Page Title
Clinical Trial Finder
Restore CFTR Protein Completed with Results
Study of VX-661 combined with ivacaftor in adults with cystic fibrosis (Vertex VX-661-103)
This study evaluated the safety and the best way to dose VX-661 in combination with ivacaftor. This study was for adults with CF who have two copies of the F508del CFTR mutation.
The first part of this study was placebo-controlled. Participants were randomly assigned to receive either VX-661 with ivacaftor, or a placebo. Participants who received VX-661 took one of two doses: either 50mg twice a day or 100mg once a day. All participants who received VX-661 also received 150mg of ivacaftor twice a day. The second part of the study was an open-label extension study. All participants first stopped taking the study treatment for a 12-week washout period. All participants then received once daily VX-661 (100 mg) plus twice daily ivacaftor (150mg) for 48 weeks.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
18 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
Must be homozygous for F508del-CFTR
Study Results
-
What We Learned:
This study showed that VX-661 plus ivacaftor was well-tolerated regardless of how it was dosed. The dosing regimen of 100mg of VX-661 given once a day, plus 150mg of ivacaftor given twice a day was selected to move forward into phase 3 studies.
-
Primary Findings:
Effectiveness:
Safety:
This study was conducted between April 2014 and May 2016. Overall, 39 people participated in the placebo-controlled part of the study. 27 of those also participated in the open-label extension.
VX-661 plus ivacaftor was well-tolerated regardless of how it was dosed. Changes in lung function were used to define the best way to dose the combination. Participants who received 100mg of VX-661 once a day plus ivacaftor had a 3% improvement in lung function, measured FEV1 percent predicted. Participants who received 50mg of VX-661 twice a day plus ivacaftor had an improvement of 0.9% in FEV1. The improvement in FEV1 was confirmed during the open label extension, with an improvement of 2.7%. The once daily VX-661 (100mg) plus twice daily ivacaftor was selected as the best dosing regimen to move forward into future studies.
These results have been provided from Clinicaltrials.gov.
-
Citation:
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
20 weeks -
Number of Study Visits:
8
Additional Information
-
Phase: ?more info
Phase Two -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
18 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
Must be homozygous for F508del-CFTR
Sign up for clinical trial alerts
Get email updates about clinical trials that matter to you.
Check the Drug Development Pipeline
We’re attacking CF from every angle. Learn about the status of CF drugs in development.
Learn More