3876delA, 2307insA, 4382delA7, 4016insT, 2347delG, 3007delG, 574delA, 2711delT, 3791delC, CFTRdele22-23, 457TAT->G, 2043delG, 2869insG, 3600+2insT, 3737delA, 4040delA, 541delC Missense mutations: A46D6, T338I8, R347P, L927P, G85E, S341P6, L467P6, I507del, V520F, A559T6, R560T, R560S, A561E, Y569D6, L1065P, R1066C, R1066M, L1077P6, H1085R6, M1101K, N1303K
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