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Restore CFTR Protein Completed with Results
VX-661 alone and in combination with ivacaftor in people with cystic fibrosis (Vertex VX-661-101)
This study looked at the safety and effectiveness of multiple dose levels of VX-661 alone and in combination with ivacaftor (Kalydeco®). Part A of this study was for people with CF who have two copies of the F508del CFTR mutation and Part B of the study was for people with CF who have one copy of the F508del CFTR mutation and one copy of the G551D CFTR mutation. In Part A, fourteen different treatment combinations were tested. Participants were randomized to receive VX-661 alone, VX-661 in combination with ivacaftor, or placebo. In Part B, participants received either VX-661 (100mg) or placebo once daily in addition to the ivacaftor that they were already receiving for clinical care.
Eligibility
See other primary eligibility criteria for more information.
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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
Participants were required to have the F508del-CFTR gene mutation on at least one allele.
Study Results
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What We Learned:
In this study, VX-661 was generally well tolerated, both when taken alone and in combination with ivacaftor. Additionally, in participants with two copies of F508del, sweat chloride was reduced and lung function improved at the two highest doses tested. In participants with one copy of G551D and one copy of F508del, sweat chloride was reduced; however, there was no significant improvement in lung function.
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Primary Findings:
Effectiveness:
This study was conducted between February 2012 and March 2014. Of the 190 participants enrolled in the study, 185 (97.4%) completed the study. In Part A, 194 participants were randomized to receive one of four doses of VX-661 alone (N=33), one of seven dose combinations of VX-661 in combination with ivacaftor (N=123), or placebo (N=38). In Part B, 18 participants were randomized to receive either VX-661 with ivacaftor (N=14) or placebo with ivacaftor (N=4). The primary efficacy endpoint was change in sweat chloride through Day 28 for both Part A and Part B. In Part A (the group with two copies of F508del), a reduction in sweat chloride was observed with VX-661 (100mg) in combination with ivacaftor (150mg) when compared with placebo (p<0.05). In Part B (participants with one copy of G551D and already being treated with ivacaftor), treatment with VX-661 resulted in a reduction in sweat chloride when compared with placebo (-17.20, p<0.05).
Researchers also measured absolute change in lung function. In Part A, significant improvements in lung function (absolute change in ppFEV1) were observed at the two higher doses of VX-661 in combination with ivacaftor (100mg and 150mg) when compared with placebo (p<0.05), with the largest increase observed at 100mg of VX-661 (3.89 percentage points). In Part B, changes in lung function were not significant for VX-661 added to existing use of ivacaftor when compared with placebo.
Safety:
Overall, VX-661 was generally well tolerated, both when taken alone and in combination with ivacaftor.
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Citation:
Am J Respir Crit Care Med ;DOI 10.1164/rccm.201704-0717OC [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
Clinical trials can be observational or interventional. In an observational study, participants are being observed while receiving routine care. In an interventional study, participants receive one or more treatments (interventions) or a placebo so that researchers can evaluate the effects on the participant’s health.
Interventional -
Randomized Study: ?more info
A clinical trial in which participants are assigned by chance to one of two or more treatment arms.
Yes -
Placebo Controlled: ?more info
A clinical trial in which a drug is studied by giving an inactive substance (a placebo) to one group of participants, while the drug being tested is given to another.
Yes -
Length of Participation:
84 days -
Number of Study Visits:
9
Additional Information
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Phase: ?more info
The steps involved in development of new drugs. Phase I focuses on initial safety in people. Phase II studies evaluate safety, correct dose and early signs of drug effect. Phase III studies are the final large studies of safety and drug effectiveness before a drug can be reviewed for approval by the FDA. Phase IV studies evaluate a drug after it has been approved by the FDA.
Phase Two -
Study Sponsor: ?more info
The company or organization that initiates and conducts the study.
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
Participants were required to have the F508del-CFTR gene mutation on at least one allele.
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