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Restore CFTR Protein Completed with Results
Phase 3 study of VX-661 and ivacaftor in people with cystic fibrosis (Vertex VX-661-106)
This study evaluated the safety and effectiveness of the drug VX-661 in combination with ivacaftor (Kalydeco®) compared with placebo. This study was for people with CF who have two copies of the F508del CFTR mutation. The study planned to enroll a total of 510 participants. Participants randomly received either placebo (twice daily) or VX-661 plus ivacaftor in the morning and an additional dose of only ivacaftor in the evening for 24 weeks.
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:
40 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Subjects must have the F508del-CFTR gene mutation in both alleles
Study Results
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What We Learned:
This study found that participants who received VX-661 in combination with ivacaftor showed improved lung function (FEV1), a decreased rate of pulmonary exacerbations, and decreased rate of pulmonary exacerbations leading to hospitalization and treatment with intravenous antibiotics when compared with placebo. Overall, the safety profile for participants treated with VX-661 plus ivacaftor was similar to that of those treated with placebo.
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Primary Findings:
Effectiveness:
This study was conducted between January 30th, 2015 and January 20th, 2017. Of the participants enrolled and who completed the entire 24-week trial, 235 participants received VX-661 plus ivacaftor and 240 received placebo. The primary objective of the study was to evaluate the effectiveness of VX-661 plus ivacaftor compared with placebo. Participant who received VX-661 plus ivacaftor resulted in a significantly greater improvement in lung function (absolute change from baseline in ppFEV1) than occurred in the participants that received placebo (3.4% FEV1 versus -0.6% FEV1, 95% CI, p<0.001).
Researchers also evaluated the effect of VX-661 plus ivacaftor on pulmonary exacerbations (PEx). They found that the rate of PEx was significantly lower in participants who received VX-661 plus ivacaftor compared to those that received placebo (0.64 vs. 0.99 PEx per year, p=0.005. Additionally the rate of PEx that led to hospitalizations or treatment with intravenous (IV) antibiotics was lower in the VX-661 plus ivacaftor group than in the placebo group (0.29 vs. 0.54 per year, 95% CI). There was no significant difference in BMI at week 24 between the VX-661 plus ivacaftor and the placebo group. In both groups, BMI increased.
Safety:
Overall, the safety profile between VX-661 plus ivacaftor and the placebo groups were similar, with less frequent serious adverse events in the VX-661 plus ivacaftor group (12.4%) compared with placebo (18.2%).
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Citation:
N Eng J Med ;DOI 10.1056/NEJMoa1709846 [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:
32 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:
12 Years and Older -
Mutation(s):
Two Copies 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
Subjects must have the F508del-CFTR gene mutation in both alleles
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