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Phase 2 study of VX-371 in people with CF who are currently taking lumacaftor/ivacaftor (Vertex VX-371-101)
This study evaluated the safety and effectiveness of the inhaled drug VX-371 (formerly P-1037) in combination with hypertonic saline in participants who have two copies of the F508del CFTR mutation and are taking lumacaftor/ivacaftor (Orkambi®).
Participants, taking lumacaftor/ivacaftor, were randomized to 1 of 4 treatment sequences. Each treatment sequence consisted of: Treatment Period 1 followed by a Washout Period and then a Treatment Period 2. Each period lasted 28 days. The first treatment sequence was VX-371+ hypertonic saline, washout, then hypertonic saline. The second sequence was hypertonic saline, washout, then VX-371 plus hypertonic saline. The third sequence was VX-371 plus placebo, washout, then placebo. The fourth sequence was placebo, washout, then VX-371 plus placebo. Participants remained on lumacaftor/ivacaftor throughout the study.
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
Patients must be homozygous for the F508del CFTR Mutation, and being treated with Orkambi
Study Results
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What We Learned:
This study found that participants taking VX-371 plus hypertonic saline did not have a significant increase in lung function (measured by percent predicted FEV1). The addition of VX-371 with or without hypertonic saline was generally well tolerated in participants already taking lumacaftor/ivacaftor. No further development in CF is planned at this time.
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Primary Findings:
Effectiveness:
This study enrolled 142 participants. The primary objective was safety and the effect of VX-371 plus hypertonic saline on lung function (measured by percent predicted FEV1). The study did not meet its primary objective. Participants who received VX-371 plus hypertonic saline on top of lumacaftor/ivacaftor had a small increase in lung function (0.1% ppFEV1). Participants who received only hypertonic saline on top of lumacaftor/ivacaftor had a slight decrease in lung function (0.1% ppFEV1).
Safety:
The addition of VX-371 with or without hypertonic saline was generally well tolerated in participants already taking lumacaftor/ivacaftor.
These results have been provided from a Vertex Press Release and have not been peer reviewed.
No further development in CF is planned at this time.
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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:
98 days -
Number of Study Visits:
12
Additional Information
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Phase: ?more info
Phase Two -
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
Patients must be homozygous for the F508del CFTR Mutation, and being treated with Orkambi

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